Chiesi Global Rare Diseases Announces Multiple Presentations at 21st Annual WORLDSymposium™ Research Meeting

– Presenting 11 abstracts with range of insights on diagnosis and management of Fabry disease and alpha-mannosidosis and clinical data on pegunigalsidase alfa and velmanase alfa – 

– Results include 10-year data from study of velmanase alfa – 

– Organizing and funding two satellite symposia showcasing insights from panel of global clinical experts in lysosomal diseases, focusing on how to optimize treatment strategies –

PARMA, FEBRUARY 3, 2025 – 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 11 poster presentations have been accepted at the 21st Annual WORLDSymposium™ Research Meeting to be held February 3-7, 2025, in San Diego, California. The presentations include long-term data up to 10 years of treatment with velmanase alfa in patients with alpha-mannosidosis. Additionally, the presentations cover advances in the understanding of patient tolerability while on treatment and strategies for incorporating both patient and caregiver perspectives in disease management.

“We are pleased to have the opportunity to present a wide range of clinical data and insights related to the diagnosis, treatment and appropriate monitoring of people living with Fabry disease and alpha-mannosidosis at the WORLDSymposium,” said Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases. “As part of our mission to improve patient outcomes for the lysosomal disease community, we are committed to continual learning, listening and partnering with patients, caregivers and families to improve disease management strategies and evaluating long-term clinical data to better understand the impact of treatment.”

Poster Presentations

Pegunigalsidase alfa and adults with Fabry disease

• Title: Lower rate of infusion-related reactions in patients with Fabry disease after switching from agalsidase beta to pegunigalsidase alfa
  Presenter: Nicola Longo, M.D., Chief of the Division of Medical Genetics at the University of Utah
• Title: Improved tolerability following enzyme replacement therapy switch to pegunigalsidase alfa: A case series from two centers of the expanded access program
  Presenter: Myrl D. Holida, Physician Assistant at the University of Iowa Health Care
• Title: Evaluating the relationship between infusion-related reactions and antidrug antibody status: Results from 111 patients with Fabry disease treated with pegunigalsidase alfa
  Presenter: Robert Hopkin, M.D., Clinical Geneticist at Cincinnati Children's Hospital Medical Center
• Title: Clinical assessment of disease severity in patients with Fabry disease treated with pegunigalsidase alfa: An integrated analysis
  Presenter: Derralynn Hughes, M.D., Professor of Experimental Haematology at the University College London
• Title: Indirect treatment comparisons of pegunigalsidase alfa vs other therapies for left ventricular mass index in Fabry disease
  Presenter: Eric Wallace, M.D., Co-Director of the University of Alabama at Birmingham Fabry Disease Clinic
• Title: Awareness of Fabry disease among non-Fabry specialists: Opportunities for education
  Presenter: Thomas Kenny, Patient Advocacy Lead, Chiesi Limited
• Title: Maximising engagement through feedback: Insights from shared decision-making toolkit for Fabry disease patients
  Presenter: Thomas Kenny, Patient Advocacy Lead, Chiesi Limited
• Title: Speaking the same language: The Fabry lexicon and the implications for how the healthcare community understands the impact of ERT
  Presenter: Thomas Kenny, Patient Advocacy Lead, Chiesi Limited

Velmanase alfa and alpha-mannosidosis

• Title: Clinical profiles of 134 patients with alpha-mannosidosis from the velmanase alfa clinical program and SPARKLE registry
  Presenter: Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany
• Title: Long-term motor function and quality of life outcomes in patients with alpha-mannosidosis: Data from two velmanase alfa extension studies over 10 years
  Presenter: Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France
• Title: Real world reflections of the patient odyssey in alpha-mannosidosis: Insights and challenges in diagnosis from caregiver interviews
  Presenter: Sonia Sgrò, Global Head Patient Advocacy, Lysosomal Storage Disorders (LSDs) at Chiesi Global Rare Diseases

Chiesi Global Rare Diseases is also organizing and funding two satellite symposia during the WORLDSymposium:

