Chiesi Global Rare Diseases to Present at the 20^th Annual WORLDSymposium™ Research Meeting

- Presenting nine abstracts with insights on the diagnosis and management of Fabry disease and alpha-mannosidosis and clinical data on pegunigalsidase alfa and velmanase alfa -

- Sponsoring three satellite symposia featuring expert panels and patient and caregiver perspectives -

- Presenting “Rare Land” film screening during Industry Expert Theater session -

- Participating in “The Patient Voice: Is Anyone Listening?” program with focus on mental health in Fabry disease -

BOSTON, February 4, 2024 - 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 nine abstracts have been accepted for presentation at the 20^th Annual WORLDSymposium™ Research Meeting to be held February 4-9, 2024, in San Diego, California. The company will also sponsor three satellite symposia, present a special film screening, and participate in the Patient Voice and Preference meeting.

Two oral presentations will provide insights on the diagnosis and management of Fabry disease and long-term clinical data on velmanase alfa treatment in patients with alpha-mannosidosis.

• On Wednesday, February 7 at 9:00 AM PST, Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France, will present an abstract titled, “Long-term efficacy of velmanase alfa treatment in patients with Alpha-mannosidosis: Pooled data from two extension studies (up to 12 years of therapy).”

• On Thursday, February 8 at 10:30 AM PST, Stuart Gaffney, M.D., Medical Manager, Chiesi Global Rare Diseases, will present an abstract titled, “Medical education needs to improve diagnosis of Fabry disease in the UK.”

The company is announcing the presentation of seven additional posters during WORLDSymposium:

• “Assessment of immunogenicity from the pegunigalsidase alfa clinical trial program: integrated analysis of de novo and treatment-boosted anti-drug antibodies” - John Bernat, M.D., Ph.D., Medical Director of the Iowa Lysosomal Storage Disorders Center

• “Tolerability of pegunigalsidase alfa across the clinical program: integrated analysis of infusion-related reactions by dosing regimens” - Derralynn Hughes, M.D., Professor of Experimental Haematology at the University College London

• “Tolerability of pegunigalsidase alfa across the clinical program: integrated analysis of infusion-related reactions by prior enzyme replacement therapy” - Ankit Mehta, M.D., FASN, Baylor University Medical Center 

• “Estimating Fabry Disease Prevalence and Treatment Rates in the United States: An Epidemiological Analysis” - Khashayar Azimpour, M.D., Ph.D., Director, Global Health Economics and Outcome Research (GHEOR), Chiesi USA

• “The Burden of Fabry disease in the United States: Results from the Adelphi Fabry Disease Specific Programme” - Patricia Dorling, PharmD, MS, Ph.D., Head of HEOR & RWE, Rare Diseases, Chiesi USA

• “A Delphi consensus approach to monitoring and integrated care coordination of patients with alpha-mannosidosis” - Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany

• “Alpha-mannosidosis international caregiver and patient survey: changes in mobility, pain or discomfort, and patients’ self-care over time” - Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France

The company will also sponsor three satellite symposia during WORLDSymposium:

• On Wednesday, February 7 beginning at 5:15 PM PST, a panel of experts including Uma Ramaswami, M.D., Royal Free Hospital in London (UK), and Ales Linhart, M.D., Charles University, Praha, Czech Republic, will participate in a symposium titled, “Perspectives on the Management of Fabry Disease.”

• On Thursday, February 8 beginning at 6:15 AM PST, Parul Jayakar, M.D., Division of Clinical Genetics at Nicklaus Children's Hospital, Miami (U.S.) and a caregiver will participate in a symposium titled, “Patient Spotlight: Alpha-mannosidosis.”

• On Thursday, February 8 beginning at 5:15 PM PST, Lisa Berry, LGC, Division of Human Genetics at Cincinnati Children's Hospital Medical Center, and Julia Hennermann, M.D., Ph.D., Center for Pediatric and Adolescent Medicine Villa Metabolica, University Medical Center Mainz, Germany, along with patient and caregiver speakers Sabina Kineen and Sophie Thomas, will participate in a symposium titled, “Unlocking the power of the patient voice: Holistic care for patients with Fabry disease and alpha-mannosidosis.”

In addition, the company will present a special screening of “Rare Land,” a film focused on alpha-mannosidosis, during the Industry Expert Theater session on Tuesday, February 6 beginning at 3:00 PM PST. An expert panel featuring Robert Hopkin, M.D., Clinical Geneticist at Cincinnati Children's Hospital Medical Center and Martin Magner, M.D., Ph.D., Department of Paediatrics and Hereditary Metabolic Disorders of the General University Hospital of Prague, will be held following the screening.

Finally, Andrés Treviño, Associate Director of North America Patient Advocacy, Rare Diseases, Chiesi USA, will participate in a new program, “The Patient Voice: Is Anyone Listening?” The presentation, titled, “Embracing the silence: Taking listening to a new level. Mindfully rare mental health in the Fabry community,” will begin at 5:05 PM PST on Sunday, February 4.

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 an international, research-focused biopharmaceuticals 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), operates in 31 countries, and counts more than 6,500 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
Rare Communication Manager
Tel: +39 348 8818985
Email: c.travagin@chiesi.com

Alessio Pappagallo
Press Office Manager
Tel: +39 339 5897483
Email: a.pappagallo@chiesi.com  

Adam Daley
Berry & Company Public Relations
Tel: +1 212 253 8881
Email: adaley@berrypr.com

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