FDA OKs First Generic Version of Miglustat

The US Food and Drug Administration (FDA) has approved an Abbreviated New Drug Application (ANDA) for Miglustat 100 mg capsules, indicated for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not an option.

A result of cooperation between Amerigen Pharmaceuticals and Dipharma, this is the first ANDA to be approved as a generic equivalent to Actelion Pharmaceuticals’ version of the drug, Zavesca.

"This marks the first approval of a series of products our group has been developing in collaboration with Amerigen for various markets,” said Marc-Olivier Geinoz, MBA, the chief executive officer of Dipharma, in a statement. "Thanks to this approval, chronically ill U.S. patients and payers will have available a high quality, more affordable alternative to current treatment. For our young company, it is a great achievement and it marks a significant milestone in our growth strategy."

The January 2016 filing of the ANDA came as the result of an exclusive collaboration between the 2 companies in developing and commercializing Miglustat 100 mg capsules, globally. Amerigen was supplied Miglustat’s active ingredient by Dipharma, which holds 2 US patents—US9079856B2 and US8802155B1—the former for a method of synthesis for miglustat and the latter for a crystalline form of the same.

Amerigen has the right to enforce Dipharma’s patents, while affiliates of Amerigen's manufacture and commercialize the finished product in the US, where it has already been launched.

Miglustat, a synthetic analog of D-glucose (an iminosugar), operates as a substitute for the glucocerebrosidase enzyme, which has a primary function of converting glucocerebroside or glucosylceramide, into ceramide and glucose, was originally approved by the FDA in July 2003. Individuals with Gaucher disease have a defect in the glucocerebrosidase enzyme, which can result in liver and spleen enlargement, changes in bone marrow and blood, as well as bone disease.

Unlike available enzyme replacement therapies (ERTs), Miglustat works via substrate reduction therapy, preventing the formation of a substance that accumulates when an enzyme doesn’t operate properly.

"We are delighted to launch this product following a fruitful collaboration with Dipharma,” John Lowry, Amerigen's president and CEO, said in a statement. “This is Amerigen's fifth U.S. product launch and the third time we have brought a first generic to market, with important savings for the American healthcare system."