First and Only Enzyme Replacement Therapy for Alpha-Mannosidosis
Chiesi Global Rare Diseases announced the FDA approval of Lamzede® (velmanase alfa-tycv) for treating 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 a deficiency in the enzyme α-mannosidase.
Understanding Alpha-Mannosidosis
Alpha-mannosidosis affects approximately one in every 500,000 to one in every 1,000,000 babies born worldwide. The disorder prevents the body's cells from properly breaking down certain groups of complex sugars, leading to their accumulation in various tissues and organs. This can result in a wide range of symptoms, including impaired hearing, speech, and mobility, as well as recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, and cognitive abnormalities.
The Role of Lamzede
Lamzede is a recombinant form of human alpha-mannosidase designed to provide or supplement the natural enzyme, aiding in the degradation of mannose-rich oligosaccharides to prevent their harmful accumulation. The therapy has been available in Europe since 2018 for the treatment of non-neurological manifestations in patients with mild to moderate AM.
Important Safety Information
- Hypersensitivity Reactions: Patients treated with Lamzede have experienced hypersensitivity reactions, including anaphylaxis. It is crucial to have appropriate medical support measures readily available during administration.
- Infusion-Associated Reactions (IARs): Symptoms such as pyrexia, chills, erythema, vomiting, cough, urticaria, rash, and conjunctivitis have been reported.
- Females of Reproductive Potential: Effective contraception is advised during treatment and for 14 days after the last dose if Lamzede is discontinued.
- Embryo-Fetal Toxicity: Based on animal studies, Lamzede may cause harm to an embryo or fetus.
- Common Adverse Reactions: The most common adverse reactions include hypersensitivity reactions, nasopharyngitis, pyrexia, headache, and arthralgia.
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases, a business unit of the Chiesi Group, is dedicated to delivering innovative therapies for people affected by rare diseases. The unit collaborates globally with the rare disease community to ensure equal access to treatments, aiming to improve the quality of life for as many people as possible.
About Chiesi Group
Chiesi Group is an international, research-focused biopharmaceutical company committed to improving people's quality of life and acting responsibly towards the community and the environment. With over 85 years of experience, Chiesi operates in 30 countries and employs more than 6,000 people, striving to become net-zero by 2035.