Morning Briefing
Summaries of health policy coverage from major news organizations
Sarepta's Stocks Plunge After FDA Received Report That Boy Was Injured In Gene-Therapy Study
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)
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鈥檛 like. Instead, it was a mistake. Shares cratered 12.6% after an analyst pointed out that a patient in one of the company鈥檚 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)
Remember when Amarin (AMRN) said, a little more than one week ago, that the Food and Drug Administration was 鈥渦nlikely鈥 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鈥檚 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)
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鈥檚 T cells (a type of white blood cell) and genetically engineering them to recognize and attack the patient鈥檚 tumors. The cells are then put back into the patient鈥檚 body. (Margolies, 8/8)
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)
