Rare Disease Report

BioMarin Discontinuing Their Duchenne Clinical Trials

JUNE 01, 2016
James Radke, PhD
Two years after BioMarin bought Prosensa and its pipeline of exon skipping drugs to treat Duchenne muscular dystrophy for $680 Million, the company has halted all attempts to get those drugs, including drisapersen, to market.

BioMarin has announced that after a recent Committee for Medicinal Products for Human Use (CHMP) meeting to see if drisapersen could get approved in Europe, the company decided to withdraw their application for market approval there. And this comes 4 months after the FDA sent the company a Complete Response Letter stating drisapersen would not get approved in the United States based on the data provided in the New Drug Application (NDA).
In addition, BioMarin stated they will discontinue clinical and regulatory development of drisapersen as well as the 3 other exon skipping drugs that they obtained from the purchase of Prosensa (BMN 044, BMN 045 and BMN 053). All 3 of those drugs were in Phase 2 studies.
In other words, all clinical trials being sponsored by BioMarin for Duchenne muscular dystrophy are being stopped.

Sarepta's Eteplirsen - Last One Standing?

This news comes on the heals of the recent announcement that the FDA is not ready to decide on another exon-skipping drug (eteplirsen) by another biotech company (Sarepta) that had a PDUFA date of May 26th. It is not known when the FDA will make its decision on that drug.

What is Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.

There are many subsets of the DMD population based on the type of mutation found in the dystrophin gene. There are currently no drugs approved in the US to directly treat any of these groups. Eteplirsen and drisapersen were designed to treat boys with Duchenne muscular dystrophy amenable to exon 51 skipping therapy.  Sarepta (and until recently BioMarin) also has other exon skipping drugs in development to treat other dystrophin gene mutations.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.