FDA Accepts BLA for Ultra Rare Enzyme Disorder

The FDA has accepted the biologics license application (BLA) and granted priority review designation for velmanase alfa, enzyme replacement therapy for alpha-mannosidosis, a rare genetic condition characterized by an inability to properly break down complex sugars in the body’s cells. It is caused by mutations of the MAN2B1 gene.

Developed by Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici, velmanase alfa is a recombinant form of human alpha-mannosidase intended to supplement the natural enzyme. The buildup of sugars can affect many organs and systems including the central nervous system. Effects of the disease vary and progress over time, and symptoms can include recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, or cognitive abnormalities. The prevalence of alpha-mannosidosis is estimated to be 1 in every 500,000 people.

The FDA set an action date in the first half of 2023 under the Prescription Drug User Fee Act.

Giacomo Chiesi

“Alpha-mannosidosis is an under recognized and progressive disease, and there may be many patients who are undiagnosed and untreated. The current management of alpha-mannosidosis is largely focused on treatment of the various symptoms,” Giacomo Chiesi, head of Chiesi Global Rare Diseases, said in a press release.

In 2018, Chiesi Group received marketing authorization from the European Commission for the therapy under the brand name Lamzede for the non-neurological manifestations in patients with mild to moderate alpha‑mannosidosis.