COMMITTED TO PATIENTS

PROCYSBI® [mercaptamine bitartrate (cysteamine) gastro-resistant hard capsules] recommended for routine commissioning by NHS England for eligible patients with nephropathic cystinosis

 NHS England has recommended mercaptamine bitartrate (cysteamine) gastro-resistant hard capsules, also referred to as “delayed-release mercaptamine bitartrate” for routine commissioning for eligible patients aged one year and above with nephropathic cystinosis.¹ Nephropathic cystinosis affects around 1 in 100,000 to 200,000 live births, with approximately 200 people living with the condition in the UK and two to three new cases diagnosed each year.^{1, 2} The treatment provides an additional cystine-depleting therapy option that can be taken twice-daily, compared to four-times-daily dosing every six hours for the currently commissioned immediate-release formulation.¹  Manchester, UK – 5th August 2026 – Chiesi UK and Ireland welcome NHS England’s decision to recommend delayed-release mercaptamine bitartrate (a cystine-depleting agent) for routine commissioning for eligible patients living with nephropathic cystinosis. The decision means eligible patients aged one year and above who meet the defined clinical criteria* can access an additional treatment option through NHS England specialised services, expanding choice for people living with this ultra-rare condition. Care for people with nephropathic cystinosis is often complex, time-consuming and demanding for both patients and their families.^2,3 With treatment options previously limited, requiring multiple daily doses, this treatment option with a twice-daily dosing schedule, can potentially help reduce disruption to daily routines for patients and carers, improve adherence, delay complications and improve patients’ quality of life.¹ "We welcome NHS England's decision to routinely commission delayed-release mercaptamine bitartrate for eligible people with nephropathic cystinosis," said David Game, Consultant Nephrologist and Clinical Lead for the Delayed-Release Mercaptamine Policy Proposal. "Nephropathic cystinosis is a lifelong condition, and this decision marks an important milestone for the cystinosis community in England. By providing eligible patients and their clinicians with access to an additional treatment option, it supports treatment decisions based on individual patient needs and reflects the collaborative efforts of clinicians, patients, families and patient organisations throughout the policy process." Nephropathic cystinosis is an ultra-rare, progressive condition affecting around 200 people in the UK.¹ The disease is caused by the accumulation of cystine, an amino acid, within cells throughout the body.¹ This can lead to tissue and organ damage, particularly in the eyes and kidneys.^1,4 Around 150 patients are estimated to be eligible for delayed-release mercaptamine bitartrate under NHS England's new commissioning policy.¹ David Garzón, Senior Director, Rare Diseases at Chiesi UK and Ireland, added: “This is an important milestone for the nephropathic cystinosis community in England, providing an additional treatment option for these patients with a twice-daily dosing regimen, that can help alleviate some of the treatment burden for patients and their families. Following earlier reimbursement decisions in Northern Ireland, Wales and Scotland, this decision marks an important step towards more equitable access across the UK, reflecting the collaborative efforts of clinicians, patient organisations, patients and families.”  Delayed-release mercaptamine bitartrate is approved across the UK and Europe, including in France, Italy, Germany and Ireland. Following NHS England's decision to routinely commission the treatment for eligible patients 1 year and above with nephropathic cystinosis, the medicine is now routinely available across all UK nations for patients meeting local access criteria. Comments from the Patient Community  Will Newman, Chairperson of Cystinosis Foundation UK, said: “We are delighted to see this decision, which represents an important step forward for the nephropathic cystinosis community in England. The approval means patients and carers can now explore whether this formulation better suits their individual circumstances, lifestyles, and tolerability needs.”   Laura Smith Van Carroll, Head of Insight & Advocacy at Metabolic Support UK, said: “We are delighted that people living with cystinosis in England will now have access to Procysbi, bringing equitable treatment choice across the UK after many years of availability in Wales, followed by Scotland and more recently Northern Ireland. For many families, this approval has the potential to reduce the burden of frequent dosing, allowing uninterrupted sleep, and giving people greater choice in how they manage this lifelong condition. While we welcome this decision, we hope future CPAG processes will provide clearer timelines and secure funding alongside approval, helping people with rare conditions access new treatments more quickly and with greater certainty.”   About Procysbi® (delayed-release mercaptamine bitartrate) Procysbi (delayed-release mercaptamine bitartrate) is a beaded, enteric-coated, delayed-release form of cysteamine, in which the microspheronised beads are further encapsulated in hard gelatin to allow oral administration every 12 hours.⁴ The treatment is administered twice-daily.  Delayed-release mercaptamine bitartrate is available in either 25 mg or 75 mg gastro-resistant hard capsules of cysteamine.⁵ Therapy should be initiated as early as possible following diagnosis and continued throughout life.  About Chiesi Group     Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.  By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet verified standards for social and environmental business practices. