PARMA, Italy, Feb. 3, 2025
- 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 �
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- Results include 10-year data from study of velmanase alfa �
-�Organizing and funding two satellite symposia showcasing insights from panel ofglobal clinical experts in lysosomal diseases, focusing on how to optimize treatment strategies �
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PARMA, Italy, Feb. 3, 2025 /PRNewswire/ -- 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
Velmanase alfa and alpha-mannosidosis
Chiesi Global Rare Diseases is also organizing and funding two satellite symposia during the WORLDSymposium:
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�
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXISPatients treated with Elfabrio have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Elfabrio administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue Elfabrio immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to Elfabrio may be considered.��
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.�
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�
WARNING: SEVERE HYPERSENSITIVITY REACTIONSHypersensitivity Reactions Including AnaphylaxisPatients treated with Lamzede have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Lamzede administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue Lamzede immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to Lamzede may be considered.��
�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.
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 DiseasesChiesi 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 GroupChiesi 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 TravaginHead of Communications, Rare DiseasesTel: +39 348 8818985Email: c.travagin@chiesi.com
Jenna UrbanCG LifeTel: +1 212 253 8881Email:�jurban@cglife.com PP-RA-00518 V1.0
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