Good afternoon from STAT’s London outpost, with Andrew Joseph right here on rotation for the vacationing Mr. Pharmalot. We’re on the level within the day once we’re debating whether or not one other cup of stimulation may snap us out of the afternoon doldrums or wreak havoc on our bedtime, however for these of you stateside, a recent day awaits. Listed here are some tidbits that can assist you in your approach. …
Sarepta Therapeutics, the maker of a gene remedy for Duchenne muscular dystrophy that’s being briefly shelved due to security issues, faces an “arduous and treacherous path” to attempt to get it again onto the market, in response to a senior U.S. Meals and Drug Administration official, per STAT’s reporting. The assertion means that the therapy’s license might be revoked, although the official didn’t rule out the likelihood that the remedy, Elevidys, may finally be introduced again to the market. However it could be troublesome for Sarepta to show that any new security protocols may get rid of the danger of liver accidents — which have been tied to the deaths of two sufferers — the official added.
In a associated story, Roche is following its associate Sarepta Therapeutics and stopping shipments of Elevidys in some nations, amid rising security issues surrounding the therapy, STAT additionally brings us. Roche, which markets Elevidys outdoors the US, mentioned it was “initiating a voluntary and momentary pause” on new orders in nations that depend on FDA rulings for his or her native approvals. It should, nevertheless, proceed to provide Elevidys to nations that authorized the gene remedy impartial of the FDA, together with Brazil and Japan. It should solely be obtainable to Duchenne sufferers who can nonetheless stroll.
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