FDA Approves Miglustat, 1st Generic Equivalent of Zavesca, to Treat Gaucher Patients

FDA Approves Miglustat, 1st Generic Equivalent of Zavesca, to Treat Gaucher Patients

A generic version of Zavesca (miglustat) — a substrate reduction therapy (SRT) approved to treat Gaucher disease — has been approved for similar use by the U.S. Food and Drug Administration (FDA), Amerigen Pharmaceuticals and Dipharma announced.

This is the first generic version of Zavesca, marketed by Actelion Pharmaceuticals, to be given approval. Miglustat, the generic, will be available in 100 mg oral capsules, a formulation equivalent to Zavesca.

Miglustat, like Zavesca, is indicated to treat adults with mild to moderate type 1 Gaucher disease who cannot use enzyme replacement therapy. Zavesca is also approved for patients with Niemann-Pick disease type C, a rare genetic disease marked by the buildup of cholesterol and lipids within tissues.

The treatment is an inhibitor of glucosylceramide synthase, an enzyme involved in the synthesis of fat molecules called glycosphingolipids. People with Gaucher do not produce enough of an enzyme, glucocerebrosidase, that breaks works to break down glycosphingolipids — in particular glucocerebroside — allowing it to accumulate.

By blocking glucosylceramide synthase, the medicine works against the accumulation of glucocerebroside inside the cells of Gaucher patients.

“Thanks to this approval, chronically ill U.S. patients and payers will have available a high quality, more affordable alternative to current treatment,” Marc-Olivier Geinoz, chief executive officer of Dipharma, said in a press release“This marks the first approval of a series of products our group has been developing in collaboration with Amerigen for various markets.”

The generic version  was developed through an Amerigen and Dipharma partnership. Dipharma, which specializes in rare diseases, holds two patents related to the chemical synthesis of miglustat and is supplying it as the active ingredient to Amerigen, which specializes in generic medications.

Amerigen affiliates are in charge of the generic’s manufacture and commercialization in the U.S.   

“We are delighted to launch this product following a fruitful collaboration with Dipharma. This is Amerigen’s fifth U.S. product launch and the third time we have brought a first generic to market,” said John Lowry, Amerigen’s president and chief executive officer.