Chiesi Global Rare Diseases Announces FDA Approval of Lamzede®(velmanase alfa-tycv) for Alpha-Mannosidosis

17 Feb 2023
pipelinereview.com
Drug Approval
- 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, MA, USA I February 16, 2023 I 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 α-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 α-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."
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.
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
SOURCE: Chiesi Global Rare Diseases
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Targets
Drugs
Chat with Hiro
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.