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,900 employees. The Group’s research and development centre in Parma works alongside six other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.  For further information please visit www.chiesi.uk.com Media contacts:  Yasmin Ghariani, Chiesi UK and Ireland Head of External Communications Phone: (+44) 161 488 5555 Email: y.ghariani@chiesi.com   UK-PCB-2600003 | July 2026   References   * Inclusion criteria can be found in the guidance here: Report template - NHSI website.     NHS England. Clinical Commissioning Policy: Delayed-release mercaptamine bitartrate for patients with nephropathic cystinosis (Age > 1 years) [URN 2338]. Available at: Report template - NHSI website. Cystinosis Foundation UK. What is Cystinosis? Available at: What Is Cystinosis? – Cystinosis Foundation UK. Available at: https://www.cystinosis.org.uk/learn-more/what-is-cystinosis/ [Last accessed July 2026] Golestaneh L, Ames EG, Doyle MH, et al. Improving Lifelong Comprehensive Care Coordination in Nephropathic Cystinosis: Multidisciplinary Perspectives. Kidney Int Rep. 2026;11(3):103735. doi:10.1016/j.ekir.2025.103735. PROCYSBI 75 mg gastro-resistant hard capsules - Summary of Product Characteristics (SmPC) - (emc). Medicines.org.uk. 2025. Available at: https://www.medicines.org.uk/emc/product/2080/smpc   All Wales Medicines Strategy Group, Final Appraisal Recommendation Advice number: 0922, April 2022. Ariceta G, Giordano V, Santos F. Effects of long-term cysteamine treatment in patients with cystinosis. Pediatr Nephrol. 2019 Apr;34(4):571-578.

Targeted nurse-led clinic launches in Manchester to promote personalised transplant care and tackle inequity in post-transplant outcomes

Between 2024 and 2025, there were over two hundred and fifty kidney transplants performed in Manchester.¹ Around 30-40% of kidney transplants do not last beyond 10 years, meaning many patients require further treatment or another transplant.² Outcomes after kidney transplantation are not experienced equally - Black transplant recipients have a 34% higher risk of graft loss, young people aged 14-23 face a 41% higher risk of transplant failure and return to dialysis, and women may experience reduced long term survival benefit.^2,3,4,5,6 A targeted, nurse-delivered transplant clinic is being introduced at Manchester Royal Infirmary through PIONEER, a collaborative working project between Chiesi UK and Ireland (Chiesi) and Manchester University NHS Foundation Trust, to support more personalised post transplant care.   Manchester, UK – 3rd August 2026 – Chiesi and Manchester University NHS Foundation Trust have today announced the launch of a targeted nurse-led transplant clinic at Manchester Royal Infirmary as part of the PIONEER (Post-Transplant Initiative for Optimising Nurse-led Engagement & Empowering Recovery) project. The clinic introduces a nurse-delivered model, providing a more personalised and tailored approach to follow-up care for post-transplant patients from deprived populations in response to growing evidence that outcomes after kidney transplantation are not experienced equally across patient groups.²   For many, a kidney transplant is a life-line, a second chance they have waited for, often for years.⁷ On average, a kidney transplant from a living donor lasts for 20-25 years, while one from a deceased donor lasts 15-20 years.⁸ However, long-term outcomes can vary, with around 30–40% of kidney transplants not lasting beyond 10 years.² When this happens, patients typically require dialysis or a further transplant to restore kidney function.   Mr David Van Dellen, Consultant Transplant & General Surgeon, Manchester Centre for Transplantation, Manchester University NHS Foundation Trust said, “our programme recognises that surgical success is only the beginning - personalised, culturally sensitive post-transplant care is essential to helping each patient achieve the best possible outcome from their transplant. By embedding equity into every stage of the transplant pathway, we aim to ensure that a transplant in Manchester means the same chance of a healthy future for every patient, regardless of background. The PIONEER project is an integral step towards this ambition, providing more personalised support and helping patients protect their transplant for the long-term.”  In the UK, registry data shows that Black kidney transplant recipients experience poorer outcomes than white patients, including higher rates of graft loss and delayed graft function - meaning the transplanted kidney is more likely to fail earlier or take longer to begin working properly after surgery.³ Younger patients are also at increased risk of transplant failure and a return to dialysis, with a 41% higher risk in those aged 14-23 compared to adults in their mid-30s to early 40s.^4,5 Evidence also suggests that women may experience reduced long-term survival benefit following transplantation compared to men.⁶   For many transplant recipients, these challenges are experienced within the wider context of social deprivation. Greater Manchester ranks among the most deprived areas in England, with a significant proportion of neighbourhoods in the most deprived decile.⁹ The clinic will serve patients from across the region, including many from other areas of high socioeconomic deprivation where additional barriers can affect long-term post-transplant care.    “A transplant can be life-changing, but it is not the end of the patient journey - and there is significant variation in the lives recipients go on to lead following a transplant,” said Shish Patel, Senior Director, Medical Affairs at Chiesi UK and Ireland. “Long-term outcomes are influenced by a range of clinical and wider social factors, and more targeted, personalised approaches are essential to improving post-transplant care for transplant recipients.”   