FDA Grants Priority Review to Chiesi’s BLA for Velmanase Alfa

The FDA has given Priority Review to Chiesi Farmaceutici business unit Chiesi Global Rare Diseases’ Biologics License Application (BLA) for the biologic velmanase alfa.

The biologic is an investigational enzyme replacement therapy to treat patients with the rare genetic disorder alpha-mannosidosis, a condition characterized by an enzyme deficiency that renders the body’s cells unable to properly break down certain groups of complex sugars.

Velmanase alfa is described by the company as a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, which helps with the degradation of mannose–rich oligosaccharides to prevent their accumulation in various tissues in the body.

Priority Review is a designation that shortens the FDA review period and is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. The agency has set an action date in the first half of 2023.