Sarepta’s Stocks Plunge After FDA Received Report That Boy Was Injured In Gene-Therapy Study
Sarepta argues that the report was submitted to the FDA in error. The biotech company said the study involves two arms, with one receiving the drug and the other placebo, and the patient could have been on either. In other pharmaceutical news: a heart drug to get reviewed by the FDA, the benefits of Medicare's decision to cover CAR-T therapy, and a call for pricing reforms.
Reuters:
Sarepta Says Adverse Event Report For DMD Gene Therapy Erroneously Submitted
Sarepta Therapeutics Inc said on Thursday it was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company's gene therapy for Duchenne muscular dystrophy. Shares of the company plunged as much as 19% earlier after a notice from the Food and Drug Administration showed a patient in the study was reported to have developed a serious illness. The stock pared losses to close down about 7% after the company's statement. (8/9)
Stat:
Sarepta's Stock Price Plummets After An 'Erroneous' Report
It was not an executive shakeup that pummeled shares of Sarepta Therapeutics (SRPT) on Thursday. It was not rumors of a takeover Wall Street didn’t like. Instead, it was a mistake. Shares cratered 12.6% after an analyst pointed out that a patient in one of the company’s ongoing clinical trials for Duchenne muscular dystrophy reported a serious side effect. The stock was then halted, and remained halted for three hours. (Sheridan, 8/8)
Stat:
Amarin Now Says FDA Plans Outside Panel Review Of Its Vascepa
Remember when Amarin (AMRN) said, a little more than one week ago, that the Food and Drug Administration was “unlikely” to convene an advisory committee meeting to review data on an expanded use of its heart drug Vascepa? Wrong. On Thursday, Amarin reversed course, announcing that, indeed, the FDA has scheduled a Vascepa advisory committee meeting for Nov. 14. The agency’s deadline for making an approval decision on the heart drug will also likely be extended into late December from Sept. 28, the company said. (Feuerstein, 8/8)
KCUR:
Medicare's Decision To Cover A Pricey Cancer Treatment Could Be A Lifesaver For KU Patients
Officials with Medicare have decided to cover an innovative but extremely expensive cancer treatment, setting the stage for more patients to get it. That's good news for the University of Kansas Health System.KU has been a pioneer in using the treatment, known as CAR T-cell therapy, which involves removing a patient’s T cells (a type of white blood cell) and genetically engineering them to recognize and attack the patient’s tumors. The cells are then put back into the patient’s body. (Margolies, 8/8)
New Hampshire Union Leader:
NH Businesses, Unions And Mayors Call For Drug Pricing Reforms
An eclectic group of mayors, union and business leaders launched a new transparency project aimed at influencing decision-makers in the government and private sectors to force pharmacy benefit managers to pass on lower drug costs to their customers and taxpayers. Based on a successful 2017 model in New Jersey that saved more than $1 billion, organizers of the PBM Accountability Project of New Hampshire claim the same reform here could save between $30 million and $40 million annually. (Landrigan, 8/8)