Traditionally, post-transplant reviews at Manchester Royal Infirmary have been conducted by surgeons during routine follow-up appointments, reflecting established models of care. The PIONEER clinic introduces a targeted, nurse-led model designed to provide additional time for education, closer monitoring and personalised support, including tailored pharmacological and non pharmacological guidance. This approach also aims to strengthen shared decision making, ensuring patients have meaningful opportunities to share their experiences and priorities as part of their ongoing care. Patients eligible for the clinic are identified and selected by clinical teams at Manchester University NHS Foundation Trust based on a range of criteria.   "Every transplant recipient’s journey is unique, and this innovative nurse-led clinic provides a more personalised approach to post-transplant care,” said Malcolm Greenwood-Morgan, Clinical Nurse Specialist, Manchester University NHS Foundation Trust. “Through holistic assessment, medicines optimisation and tailored support, we can identify challenges and work with patients to address barriers that may affect their long-term health and transplant success. By focusing on what matters most to each patient, the clinic aims to improve treatment adherence, enhance wellbeing and quality of life, reduce health inequalities and empower patients to play an active role in achieving the best possible outcomes from their transplant.”   The PIONEER clinic, which launched today, is a 2-year project and will run until January 2028 and evaluate both clinical and patient-reported outcomes. Insights from the programme will be used to inform future approaches to personalised post-transplant care.    About PIONEER     PIONEER is a collaborative working project between Chiesi and Manchester University NHS Foundation Trust, designed to support more personalised post transplant care for post-transplant patients from deprived populations. As part of the project, a targeted nurse-led clinic is being introduced at Manchester Royal Infirmary, providing additional time for education, monitoring and tailored support alongside routine follow-up care. PIONEER commenced in January 2026 and will run for 24 months.    For more information visit: PIONEER | Chiesi Limited Website   About Manchester University NHS Foundation Trust (MFT)    Manchester University NHS Foundation Trust is the largest NHS Trust in the country and a leading provider of specialist healthcare services. Its ten hospitals are home to 28,000 staff including world class clinicians and academic staff committed to finding patients the best care and treatments. Its hospitals are Manchester Royal Infirmary, Saint Mary's Managed Clinical Service, Royal Manchester Children's Hospital, Manchester Royal Eye Hospital, University Dental Hospital of Manchester, Trafford General Hospital, Altrincham Hospital, Wythenshawe Hospital, Withington Hospital and North Manchester General Hospital. More information is available at www.mft.nhs.uk   About Chiesi Group      Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.    By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet verified standards for social and environmental business practices. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.    With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,900 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.    For further information please visit Home | Chiesi Limited Website    Media contacts:  Eireann Clapham, Chiesi UK External Communications Specialist  Phone: 07918 613855 Email: e.clapham@chiesi.com   UK-CHI-2600566 | July 2026    References NHS Blood and Transplant. Activity Report 2024/2025.  Available at: https://nhsbtdbe.blob.core.windows.net/umbraco-assets-corp/36694/activity-report-2024-2025-final.pdf.   British Transplantation Society (2023). UK Guideline for the Management of the Patient with a Failing Kidney. Available at: https://bts.org.uk/uk-guideline-for-the-management-of-the-patient-with-a-failing-kidney-transplant/.  Pisavadia B, Arshad A, Chappelow I, Nightingale P, Anderson B, Nath J, et al. Ethnicity matching and outcomes after kidney transplantation in the United Kingdom. Eller K, editor. PLOS ONE. 2018 Apr 13;13(4):e0195038. Hamilton AJ, Plumb LA, Casula A, Sinha MD. Associations with kidney transplant survival and eGFR decline in children and young adults in the United Kingdom: a retrospective cohort study. BMC Nephrology. 2020 Nov 18;21(1). Pankhurst T, Evison F, Mytton J, Williamson S, Kerecuk L, Lipkin G. Young adults have worse kidney transplant outcomes than other age groups. Nephrology Dialysis Transplantation [Internet]. 2020 Jun 1;35(6):1043–51. Available at: https://academic.oup.com/ndt/article/35/6/1043/5847834?login=true.  Katz-Greenberg G, Shah S. Sex and Gender Differences in Kidney Transplantation. 2022 Mar 1 [cited 2022 Jul 18];42(2):219–29. Available at: https://www.seminarsinnephrology.org/article/S0270-9295(22)00019-5/fulltext.  NHS Blood and Transplant. How Long Is the Wait for a kidney? Organ transplantation - NHS Blood and Transplant. 2021. Available at: https://www.nhsbt.nhs.uk/organ-transplantation/kidney/receiving-a-kidney/how-long-is-the-wait-for-a-kidney/. NHS Blood and Transplant. Kidney transplant FAQs. Organ transplantation. Available at: https://www.nhsbt.nhs.uk/organ-transplantation/kidney/is-a-kidney-transplant-right-for-you/kidney-transplant-faqs/.  CDRC. Index of Multiple Deprivation (IMD) | CDRC Data [Internet]. data.cdrc.ac.uk. Consumer Data Research Centre; 2020. Available at: https://data.cdrc.ac.uk/dataset/index-multiple-deprivation-imd.

