Biomarin withdraws regulatory submissions for Duchenne muscular dystrophy drug

pharmafile | June 1, 2016 | News story | Research and Development, Sales and Marketing BioMarin, Duchenne Muscular Dystrophy, EMA, FDA, Kyndrisa, failure, rejection 

Biomarin (NASDAQ: BMRN) has announced that it has withdrawn its marketing authorisation application for Duchenne muscular dystrophy drug, Kyndrisa (drisapersen), from the European Medicines Agency (EMA).

The drug was rejected by the US Food and Drug Administration (FDA) via a complete response letter sent to the company in January. Their decision not to pursue the marketing authorisation application with the EMA comes following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting where it was made clear that a negative opinion on the drug would be given.

Biomarin will now discontinue clinical and regulatory development of Kyndrisa as well as three other follow-on products, BMN 044, BMN 045 and BMN 053, which are currently in Phase II studies for specific forms of Duchenne muscular dystrophy. The company has, however, indicated that it expects to achieve non-GAAP break-even or better in 2017.

Jean-Jacques Bienaimé, chairman and CEO at Biomarin, comments: “The withdrawl of the MAA and discontinuation of our current experimental drugs for Duchenne is a difficult but necessary decision at this time. We want to extend our sincere gratitude to all of the families and caregivers who supported our efforts over the last year to bring Kyndrisa to patients with Duchenne. Our plan now is to invest in research of next generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder.”

There are few treatments available for this debilitating genetic disease, which often kills by the age of 20. Sarepta Therapeutics is currently waiting on a delayed FDA decision on its own treatment for the disease, which has received negative reviews from the FDA previously.

Biomarin’s stock was down almost 3% in after-hours trading at the time of writing.

Sean Murray

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