First Generic Version of Miglustat Approved by FDA

Final approval was given this morning to an Abbreviated New Drug Application (ANDA) for Miglustat 100 mg capsules for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not an option.

Amerigen Pharmaceuticals Limited and Dipharma S.A. submitted the application in January 2016; this is the first ANDA to be approved as a generic equivalent to Actelion Pharmaceuticals’ version of the drug, Zavesca.

Filing the ANDA came as the result of an exclusive collaboration between the 2 companies in developing and commercializing Miglustat 100 mg capsules worldwide. Miglustat active ingredient is supplied to Amerigen by Dipharma who holds 2 granted U.S. patents — US9079856B2 and US8802155B1 – the former for a method of synthesis for miglustat and the latter for a crystalline form of the same.

"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, Chief Executive Officer of Dipharma in a press release. "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."

Amerigen has the right to enforce these patents in the U.S. whilst Amerigen's affiliates will manufacture the finished product and commercialize it in the U.S., where it has already been launched.

Miglustat, when marketed as Zavesca, was originally approved by the FDA in July 2003. It is a synthetic analogue of D-glucose, known as an iminosugar, and operates as a substitute for the glucocerebrosidase enzyme, whose primary function is to convert glucocerebroside, or glucosylceramide, into ceramide and glucose. Individuals with Gaucher disease have a defect in the glucocerebrosidase enzyme, leading to the build up of glucocerebroside, which can cause liver and spleen enlargement, changes in the bone marrow and blood, and 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,” said John Lowry, Amerigen's President and CEO. “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."

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