Chiesi Global Rare Diseases Announces European Commission Approval of LOJUXTA® (lomitapide) ▼ Capsules for Paediatric Use in Homozygous Familial Hypercholesterolaemia (HoFH)

European Commission approval expands the indication of lomitapide in the European Union (EU) to include children 5 years of age and older with HoFH, an ultra-rare genetic disorder affecting LDL-cholesterol levels.    PARMA, Italy – June 5, 2026 – Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced that the European Commission (EC) has approved lomitapide capsules for use in children 5 years of age and older with Homozygous Familial Hypercholesterolaemia (HoFH), for use alongside diet and other lipid-lowering treatments, including LDL-apheresis where available.    The EC decision follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending the expanded indication. Lomitapide has been approved for use in the European Union (EU) for adult patients with HoFH since 2013.   "This European Commission approval marks an important milestone for children living with HoFH and their families," said Mitch Goldman, MD, PhD, SVP Research & Development, Chiesi Global Rare Diseases. "HoFH is a condition that can begin causing irreversible cardiovascular damage from the earliest years of life, and the unmet need remains for younger patients despite existing treatment options. By expanding the indication of lomitapide to children aged 5 years and older, we are offering families and their physicians a meaningful treatment option that is backed by robust clinical evidence. This approval reflects our deepest commitment: ensuring that having a rare disease never means being overlooked at any stage of life, across generations.”   The EC approval is based on evidence from a Phase 3, open-label, single-arm, multicentre study evaluating lomitapide in 43 pediatric participants aged 5 to 17 years with HoFH. The APH-19 study achieved its primary endpoint, demonstrating a mean 53.5% reduction in LDL‑C from baseline at week 24 (p<0.0001). Significant reductions (all p<0.0001) were also achieved in non‑HDL‑C, total cholesterol, VLDL‑C, apolipoprotein B, and triglycerides at week 24 (secondary outcomes). Adverse events were mostly mild, and gastrointestinal and hepatic in nature. Adverse events of special interest were reported for five (12%) patients (gastrointestinal in two patients and hepatic in three). One serious treatment-emergent adverse event was reported (also classed as an adverse event of special interest): an increase in hepatic enzymes, resulting in two dose interruptions, two dose reductions, and a repeated dose escalation. Overall, no new adverse event signals were identified, and the results were consistent with the known profile of lomitapide.¹   About HoFH Homozygous familial hypercholesterolaemia (HoFH) is a rare condition causing very high levels of cholesterol in the body since birth.² In HoFH, the arteries become clogged, with people affected often having more than 10 times the target, or acceptable level of so called bad cholesterol in their blood (LDL-C), leading to severe heart disease, premature atherosclerosis and cardiovascular events.² Early diagnosis and management in HoFH are critical, however, many patients remain undiagnosed and undertreated.^2,3   About lomitapide Lomitapide is a prescription-only medicine used along with a low-fat diet, exercise and other low-density lipoprotein (LDL) lowering medicines to reduce LDL-C in adults and children 5 years of age and older with a type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).⁴ Genetic confirmation of HoFH should be obtained whenever possible. Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolaemia (e.g., nephrotic syndrome, hypothyroidism) must be excluded.   About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.   By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.   With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.   About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.   Chiesi Global Rare Diseases Media Contact Sky Striar LifeSci Communications Email: sstriar@lifescicomms.com   References 1.    Masana, L., Zambon, A., Schmitt, C. P., Taylan, C., Driemeyer, J., Cohen, H., Buonuomo, P. S., Alashwal, A., Al-Dubayee, M., Kholaif, N., Diaz-Diaz, J. L., Maatouk, F., Martinez-Hervas, S., Mangal, B., Löwe, S., & Cunningham, T. (2024). Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): Results from the efficacy phase of an open-label, multicentre, phase 3 study. The Lancet Diabetes & Endocrinology, 12(12), 880–889.   2.    Borberg H. The lower the better: target values after LDL-Apheresis and semi selective LDL-elimination therapies. Transfus Apher Sci. 2013;48(2):203-206.   3.    Cuchel M, Raal FJ, Hegele RA, et al. 2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance. Eur Heart J. 2023;44(25): 2277-2291.   4.    European Medicines Agency. (2025, January 27). Lojuxta, INN-lomitapide: EPAR product information. https://www.ema.europa.eu/en/documents/product-information/lojuxta-epar-product-information_en.pdf    UK-CHI-2600344 | June 2026

Chiesi Global Rare Diseases to Launch the Second Edition of Find For Rare at 2026 ERA Congress to Advance Research in Lysosomal Storage Disorders

