Tuesday, March 28 @ 1pm EDT Behavioral Science in Clinical Trials


Lamzede Gets Approved for Rare Disease Alpha-Mannosidosis

Lamzede Gets Approved for Rare Disease Alpha-Mannosidosis

Lamzede is FDA approved to treat alpha-mannosidosis, an extremely rare genetic metabolic disease affecting approximately one in 500,000 people.

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients. Given this approval, Lamzede is the first and only enzyme replacement therapy indicated for AM.

Alpha-mannosidosis is an extremely rare genetic metabolic disease affecting approximately one in 500,000 people. The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues). A lack of this enzyme results in a toxic build-up of mannose-containing sugars in the cells of the body, including the central nervous system (CNS).

The disease usually clearly presents between birth and childhood, though it is often not recognized and patients can be undiagnosed and untreated for long periods. Additionally, the effects of the disease can vary substantially from person to person, and how it presents can change over the patient’s lifespan, too. Some of the most common symptoms include a characteristic facial structure (large head, low hairline, prominent forehead), excessive gum growth, enlarged tongue, skeletal abnormalities, muscle weakness and immune deficiencies.

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Lamzede Efficacy and Functionality

Lamzede is a recombinant form of human alpha-mannosidase. Its use is intended to supplement the naturally occurring levels of the enzyme to re-establish normal cellular function. In doing so, the enzyme can degrade the mannose-containing oligosaccharides and prevent their accumulation throughout the body. Typically, the medication is administered as a 10 mg injection once a week.

The efficacy of Lamzede was evaluated in a Phase III multicenter, randomized, double-blind, placebo-controlled, parallel-group study including adult and pediatric patients with alpha-mannosidosis. The study evaluated efficacy of 1 mg/kg Lamzede given as a once-weekly intravenous infusion over 52 weeks. Twenty-five patients were enrolled (14 male and 11 female), including 13 adults (aged ≥ 18 years) and 12 pediatric patients (ages ≥ 6 and < 18 years).

Fifteen patients (eight adults, seven children) received Lamzede, while 10 patients (five of each age group) received placebo. Results were assessed at 12 months with a three-minute stair climbing test, a six-minute walking test and measuring forced vital capacity. All the results were in favor of the treatment group and were additionally supported by reduced oligosaccharides in the serum from baseline.

Common adverse reactions to treatment with Lamzede include hypersensitivity reactions like anaphylaxis, nasopharyngitis, headaches, increased body temperature and joint stiffness. Female patients must also be advised to use contraception during treatment and for 14 days after their last dose if treatment is discontinued. They also cannot be pregnant at the onset of treatment, as Lamzede can harm the embryo or fetus.

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Accessing Alpha-Mannosidosis Treatment

The Chiesi Group previously received marketing authorization from the European Commission for Lamzede in 2018. The authorization was for treating non-neurological manifestations of alpha-mannosidosis in patients with mild to moderate alpha-mannosidosis. Additionally, with its new FDA approval, Lamzede has received orphan drug designation for the described indication.

In terms of pricing, Chiesi Group has not disclosed costs of Lamzede yet. However, once available as a treatment, physicians and patients can access the support program (Chiesi Total Care) found on the company website. Their goal is to make the medication accessible to eligible patients.