FDA Issues Complete Response Letter for BioMarin’s DMD Candidate

January 21, 2016

The FDA has delayed BioMarin’s plans to market drisapersen, the company’s Duchenne muscular dystrophy candidate, with a complete response letter calling for more evidence of the therapy’s benefits.

BioMarin says it is evaluating the FDA’s response letter and will cooperate with the agency to weigh future plans for the candidate, which would have been the first treatment for the deadly disease to come to market.

The company adds that it is studying drisapersen — whose proposed name is Kyndrisa — through ongoing extension studies. The candidate’s regulatory filing in Europe is being reviewed, and BioMarin expects a recommendation from the Committee for Medicinal Products for Human Use around June and the final call by the second half of this year.

BioMarin spokeswoman Debra Charlesworth called the decision a disappointment.

She added that the FDA has asked for another clinical study of drisapersen, but the drugmaker still needs to meet with the agency to discuss specifics on what the trial should include. The company’s next steps will follow that meeting and the European decision, she said.

The CRL comes weeks after the agency informed BioMarin that it was pushing back the PDUFA date of Dec. 27.

Although dozens of Duchenne families pleaded for Kyndrisa’s approval, praising broad benefits, such as improved energy, the clinical results in 326 patients led FDA advisors to decide that the candidate’s efficacy was inconsistent.

News of the CRL was not a surprise to analysts following FDA’s advisory committee meeting. In a Jan. 14 research note, Evercore ISI analyst Mark Schoenebaum said a survey conducted last week showed that only 28 percent of buy-side investors expected an approval.

Now the Duchenne community — as well as BioMarin — will look to competitor Sarepta Therapeutics, whose DMD candidate eteplirsen was scheduled to go before an FDA advisory committee Jan. 22. That meeting has been postponed due to weather.