-- At ERA 2026, Chiesi will contribute to advancing the scientific understanding of Fabry disease and nephropathic cystinosis through real-world experience and clinical outcomes, supporting the needs of the community of today --   -- Chiesi will also launch the second edition of Find For Rare, an independently assessed, expert led research grant initiative that supports innovative research in Fabry disease, alpha-mannosidosis and nephropathic cystinosis to advance disease understanding and care for generations to come --   PARMA, Italy – June 3, 2026 – Chiesi Global Rare Diseases, a business unit of the Chiesi Group, today announced its participation in the 63rd European Renal Association (ERA) Congress, taking place June 3–6, 2026, in Glasgow, Scotland, re-affirming its long-standing commitment to advancing understanding and care in lysosomal storage disorders (LSDs) over time. At ERA 2026, this commitment comes to life through the Company’s contribution to ongoing scientific dialogue in areas such as Fabry disease and nephropathic cystinosis, while also extending beyond the congress through the launch of the second edition of Find For Rare, its research grant initiative designed to support future innovation in LSDs.   At ERA, Chiesi’s scientific contributions will highlight real-world experience and clinical outcomes across Fabry disease and nephropathic cystinosis, focusing on treatment tolerability and immunogenicity in Fabry disease and nephrology-led multidisciplinary coordination to optimize care and outcomes for cystinosis. Through continued research and collaboration across disciplines, Chiesi Global Rare Diseases strives to create lasting, meaningful impact for the rare disease community for generations to come. This commitment also extends to rare conditions with significant renal impact, such as primary hyperoxaluria type 1 (pH1), which is part of the company’s emerging pipeline focused on advancing innovative approaches for rare kidney diseases.   Building on this commitment, Chiesi Global Rare Diseases will launch, during ERA Congress, the second edition of Find For Rare -an independently assessed, expert led research grant initiative designed to support innovative research in three lysosomal storage disorders: Fabry disease, alpha-mannosidosis and nephropathic cystinosis. By providing funding opportunities for original research projects and fostering collaboration across the scientific community, Find For Rare aims to help advance understanding of these complex conditions and contribute to improving disease management over time. Through this initiative, Chiesi seeks to help enable the research that will shape future care, extending today’s scientific dialogue into meaningful progress for those who come next. For more information, visit www.findforare.com.   “Scientific progress in rare diseases is built over time, through continuous dialogue and collaboration,” said Enrico Piccinini, Senior VP Europe and International, Chiesi Global Rare Diseases. “At ERA, we are proud to contribute to advancing understanding of lysosomal storage disorders. At the same time, with Find For Rare, we aim to go one step further supporting innovative research that can expand knowledge and help shape the future of care for generations to come.”   About Fabry Disease Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the enzyme alpha-galactosidase A. This deficiency results in an accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body’s cells, affecting the heart, kidneys, skin, nervous system, and other organs.¹  Fabry disease can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke.²   The condition affects both males and females and can present from childhood through adulthood, often with delayed diagnosis or misdiagnosis. While Fabry disease is rare, early detection and access to appropriate treatment — such as enzyme replacement therapy or pharmacological chaperone therapy — are critical in managing symptoms and slowing disease progression.¹ About Cystinosis Cystinosis is an ultra-rare genetic condition which affects fewer than 2 people in every million.3 In people with cystinosis, an amino acid called cystine accumulates in a part of the cell called the lysosome, and the cells are unable to remove it.³ When cystine builds up, it forms crystals within lysosomes that can cause long-term damage to organs, including at first the kidneys, eyes, muscles, pancreas, and brain.³ This damage cannot be reversed, but it can be delayed or reduced.⁴ There are three types of cystinosis. We are focusing on Nephropathic Cystinosis (NC), which is the most severe form of Cystinosis (95% of all cases) affecting about 2,000 patients worldwide, ~700 in Europe, ~500 in the US.⁵ Because cystinosis is a multi-organ disease, its symptoms are very broad and diverse. Newborns with nephropathic cystinosis do not present any symptoms, but within the first months of life they often present signs that suggest their kidneys are not working as well as they should. This will result, sooner or later, depending on when the treatment starts, in the need for kidney transplant in all people living with nephropathic cystinosis forms. Patients may also have impaired growth, loss of appetite or rickets.^3,4 Although in nephropathic cystinosis, the kidneys are affected first, almost every organ in the body is at risk of damage,^3,4 Continuous, lifelong cystine accumulation potentially damages all organs and tissues, resulting in severe complications, such as kidney failure, blindness, muscle wasting, central nervous system damage, and reduced lung function.