The firm said that the FDA may determine that additional steps beyond Siromilus are recommended or required for non-ambulatory Duchenne muscular dystrophy patients on Elevidys.
Citi said on Wednesday that the U.S. Food and Drug Administration may take a “more comprehensive look” at the level of immunomodulation required for Sarepta Therapeutics’ gene therapy (SRPT) Elevidys.
The FDA may determine that additional steps beyond Siromilus are recommended or required for non-ambulatory Duchenne muscular dystrophy (DMD) patients on Elevidys, Citi said, as per TheFly. Siromilus belongs to a group of medicines known as immunosuppressive agents and has shown promise in managing certain aspects of liver disease and its complications.
Citi has a ‘Sell’ rating and a $14 price target on Sarepta shares. On Stocktwits, retail sentiment around Sarepta fell from ‘bullish’ to ‘neutral’ territory over the past 24 hours, while message volume stayed at ‘low’ levels.
Citi made the observations in light of the FDA’s recommendations for Rocket Pharmaceuticals (RCKT) made public earlier in the day.
Rocket said earlier on Wednesday that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its mid-stage study of RP-A501 for the treatment of Danon disease. The hold, imposed roughly three months ago following the death of a patient in the trial, was lifted after Rocket satisfactorily addressed issues outlined in the clinical hold, the company said.
The agency allowed the trial to resume at an adjusted dose of RP-A501 in three patients, with a minimum four-week interval between dosing and with an immunomodulatory regimen more closely reflecting the one administered in an early-stage study pediatric cohort.
Sarepta, like Rocket Pharma, has been plagued by patient deaths. It’s gene therapy, Elevidys, a medicine intended for the treatment of Duchenne muscular dystrophy (DMD), created safety concerns following the death of two patients after treatment earlier this year. Both non-ambulatory patients suffered from acute liver failure.
Sarepta has now resumed shipments for ambulatory individuals after a temporary pause initiated in late July. The company is now working with the FDA on the safety labelling process and risk-mitigation approach for non-ambulatory individuals.
SRPT stock is down by 83% this year and by about 86% over the past 12 months.
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