Chiesi Global Rare Diseases Announces Publication of Long-Term Assessment of ELFABRIO^® (pegunigalsidase alfa-iwxj) in Fabry Disease

- Data published in Genetics in Medicine demonstrate continuous benefits up to six years of treatment -

BOSTON, March 7, 2024 - Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, today announced the publication of long-term data up to six years evaluating the safety and efficacy of treatment with ELFABRIO (pegunigalsidase alfa-iwxj) in adult patients with Fabry disease. The data are published in Genetics in Medicine, an official journal of the American College of Medical Genetics and Genomics.

ELFABRIO is a PEGylated α-Gal A enzyme replacement therapy (ERT) that is approved for the treatment of adults with Fabry disease in the United States, European Union and Great Britian. In a Phase 1/2 dose-ranging study, 15 ERT-naïve adults with Fabry disease completed 12 months of treatment with ELFABRIO and enrolled in a 60-month, open-label, extension study.

Both males and females achieved a mean reduction in plasma globotriaosylceramide (lyso-Gb3) concentrations at 60 months of >70% from baseline (91% reduction for males and 72% reduction for females). Kidney function was stable with an overall mean (SE) annualized estimated glomerular filtration rate (eGFR) slope of -1.6 (0.8) mL/min/1.73 m²/y. Most treatment-emergent adverse events and all infusion-related reactions were mild to moderate. Of the five male patients who were anti-pegunigalsidase alfa IgG positive after baseline, four were transiently positive and one had sustained positivity.

“We launched Chiesi Global Rare Diseases to help society tackle the global issue of rare diseases, and we are focused on the long-term support of Fabry disease patients and their community,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases. “Open-label extension studies like this are important because we have the opportunity to remain close to the patients, caregivers and their providers, always learning from them and responding to their needs.”

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.

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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