Biomarin Pharmaceutical’s Kyndrisa was not enough to convince the FDA of its efficacy


A drug for treating Duchene Muscular Dystrophy has been deemed unsatisfactory in action by the FDA. Both Parent and patient testimony about the changes they experienced after taking Biomarin Pharmaceutical Inc. Kyndrisa was not enough to convince the FDA advisory committee of its efficacy.

A bench of outside advisors to the regulatory agency United States FDA said that the data from BioMarin Pharma Inc. on its experimental drug for treating a chronic muscle wastage disorder was not enough to pave the way for its approval.

The FDA panel did not vote since 15 out of 17 members were of the opinion that paucity of statistical significance in BioMarin’s late-stage study weakened the findings from two earlier studies. The drug in question is ‘Drisapersen’ which is being developed to treat Duchenne Muscular Dystrophy.

DMD affects one in 3,600 newborn boys and leads to a rapid degeneration of muscles. The fate of the drug will be decided on December 27 after the evaluation of the panel’s comments.

Another drugmaker, Sarepta Therapeutics Inc is also developing the DMD treatment eteplirsen whose action is much akin to BroMarin’s drisapersen and skips a faulty section of the gene to produce dystrophin, the lack of which causes DMD. If the FDA goes along with the concerns of the panel, more trials will be required, and it will consume time that patients with DMD don’t have.

Sarepta shares went down 1% at $36.98 after bell while the shares of another company PTC Therapeutics Inc which is also developing a treatment for DMD fell by 5% in after-hours trading to $30. BioMarin’s stock was placed on halt for all of Tuesday.

RBC Capital Markets analyst Michael Yee feels that there is a 50 to 60% chance of approvals. The fact that some patients benefited from the drug and the willingness of the patients to take the risk could turn over the opinion to positive for the drug.