Data does not support nod for BioMarin’s DMD drug: FDA staff

By Vidya L Nathan

(Reuters) – Data on BioMarin Pharmaceutical Inc’s experimental treatment for a muscle-wasting disease did not warrant an approval of the drug at this time, the U.S. Food and Drug Administration’s health regulator’s staff reviewers said.

BioMarin’s shares, which have lost a quarter of their value in the past four months, were down about 5 percent at $98.26 on Friday. Sarepta Therapeutics Inc, which is developing a rival treatment, jumped 28 percent to $33.37.

The FDA staff reviewers said the drug, drisapersen, did not improve the production of a protein called dystrophin, the lack of which causes Duchenne’s muscular dystrophy.

The disease affects one in 3,600 newborn boys and there is no approved treatment.

Drugs for rare diseases cost more as there are few available treatment options and only a handful of companies are involved in developing them.

“The long term study does not provide supportive evidence of effectiveness for drisapersen at this time,” staff members said in a document released on Friday. (http://1.usa.gov/1I3wGfR)

The document comes two days before a panel of outside experts vote on the drug’s approval. While the FDA is not obligated to follow the panel’s recommendation, it mostly does so. The FDA is slated to decide on drisapersen’s approval by Dec. 27.

“In our view, the FDA has made a very clear statement that there is no reason to approve drisapersen,” Baird Equity Research’s analyst Brian Skorney said.

The FDA staff reviewers also expressed concerns over drisapersen’s safety and recommended a boxed warning, which indicates severe side effects, if the drug is approved.

The staff also noted kidney problems seen in 61 percent patients who were given the drug and a severe reduction in platelets, that increased the risk of death on long-term use of the drug.

Some analysts, however, said the drug could still get the nod from the FDA.

RBC Capital Market’s Michael Yee said voting questions put forward by the staff reviewers to an outside panel of experts do not include a vote on the potential approval.

The company could have further discussions with the regulator and identify a path to approval, Yee said.

Piper Jaffray’s Joshua Schimmer said that while the FDA staff was highly critical of the drug, they stopped short of recommending against drisapersen’s approval.

BioMarin, which got drisapersen when it bought Prosensa Holding NV for $840 million last year, is still testing the long-term effects of drisapersen in DMD patients in a study that is expected to be completed by April.

(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D’Souza and Saumyadeb Chakrabarty)

• Health
• Pharmaceuticals Drug Trials
• BioMarin Pharmaceutical Inc
• Food and Drug Administration