Chiesi Global Rare Diseases Announces Presentations at SSIEM Annual Symposium 2024

- Nine posters accepted for presentation with focus on advances in the treatment of Fabry disease and alpha-mannosidosis -

- Company also sponsoring two industry symposia featuring perspectives from global clinical and patient advocacy experts in lysosomal storage disorders -

- Sustainability report highlighted at company booth -

PARMA, SEPTEMBER 3, 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 nine poster presentations and two sponsored symposia at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024 being held September 3-6 in Porto, Portugal.

“At SSIEM 2024 we have an opportunity to collaborate with all stakeholders for the benefit of people living with rare diseases and look forward to sharing updates from our research into conditions that are often misunderstood including Fabry disease and alpha-mannosidosis (AM), reflecting our commitment to the patient voice,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “We are also proud to use our presence at SSIEM 2024 to highlight the results of our recent sustainability report and our commitment to creating positive impact on patients, people, the planet and the communities we serve.”

The poster presentations include five abstracts that will focus on advances in the understanding and management of Fabry disease as well as integrated clinical data on pegunigalsidase alfa treatment in patients with Fabry disease.

• A poster, titled, “Co-Creation of a Shared Decision-Making Tool for Patients with Fabry Disease (FD) and their Treating Clinicians,” is being presented by Derralynn Hughes, M.D., Professor of Experimental Haematology at the University College London.

• Another poster, titled, “Rapid cellular uptake and high stability of recombinant pegunigalsidase alfa (PA) in fibroblasts from Fabry patients,” is being presented by Abdullah Hoter, Ph.D., postdoc in Biochemistry and Molecular Biology at Cairo University.

• Prof. Hughes is also presenting a poster, titled, “Assessment of Immunogenicity from the Pegunigalsidase Alfa Clinical Trial Program: Integrated Analysis of De Novo and Treatment-Boosted Anti-Drug Antibodies.”

• David Warnock, M.D., The University of Alabama at Birmingham, is presenting a poster, titled, “Impact of Baseline Proteinuria on Renal Outcomes in the BALANCE Study of Pegunigalsidase Alfa vs Agalsidase Beta in Fabry Disease.”

• Ozlem Goker-Alpan, M.D., founder and Chief Executive Officer of Lysosomal and Rare Disorders Research and Treatment Center, is presenting a poster, titled, “Lower Rate of Infusion-Related Reactions in Patients with Fabry Disease After Switching from Agalsidase Beta to Pegunigalsidase Alfa.”

An additional four posters are being presented with a focus on advances in the understanding and management of AM and long-term efficacy and safety data on velmanase alfa treatment in patients with AM.

• A poster, titled, “Evaluating the relationship between antidrug antibodies and infusion-related reactions in patients with alpha-mannosidosis treated with velmanase alfa,” is being presented by Nathalie Guffon, M.D., Reference Centre for Inherited Metabolic Diseases (CERLYMM), Hospices Civils of Lyon, Lyon, France.

• Another poster, titled, “Quality of life and burden of caregivers of patients with alpha-mannosidosis: Results from an international survey,” is being presented by Julia Hennermann, M.D., Ph.D., Villa Metabolica, University Medical Center Mainz, Mainz, Germany.

• Dr. Guffon is also presenting a poster, titled, “Lamzede LAMAN-07/09 Efficacy: Long term efficacy of velmanase alfa treatment in patients with Alpha mannosidosis: Pooled data up to 12 years from two extension studies.”

• Karolina Stepien, M.D., Adult Inherited Metabolic Disease, Northern Care Alliance National Health Service (NHS) Foundation Trust, Salford Royal Organization, Salford, UK, is presenting a poster, titled, “Alpha-mannosidosis international caregiver and patient survey: changes in mobility, pain or discomfort, and patients’ self-care over time.”

Chiesi Global Rare Diseases is also sponsoring two symposia during SSIEM 2024. On Wednesday, September 4, from 7:30-8:30am WEST, the symposium, “How to deal with uncertainty in LSDs to improve patient care: the alpha-mannosidosis case,” features Dr. Stepien and Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany, and is being moderated by co-chairs Maria Carmo Macario, M.D., Centro Hospitalar e Universitario de Coimbra, Portugal, and Christina Lampe, M.D., Director of the Centre for Rare Diseases, University Hospital of Giessen, Germany. The faculty will review real clinical cases and dive into the pathogenesis, clinical presentation and management of AM, with a specific focus on translating the latest data from pharmacological studies into daily practice.

On Thursday, September 5, from 12:30-1:30pm WEST, the symposium, “Empowering voices in Fabry disease: collaborative advances in treatment and patient care,” features Prof. Hughes, Ales Linhart, M.D., Charles University, Praha, Czech Republic, Lisa Berry, LGC, Division of Human Genetics at Cincinnati Children's Hospital Medical Center, and Lorella Terzi, who is living with Fabry disease, and is being led by Olga Azevedo, M.D., Director of the Reference Centre on Lysosomal Storage Disorders (LSD) in Hospital Senhora da Oliveira, Guimarães, Portugal. Participants will delve into the latest clinical trial data, alongside integrating the patient voice with clinical perspectives.

Chiesi Global Rare Diseases is also the sponsor of booth PP01, where the company will focus on its research initiatives and commitment to sustainability. The company announced the launch of a new research grant initiative, Find for Rare, with an invitation to scientists from outside the Americas to submit for grants of up to €50,000 to support research into rare lysosomal storage disorders. The company is also highlighting findings from a published sustainability report. Head of Shared Value and Sustainability, Cecilia Plicco, is hosting a series of open sessions at the company’s booth, titled, “Building a Sustainable Future Requires a Collective Effort,” to discuss the full integration of sustainability and shared value into the Chiesi strategy.

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
Rare Communication Manager
Tel: +39 348 8818985
Email: c.travagin@chiesi.com

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

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