BioMarin Fails to Win Approval for Drug to Treat Duchenne

  • FDA told company it didn't hit `substantial evidence' standard
  • Stock declines, analyst said investors had expected rejection

BioMarin Pharmaceutical Inc. failed to win U.S. approval for a drug to treat Duchenne muscular dystrophy, a deadly genetic disease that usually affects young boys.

The Food and Drug Administration rejected the once-a-week injection known as Kyndrisa, according to a statement from the company Thursday. A panel of outside advisers voted in November that the medication, known chemically as drisapersen, didn’t conclusively show improved walking ability in patients with the muscle-wasting disease.

“FDA has concluded that the standard of substantial evidence of effectiveness has not been met,” BioMarin said in the statement announcing the rejection. “BioMarin is reviewing the Complete Response Letter and will work with the FDA to determine the appropriate next steps regarding this application.”

The FDA asked BioMarin to conduct anther trial before it reconsiders the drug, Debra Charlesworth, a company spokeswoman, said in a phone interview. The agency hasn’t provided specific details of what it wants yet, and the company plans to meet with the agency to discuss the matter.

The shares dropped 5.1 percent to $79.82 at 9:53 a.m. in New York. They’ve fallen 42 percent in the last six months, as of Wednesday’s close. Sarepta Therapeutics Inc., which is developing a competing drug and has a ruling from the FDA scheduled next month, fell 4.1 percent to $30.72.

Michael Yee, an analyst with RBC Capital Markets, said the rejection wasn’t a surprise and could remove a negative overhang on the stock.

“Investors nearly fully expected a rejection of drisapersen,” he said in a note to clients Thursday. “The stock should bounce back now that this is out of the way and we can move on to the next pipeline catalysts.”

Other Products

BioMarin Chief Executive Officer Jean-Jacques Bienaime has said that the scrutiny of Kyndrisa has left other products in the drugmaker’s pipeline underappreciated.

“There’s been an obsession with DMD in the past three months, which in some ways is understandable, but there are huge things that are missed by some analysts and investors,” Bienaime said in an interview published this week. The company plans to unveil data from several other experimental drugs later this year.

BioMarin is disappointed by the FDA’s decision but feels good about the coming year, Charlesworth said. The drugmaker expects to potentially file applications in 2016 to sell treatments for a genetic condition called phenylketonuria that causes a build up of a certain amino acid in the blood, and for a neurodegenerative condition known as Batten disease, Charlesworth said.

She declined to comment on whether BioMarin will conduct another clinical trial to attempt again to obtain FDA approval for Kyndrisa.

Warning, or Opportunity?

The rejection of Kyndrisa could be seen as a warning sign or an opportunity for Sarepta, which is developing a competing treatment for Duchenne muscular dystrophy. The company’s drug for the disease faces FDA advisers on Jan. 22 and the agency will decide whether to approve it by Feb. 26. The rejection doesn’t bode well for PTC Therapeutics Inc., which is developing a treatment for Duchenne muscular dystrophy that also has had issues with inconclusiveness in clinical trials.

Duchenne muscular dystrophy is a progressive disease in which the body lacks a protein, called dystrophin, that keeps muscles intact. It strikes in childhood, and patients typically end up in wheelchairs. Many die by age 25 from lung disorders, according to the National Institutes of Health. Kyndrisa is designed to fix a mutation in patients’ genetic code, in effect “skipping” the mutation to help the body produce dystrophin.

Two of the three clinical trials that BioMarin relied on to persuade the FDA to approve Kyndrisa failed to meet their main goal, which was to help patients taking the drug to walk farther in six minutes -- a standard assessment used in treatments for similar diseases. Kyndrisa was meant for patients with a certain genetic mutation, making about 2,300 who would have been eligible to take the drug.

European regulators are still reviewing BioMarin’s application to sell Kyndrisa there. The company said it will continue ongoing clinical trials of the drug.

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