BioMarin CEO, waiting on FDA on Duchenne drug, eyes Europe

[January 11, 2016]  By Caroline Humer

SAN FRANCISCO (Reuters) - BioMarin Pharmaceutical is expecting U.S. regulators to take action this month on its drug to treat a fatal muscle-wasting disease but is also eyeing the European market, its chief executive officer said in an interview.

The company, which specializes in "orphan" drugs for diseases that affect small numbers of patients and sell for hundreds of thousands of dollars, received an unfavorable opinion from a U.S. regulatory advisory committee in November on the experimental treatment for Duchenne muscular dystrophy.

The move knocked shares back about 10 percent over two days and they are now trading at around $95, far from their peak of $150 in July. Like many drug companies, its shares have fallen on concerns about how political backlash against high drug prices in the United States could affect profits.

In December, BioMarin said the U.S. Food & Drug Administration told the company it would miss its agreed deadline for making a decision on approval. The FDA also said to expect word in early January, the company said.

BioMarin Chief Executive Officer Jean-Jacques Bienaimé said in an interview on Sunday ahead of the annual JP Morgan Healthcare Conference that the decision delay could indicate a disagreement within the FDA about how to proceed.

The FDA could be considering a conditional approval that would require another trial, it could push back the date by which it promises to make a decision, or it could make other requests by issuing what is known as a complete response letter, Bienaime said.

"The fact that they haven't done that could mean there's disagreement," he said.

With BioMarin shares at about $95 or $100, he said, FDA approval was no longer priced into the stock.

BioMarin said that the drug had another possible market in Europe, where there were more than 5,000 patients eligible for the drug. It was expecting to hear from the European regulatory advisory committee on the drug, known as Kyndrisa, in April or May with a final decision in the third quarter.

The disease affects one in 3,600 newborn boys and causes rapid muscle degeneration.

Competitor Sarepta Therapeutics Inc will go before the FDA advisory committee for review in less than two weeks.

(Additional reporting by Carl O'Donnell; Editing by Stephen Coates)

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