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The FDA announced Monday at Sarepta Therapeutics a "surprise" rejection, informing the company in a full response letter (CRL) that it was spending its second drug against Duchenne Muscular Dystrophy (DMD) because of risks of infection by wearing perfusion and renal toxicity animal studies.
The first action of Sarepta will be to meet with the FDA to negotiate a black box warning or other labeling of the drug, the golodirsen, to clarify the risk of kidney toxicity and the risk of infection, rather than carry more studies, wrote ISC Evercore analysts in a note on Tuesday. But the implications of rejection go beyond golodirsen.
"The real drivers of LCR's ethics of golodirsen will continue to be a debate based on limited public disclosure of the granularity of the facts, but the overall conclusion is clear in our mind – there is a regulation" that raises the bar higher , "The analysts wrote. This could be bad news for other Sarepta programs, namely its microdystrophin gene therapy, which Evercore ISI analysts consider to be "the main driver of evaluation." [Sarepta] Stock."
RELATED: FDA surprises Sarepta by rejecting his second drug Duchenne
Sarepta pioneered its pipeline with its first DMD drug, Exondys 51, which works in approximately 13% of patients. The company got a quick approval for the drug in 2016, using a surrogate biomarker assessment criterion and without showing any test data proving that the treatment improves muscle function. She promised to conduct a post-approval confirmation test to provide this information, but three years later, the FDA is still waiting.
At the time, the approval was controversial given the limited effectiveness of the treatment and appeared to result in a split of the FDA, some advocating a more relaxed approach to the approval of the other while others opponent vehemently.
Although Sarepta stated that the rejection was not related to efficiency, it should be noted that he used the same strategy for golodirsen. Analysts have written that the microdystrophin works with what it thinks the FDA will accept for approval:
"Yesterday's retreat of golodirsen is a reminder that the consensus pathway for consensus for μdystrophin is based on the" assumption "of the SRPT on what the FDA will accept for approval, including : (1) the transition between 102 and 103 studies and concomitant (2). ) expression / function correlation at 12 months using [the North Star Ambulatory Assessment] as a functional endpoint, "the analysts wrote.
RELATED: Sarepta plunges into a controversial report on hospitalization in Duchenne
Although the promise of Sarepta's microdystrophin gene therapy has been attractive enough that "the share price has largely been able to absorb / greatly reduce the increased implied risk," analysts "wonder" whether of society will swing in the opposite direction. The communications of Sarepta.
The recent communication strategy of Sarepta "on many points is atypical compared to the traditional communication of biotechnology companies – press release yesterday [about golodirsen] is obviously only the most recent example, "the analysts wrote.
Golodirsen rejection follows entry into the FDA adverse event reporting system (FAERS), discovered earlier this month, that a 7-year-old child was enrolled in an SRM-9001 gene therapy study Sarepta was hospitalized after developing rhabdomyolysis. A few hours later, the company said the report was "wrongly submitted" to the FAERS database, "a post-marketing surveillance database for approved therapies."
"Our investigation to date indicates that this report was not submitted to the FAERS database by a Sarepta employee or by the principal investigator of the study," the company said in a statement. a statement.
The child was hospitalized in February but was only revealed months later, raising questions as to whether Sarepta was trying to keep the hospital in secret. The news followed a similar hiccup at the golodirsen in February 2018, when the company stopped taking a British study because of "a serious adverse event that could possibly be linked to the experimental drug product," Sarepta said in a statement. communicated by e-mail.
In an article posted on Facebook by a parent whose child participated in this study, the adverse event was actually a rhabdomyolysis.
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