FDA gives the go-ahead to restart Elevidys shipments

It turns out that the pause on shipments of Sarepta's Duchenne muscular dystrophy (DMD) gene therapy Elevidys over safety concerns has been short-lived.

The FDA's request to suspend distribution of the gene therapy, initially resisted by the company, ultimately lasted only around 10 days, and Sarepta has confirmed they have now resumed – at least for ambulatory DMD patients.

In a statement, the Cambridge, Massachusetts-based company said that shipments of Elevidys (delandistrogene moxeparvovec) will restart while it continues dialogue with the FDA "on next steps in the safety labelling process and risk-mitigation approach for non-ambulatory patients."

The FDA asked for a halt to distribution of the gene therapy on 18th July, after three deaths due to acute liver failure among patients treated with Elevidys and SRP-9004, a similar gene therapy in clinical development for limb girdle muscular dystrophy (LGMD).

Late last week, the FDA said it was also investigating a fourth death in June of an eight-year-old boy in Brazil who had been treated with Elevidys, although Sarepta and its ex-US commercial partner for the gene therapy, Roche, said it was unlikely to be related to treatment. That view was confirmed by the US regulator yesterday.

A voluntary hold remains on the use of Elevidys in sicker, non-ambulatory patients, the group in which the two fatalities occurred. Elevidys has been granted full FDA approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory patients.

Roche also temporarily paused shipments of Elevidys in some markets outside the US after the FDA's request, extending to countries that reference the FDA as the grounds for approval – which does not include Brazil – but said it would continue to distribute the drug in other markets where the gene therapy has launched.

Sarepta chief executive Doug Ingram said he was "very pleased" that the FDA had completed its review of available safety information promptly, adding that it "evinces a commitment to the Duchenne population."

Patient advocacy group Parent Project Muscular Dystrophy (PPMD) said it "deeply appreciates" the swift resolution of the investigation, adding that – according to Sarepta - the company will be reaching out to sites of care that had infusions scheduled for July and August to inform them of the availability of Elevidys for ambulatory patients.

A letter from Sarepta's head of patient affairs, Wendy Erler, to the DMD patient community said the company will work with the FDA on recommendations for supplemental immunosuppression that could enhance the safety of Elevidys in non-ambulatory patients.

There is no doubt that the move to resume shipments of Elevidys, which contributed sales of around $282 million in the second quarter out of total product revenues of $513 million, is a massive relief for Sarepta, whose management came under pressure from shareholders as the safety review rumbled on.

The company has just implemented a massive restructuring of its business to cut costs, pausing development of its gene therapy pipeline, along with sweeping job cuts, and narrowing its R&D efforts around small interfering RNA therapies.

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