• Title: Enzyme Replacement Therapy: Time to Act in Lysosomal Diseases
  Date and Time: Tuesday, February 4, 2025 from 5:45-6:45 p.m. PT
  A panel of global clinical experts will discuss current knowledge of lysosomal diseases and appropriate management strategies for patients on enzyme replacement therapy including the potential for switching therapies given the availability of multiple treatment options.
• Title: Red Light, Green Light: What About the Yellow Light? Exploring the Complexities of Treatment Tolerability in Fabry Disease
  Date and Time: Wednesday, February 5, 2025 from 12:15-1:15 p.m. PT
  Presenters will explore the mechanisms and patient-specific factors that influence the tolerability of enzyme replacement therapies in Fabry disease, discuss pegunigalsidase alfa tolerability data, and highlight potential future directions towards enhancing patient outcomes. 

Indication and Important Safety Information for Elfabrio^® (pegunigalsidase alfa-iwxj) 

Indication  

Elfabrio^® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease. 

Important Safety Information 

Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs), and instruct them to seek medical care immediately if such symptoms occur.

• If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment.
• If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose.

In clinical trials, 20 (14%) Elfabrio-treated patients experienced hypersensitivity reactions. Four Elfabrio-treated patients (3%) experienced anaphylaxis reactions that occurred within 5 to 40 minutes of the start of the initial infusion. The signs and symptoms of hypersensitivity reactions and anaphylaxis included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema. 

In clinical trials, 41 (29%) Elfabrio-treated patients experienced one or more infusion-associated reactions, including hypersensitivity, nausea, chills, pruritus, rash, chest pain, dizziness, vomiting, asthenia, pain, sneezing, dyspnea, nasal congestion, throat irritation, abdominal pain, erythema, diarrhea, burning sensation, neuralgia, headache, paresthesia, tremor, agitation, increased body temperature, flushing, bradycardia, myalgia, hypertension, and hypotension.

A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted. 

When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels). 

The most common adverse reactions (≥15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.

Please see Full Prescribing Information for Elfabrio.

Indication and Important Safety Information for Lamzede^® (velmanase alfa-tycv) 

Indication

Lamzede^® (velmanase alfa-tycv) is indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients.

Important Safety Information 

Considerations Due to Hypersensitivity Reactions and/or Infusion-Associated Reactions (IARs) 

Prior to Lamzede administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and IARs and instruct them to seek medical care immediately if such symptoms occur.

• If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Lamzede administration and initiate appropriate medical treatment.
• In the event of a mild to moderate hypersensitivity reaction or a mild to moderate IAR, consider temporarily holding the infusion for 15 to 30 minutes, slowing the infusion rate to 25% to 50% of the recommended rate, and initiating appropriate medical treatment. 

Hypersensitivity Reactions Including Anaphylaxis 

Anaphylaxis and severe hypersensitivity signs and symptoms included cyanosis, hypotension, emesis, urticaria, erythema, facial swelling, pyrexia, and tremor. 

Infusion-Associated Reactions (IARs) 

The most frequent symptoms of IARs that occurred in >10% of the population were pyrexia, chills, erythema, vomiting, cough, urticaria, rash, and conjunctivitis.  

Females of Reproductive Potential  

Advise females of reproductive potential to use effective contraception during treatment and for 14 days after the last dose if Lamzede is discontinued. For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with Lamzede. 

Embryo-Fetal Toxicity 

Based on findings from animal reproduction studies, Lamzede may cause embryo-fetal harm when administered to a pregnant female. 

Common Adverse Reactions 

The most common adverse reactions (incidence >20%) are hypersensitivity reactions including anaphylaxis, nasopharyngitis, pyrexia, headache, and arthralgia. 

Please see Full Prescribing Information for Lamzede.

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 affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a 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.

For more information visit www.chiesirarediseases.com.

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.com.

Chiesi Group Media Contacts

Chiara Travagin
Head of Communications, Rare Diseases
Tel: +39 348 8818985
Email: c.travagin@chiesi.com

Jenna Urban
CG Life
Tel: +1 212 253 8881
Email: jurban@cglife.com

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