^3,4   About Lysosomal Storage Disorders LSDs are inborn errors of metabolism that are characterised by an abnormal build-up of substances in the body's cells as a result of enzyme deficiencies.⁶ The build-up of these substances can affect different parts of the body, including the skeleton, central nervous system (brain), lungs, heart, and eyes. Whilst there has been progress in clinical knowledge, more research in LSDs can be beneficial.⁶   About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The Company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.   By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet verified standards of social and environmental impact. The Company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.   With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,900 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. Chiesi Global Rare Diseases Media Contact Sky Striar LifeSci Communications Email: sstriar@lifescicomms.com   References 1)    Mehta, A., & Hughes, D. A. (2024). Fabry disease. In M. P. Adam, S. Bick, G. M. Mirzaa, et al. (Eds.), GeneReviews®. University of Washington, Seattle. 2)    Cleveland Clinic. Fabry disease: Symptoms & causes. 3)    Levtchenko, E., Servais, A., Hulton, S. A., Ariceta, G., Emma, F., Game, D. S., Lange, K., Lapatto, R., Liang, H., Sberro-Soussan, R., Topaloglu, R., Das, A. M., Webb, N. J. A., & Wanner, C. (2022). Expert guidance on the multidisciplinary management of cystinosis in adolescent and adult patients. Clinical kidney journal, 15(9), 1675–1684. 4)    Bäumner, S., & Weber, L. T. (2018). Nephropathic Cystinosis: Symptoms, Treatment, and Perspectives of a Systemic Disease. Frontiers in pediatrics, 6, 58. 5)    Nesterova, G., & Gahl, W. A. (2013). Cystinosis: the evolution of a treatable disease. Pediatric nephrology (Berlin, Germany), 28(1), 51–5 6)    NCBI. Lysosomal Storage Disease. Available at: https://www.ncbi.nlm.nih.gov/books/ UK-CHI-2600340 | June 2026

Chiesi Receives MHRA Approval for Additional Dosing Regimen of Elfabrio®▼ (pegunigalsidase alfa) 2mg/kg administered every-four-weeks in adults stable with an enzyme replacement therapy

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved an additional dosing regimen of 2mg/kg administered every-four-weeks for pegunigalsidase alfa in adults stable with an enzyme replacement therapy (ERT). Pegunigalsidase alfa is a long-term ERT for adult patients with Fabry disease – a rare disease affecting approximately 1 in 40,000 people in the UK.^1,2 This adjustment in dosing regimen would reduce the burden and frequency with which eligible UK adult Fabry patients require infusions, from once-every-two-weeks to once-every-four-weeks.   Manchester, UK – 14 May 2026 – Chiesi UK and Ireland has today announced that the MHRA has approved an additional 2mg/kg body weight every-four-week dosing regimen for pegunigalsidase alfa for Fabry adults stable with an ERT. This follows a recent positive European Medicines Agency (EMA) decision in March 2026.    Pegunigalsidase alfa is a long-term enzyme replacement therapy (ERT) for adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase) - a rare disease affecting approximately 1 in 40,000 people in the UK.^1,2 It was originally approved by the MHRA in August 2023, with a dosing regimen of 1mg/kg body weight administered every-two-weeks. The approval for the additional dosing regimen means that it would halve the number of infusions which eligible UK adult Fabry patients require, from approximately twenty-six per year to thirteen.     Derralynn Hughes, Professor of Experimental Haematology, Director of Research and Innovation, and Co-Clinical Director of the NCL Cancer Alliance said: “For many people living with Fabry disease, regular fortnightly infusions can have a significant impact on day-to-day life, affecting work, family routines, and overall quality of life. The patient community consistently highlights the need for treatment approaches that help reduce this burden where clinically appropriate, and we look forward to being able to offer infusions every-four-weeks to those who are stable on their current ERT, with appropriate monitoring.”   The MHRA approval is informed by results from an open-label, switch-over study, BRIGHT (formally PB-102-F50), designed to assess the adverse event profile, efficacy, and pharmacokinetics (PK) of the additional dosing regimen of pegunigalsidase alfa 2 mg/kg body weight every-four-weeks for 52 weeks,³  and its ongoing open-label extension study CLI-06657AA1-03 (formerly PB-102-F51).⁴   Bob Stevens, Group Chief Executive Officer of the MPS Society and Chief Executive Officer and Chair of Rare Disease Research Partners, said: “People living with Fabry disease plan their lives around treatment. The possibility of attending infusions once every four weeks instead of two would offer greater flexibility and may help ease some of the practical challenges that come with long-term treatment. It is encouraging to see continued efforts to deliver options that may better reflect the needs of the Fabry community.”   