Tag Archives: dmd

Sarepta Gets Second Wind, As FDA Delays Drug Decision — Again

Biotech Sarepta Therapeutics ( SRPT ) soared in early trading Wednesday after the Food and Drug Administration delayed making a decision on whether to approve its muscular dystrophy drug, giving new hope for a product that Wall Street had generally given low odds. The FDA’s self-imposed deadline had been Thursday, which itself was pushed back three months from the original decision date. Sarepta’s statement didn’t mention a new deadline, just saying that the reviewers “will not be able to complete their work” by the current deadline. Sarepta’s drug eteplirsen, for a subgroup of patients with Duchenne muscular dystrophy (DMD), had always had a tough case to make based on a clinical trial with only 12 subjects and no internal control group. The odds of success looked even lower when the majority of an FDA advisory committee last month voted not to recommend approval. However, families of patients with DMD, who generally don’t live past the age of 30, have been lobbying hard for some treatment to come on the market, since currently none are available. Analyst Simos Simeonidis of RBC Capital Markets suspects that this is the reason for the delay, but he still gives the drug only a 30% chance of approval. “Despite the fact that today’s news slightly increases the chances of an approval, we have a hard time imagining the agency setting the precedent of approving a drug with such a limited dataset,” Simeonidis wrote in a research note. “On the other hand, we cannot overlook the tremendous pressure being put on the FDA, and it is very difficult to gauge what may happen behind the scenes at the agency at the last minute.” Sarepta stock shot up 20% soon after the opening bell on the stock market today , near 22.

2 Hotly Debated Drugs Due For FDA Decisions This Week

The FDA is due to decide whether to approve two of the most divisive drugs in the biotech industry this week, both of them crucial for the companies that make them, Sarepta Therapeutics and Intercept Pharmaceuticals. Thursday is the deadline for eteplirsen, Sarepta Therapeutics ‘ ( SRPT ) treatment for Duchenne muscular dystrophy (DMD). Just getting to the filing took years of negotiation between Sarepta and the FDA, and May 26 is three months later than the decision was originally supposed to be made. And after all that, Wall Street is placing low odds on the drug’s approval. That’s based mainly on last month’s advisory committee meeting on eteplirsen, which voted 7 to 3 (with 3 abstentions) that the drug’s effectiveness wasn’t adequately proven by Sarepta’s tiny clinical trial. Sarepta stock hit a four-year low of 8, on April 26, as multiple analysts downgraded it on the assumption the drug will be rejected. Nonetheless, DMD is a devastating disease without a current treatment, and the families of the children affected have turned into a formidable lobby to get something on the market — especially after BioMarin Pharmaceutical ‘s ( BMRN ) rival drug Kyndrisa was rejected in January. Some observers say the families’ desperation might be enough to move the agency. Oppenheimer analyst Christopher Marai on May 2 upgraded Sarepta on the grounds that, in the past, the FDA has been more flexible in such cases of unmet need. He also pointed out that the stock was now cheap. Sarepta stock has remained volatile but has steadied somewhat the past couple of weeks. On Friday, shares jumped 8.8%, to 19.15. Intercept’s Liver-Disease Treatment Expected To Get OK A day after the Sarepta decision, Intercept Pharmaceuticals ‘ ( ICPT ) liver-disease treatment Ocaliva is due for a verdict. Ocaliva’s advisory committee vote last month went the opposite way of eteplirsen’s, with the panel unanimously supporting approval for the drug, which treats the rare disease primary biliary cholangitis (PBC). As a result, Wall Street generally expects approval. Bears, however, are still growling about Ocaliva’s safety, which they say could limit the drug’s label. The day after the panel vote, Morgan Stanley analyst Andrew Berens drove down Intercept’s stock with the theory that the FDA would discourage use in patients with advanced cirrhosis. This might affect not only the relatively small PBC market, but also the much, much bigger possibilities for Ocaliva in non-alcoholic steatohepatitis (NASH). Intercept is testing the drug in NASH, and approval wouldn’t be for a couple of years, but it’s a major part of the stock’s bull thesis, as analysts see annual sales in the multi-billions. Intercept stock has been on a downtrend this month, but it rose 2.4% this past week. On Friday, shares closed up 2.2%, at 134.28.

2 Hotly Debated Drugs Due For FDA Decisions Next Week

In the week before Memorial Day, the FDA is due to decide whether to approve two of the most divisive drugs in the biotech industry, both of them crucial for the companies that make them, Sarepta Therapeutics and Intercept Pharmaceuticals. Thursday is the deadline for eteplirsen, Sarepta Therapeutics ‘ ( SRPT ) treatment for Duchenne muscular dystrophy (DMD). Just getting to the filing took years of negotiation between Sarepta and the FDA, and May 26 is three months later than the decision was originally supposed to be made. And after all that, Wall Street is placing low odds on the drug’s approval. That’s based mainly on last month’s advisory committee meeting on eteplirsen, which voted 7 to 3 (with 3 abstentions) that the drug’s effectiveness wasn’t adequately proven by Sarepta’s tiny clinical trial. Sarepta stock hit a four-year low of 8, on April 26, as multiple analysts downgraded it on the assumption the drug will be rejected. Nonetheless, DMD is a devastating disease without a current treatment, and the families of the children affected have turned into a formidable lobby to get something on the market — especially after BioMarin Pharmaceutical ‘s ( BMRN ) rival drug Kyndrisa was rejected in January. Some observers say the families’ desperation might be enough to move the agency. Oppenheimer analyst Christopher Marai on May 2 upgraded Sarepta on the grounds that, in the past, the FDA has been more flexible in such cases of unmet need. He also pointed out that the stock was now cheap. Sarepta stock has remained volatile but has steadied somewhat the past couple of weeks. On Friday, shares jumped 8.8%, to 19.15. Intercept’s Liver-Disease Treatment Expected To Get OK A day after the Sarepta decision, Intercept Pharmaceuticals ‘ ( ICPT ) liver-disease treatment Ocaliva is due for a verdict. Ocaliva’s advisory committee vote last month went the opposite way of eteplirsen’s, with the panel unanimously supporting approval for the drug, which treats the rare disease primary biliary cholangitis (PBC). As a result, Wall Street generally expects approval. Bears, however, are still growling about Ocaliva’s safety, which they say could limit the drug’s label. The day after the panel vote, Morgan Stanley analyst Andrew Berens drove down Intercept’s stock with the theory that the FDA would discourage use in patients with advanced cirrhosis. This might affect not only the relatively small PBC market, but also the much, much bigger possibilities for Ocaliva in non-alcoholic steatohepatitis (NASH). Intercept is testing the drug in NASH, and approval wouldn’t be for a couple of years, but it’s a major part of the stock’s bull thesis, as analysts see annual sales in the multi-billions. Intercept stock has been on a downtrend this month, but it rose 2.4% this past week. On Friday, shares closed up 2.2%, at 134.28.