Subject: FDA
Chiesi Global Rare Diseases Announces FDA Approval of Lamzede®(velmanase alfa-tycv) for Alpha-Mannosidosis
- First and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients -
- Ultra-rare progressive disease presents with wide range of symptoms -
BOSTON, Feb. 16, 2023 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today that the U.S. Food and Drug Administration (FDA) has approved Lamzede® (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme ?-mannosidase.
"Today's approval of Lamzede represents a major milestone for people living with alpha-mannosidosis. Lamzede is the first and only enzyme replacement therapy approved for alpha-mannosidosis in the United States, an achievement based on years of clinical development, as well as the dedication of our employees, clinicians, patients and their families," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "Alpha-mannosidosis presents with a variety of symptoms including impaired hearing, speech and mobility that progress from childhood into adulthood and is often under-recognized, causing some patients to be left undiagnosed or untreated. Lamzede is designed to provide an exogenous source of the ?-mannosidase enzyme and we look forward to offering this medicine to patients in the United States who are eagerly awaiting a treatment option."
The prevalence of AM is approximately one in every 500,000 to one in every 1,000,000 babies born worldwide. AM results in the body's cells being unable to properly break down certain groups of complex sugars. The buildup of sugars can affect many of the body's organs and systems. Effects of the disease vary significantly from person to person and progress over time. Symptoms may change as a patient gets older and can include recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, and cognitive abnormalities.
Lamzede is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, an enzyme that is involved in the degradation of mannose?rich oligosaccharides to prevent their accumulation in various tissues in the body. In 2018, Chiesi Group received marketing authorization from the European Commission for Lamzede for the treatment of non-neurological manifestations in patients with mild to moderate AM.
"We are thrilled to witness this milestone for the alpha-mannosidosis community," said Mark Stark, treasurer at the International Society for Mannosidosis & Related Disorders (ISMRD) and father of a son living with AM. "I have watched firsthand how alpha-mannosidosis can progress and impact daily life with my son and this approval gives hope to patients, caregivers and families impacted by this devastating disease. ISMRD is excited to continue to collaborate with Chiesi and we are thankful for their efforts to develop Lamzede to give patients in the U.S. a much-needed treatment option."
Important Safety Information
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 REACTIONS
Hypersensitivity Reactions Including Anaphylaxis
Patients 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.
- 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.
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 at becoming net-zero by 2035.
With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 30 countries, and counts more than 6,000 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
Media Contact
Jenna Urban
Berry & Company Public Relations
1-212-253-8881
[email protected]
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SOURCE Chiesi Global Rare Diseases
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