David Garzón, Senior Director, Rare Diseases, UK and Ireland said: “We are pleased to receive MHRA approval for the additional dosing option for pegunigalsidase alfa in adults stable with an ERT. This is a positive step forward in improving quality of life for patients and their families. Our sincere thanks to the community who have collaborated throughout the regulatory approval process and continue to stand side by side with us to benefit patient care.”    For patients switched to pegunigalsidase alfa 2 mg/kg body weight once-every-four-weeks, regular monitoring (e.g. after 3, 6, 12, 18 and 24 months) should be performed. Monitoring should include at least the evaluation of lyso-Gb3, renal (eGFR, proteinuria), cardiac (LVMi, NT-proBNP, troponin or ECG), and biochemical parameters. A change in any individual parameter should be interpreted in the context of the patient's overall clinical status, and clinically relevant deterioration should prompt re-evaluation of the treatment regimen.⁵  During the study, safety and tolerability profiles of the E4W dosing regimen were consistent with previousstudies, with no severe or serious treatment-related TEAEs and all infusion-related reactions being mild or moderate in severity.   Eligible people in the UK may be prescribed the new dosing regimen of pegunigalsidase alfa from the date of approval if determined appropriate by their healthcare professional.   About Fabry Disease    Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the enzyme alpha-galactosidase A. This deficiency results in an accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body’s cells, affecting the heart, kidneys, skin, nervous system, and other organs.⁶  Fabry disease can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke.⁷   The condition affects both males and females and can present from childhood through adulthood, often with delayed diagnosis or misdiagnosis. While Fabry disease is rare, early detection and access to appropriate treatment — such as enzyme replacement therapy or pharmacological chaperone therapy — are critical for long term disease management.⁶    About Pegunigalsidase alfa    Pegunigalsidase alfa, a PEGylated enzyme replacement therapy (ERT) to treat adults with Fabry disease, is a plant cell culture-expressed, and chemically modified stabilised recombinant version of the α–Galactosidase–A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short polyethylene glycol (PEG) moieties, resulting in a molecule with stable pharmacokinetic parameters. Pegunigalsidase alfa has been observed to have plasma half-life ranging from 53 – 134 hour.¹ Clinical studies have not shown that differences in pharmacological characteristics, including half-life, result in clinically meaningful differences in efficacy or clinical outcomes.   About Chiesi Group     Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.   By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.   With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.   For further information please visit www.chiesi.uk.com.   About Chiesi Global Rare Diseases    Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.    Chiesi media contact   Yasmin Ghariani, Chiesi UK Head of Communications Phone: (+44) 161 488 5555 Email: y.ghariani@chiesi.com   References Medicines and Healthcare products Regulatory Agency. (2026). SPC Pegunigalsidase alfa. Summary of Product Characteristics Kidney Care UK. (2026). Fabry disease (alpha-galactosidase A deficiency). Holida, M, et al., (2024). A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme. Journal of Inherited Metabolic Disease. doi:10.1002/jimd.12795.  Bernat et al., (2025). Extending the interval between pegunigalsidase alfa infusions in patients with Fabry disease: five-year interim results from the ongoing BRIGHT51 study Abstract presented at ICIEM Congress 2025. Elfabrio 2 mg/mL concentrate for solution for infusion - Summary of Product Characteristics (SmPC) - (emc) | 14960. 2025. Available at: https://www.medicines.org.uk/emc/product/14960/smpc%20 Mehta, A., & Hughes, D. A., (2024). Fabry disease. In M. P. Adam, S. Bick, G. M. Mirzaa, et al. (Eds.), GeneReviews®. University of Washington, Seattle. Cleveland Clinic. (2025, October 9). Fabry disease: Symptoms & causes.  UK-CHI-2600071 | April 2026         

Chiesi and Bespak partner to advance Carbon Minimal Inhaler production with UK manufacturing site

Highlights Agreement builds on existing Chiesi-Bespak collaboration, reinforcing a trusted relationship as Chiesi works to evolve a portfolio of extrafine formulation Carbon Minimal Inhalers¹, ensuring patients retain continuity of care while reducing carbon emissions Bespak's leadership in low-GWP propellant inhaler manufacturing along with significant expansion at its Holmes Chapel site strengthens Chiesi's global supply chain resilience Partnership aligns with the science-based climate commitments of both companies  Parma (Italy) and Holmes Chapel (UK) – 11 March 2026 – Chiesi Group ("Chiesi"), the international research focused biopharmaceutical company and a certified B Corp, and Bespak, the specialist inhalation CDMO focused on pulmonary and nasal drug delivery, today announced an expansion of their long-standing partnership, increasing pressurized metered dose inhaler (pMDI) manufacturing capacity at Bespak's Holmes Chapel site to support the next phase of Chiesi's Carbon Minimal Inhaler (CMI) program. Building on years of collaboration, the agreement reflects a shared long-term vision: delivering lower carbon inhaled therapies through CMIs at scale, without compromising clinical choice or continuity of care for patients. Both companies are committed to addressing climate change through measurable, science-based action. Chiesi's ambition to reach Net Zero targets by 2035, and Bespak's validated decarbonization roadmap, underpin a partnership grounded in shared sustainability principles. Maria Paola Chiesi, Chiesi Group Vice Chair, said: "At Chiesi, sustainability is not an add-on; it is a commitment that guides our strategic choices. We know that inhalers are essential treatments, and that the environmental impact associated with them must be addressed without shifting the burden onto patients. The partnership with Bespak reinforces our efforts to reduce emissions across the value chain, while protecting access, quality and trust. Climate action and patient care must continue to advance hand in hand." To meet the needs of patients and reduce impact on the environment, Chiesi is working to be the only company to offer a portfolio of extrafine formulation Carbon Minimal Inhalers, including both dry powder inhalers (DPIs) and next generation propellant pMDIs. Chiesi's CMI program is designed to significantly reduce the carbon footprint of pMDIs by up to 90%, through the transition to a next-generation, low global warming potential (GWP) propellant, while maintaining established treatment options and device familiarity for patients. Reinforcing the partnership with Bespak adds industrial scale and resilience to Chiesi's journey, supporting a phased and responsible transition. The expanded collaboration further strengthens Bespak's position at the forefront of the global industry's transition to next-generation low GWP propellants, and its Holmes Chapel site as a specialist pMDI manufacturing hub within the global pharmaceutical supply chain. Positioned in the North West of England's inhalation R&D and manufacturing cluster, the site contributes high value skills, advanced technical expertise and long-term investment in sustainable inhaler manufacturing, for the benefit of patients and healthcare systems worldwide. Giuseppe Accogli, Chiesi Group CEO, said: "This agreement strengthens an already established partnership with Bespak, and is a concrete example of how we translate our ambition into action. By working with trusted partners across our value chain, we can deliver sustainable innovation at scale while ensuring that patients receive their needed therapies." Chris Hirst, Bespak CEO, said: "Our collaboration with Chiesi has grown over time around a shared commitment to patient safety, technical excellence and sustainability. By deepening this partnership, we are accelerating the transition to low carbon pMDIs and reinforcing the UK's role as a center of excellence for sustainable inhalation manufacturing. This is a position being recognized by the wider industry, leading to our Holmes Chapel site being selected as a key source of supply by leading brand owners like Chiesi, cementing our role as a strategic supply chain partner for the next generation propellant inhalers and innovative nasally-delivered therapies." About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By adopting the legal form of Benefit Corporation in Italy, the US, France and Colombia, Chiesi's commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group's research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For more information, visit chiesi.com or the website of your local Chiesi affiliate. About Bespak Bespak is a specialist inhalation contract development and manufacturing organisation (CDMO) focused on pulmonary and nasal drug delivery. Trusted by the world's leading pharmaceutical companies, Bespak delivers end-to-end capabilities across product development, clinical supply and commercial manufacturing of inhaled therapies for global supply. Headquartered in Holmes Chapel, UK, with specialist manufacturing sites in Holmes Chapel and King's Lynn, UK, Bespak develops and supplies pressurised metered dose inhaler (pMDI) products, valves and actuators, complex dry powder inhaler (DPI) devices, nasal products and devices, and supports emerging inhalation technologies, including innovative soft mist systems. Sustainability underpins every step of how Bespak operates and innovates. The company has taken clear and measurable steps to align with key United Nations (UN) Sustainable Development Goals (SDGs), is a signatory of the United Nations Global Compact (UNGC) and has set approved net-zero and near-term company-wide emissions targets with the Science Based Targets initiative (SBTi). Through collaboration and targeted investment, Bespak is accelerating the industry's transition to more sustainable inhaled medicines. Built on a long history of inhalation experience and ready for the future, Bespak is a long-term innovation partner creating lasting impact for patients and the planet. Press Info Chiesi Group Anna Bonisoli Alquati, Head of Global External Communications Email: mediarelations@chiesi.com Michela Lijoi, Global External Communications Sr. Manager Phone: +39 328.6353044 Email: m.lijoi@chiesi.com Bespak Notch Communications Ltd Email: bespak@notchcommunications.co.uk References In a limited number of countries, including the United Kingdom, non‑extrafine products will also be transitioned to CMI.