Sarepta backs down and pauses Elevidys shipments

The FDA appears to have won its staring contest with Sarepta over shipments of Elevidys, after the company blinked and agreed to the regulator's request to temporarily suspend shipments of the Duchenne muscular dystrophy (DMD) gene therapy.

The FDA asked for a halt to distribution of the gene therapy on Friday, after three deaths among patients treated with Elevidys (delandistrogene moxeparvovec) and a similar experimental gene therapy for limb girdle muscular dystrophy (LGMD), but Sarepta initially refused to comply.

Now, the company has said it will call a halt to shipping in the US at the close of business today, saying in a statement that this will allow it "the necessary time to respond to any requests for information and […] complete the Elevidys safety labelling supplement process," implying the therapy could soon return to the market with tighter warnings. The company hasn't given an indication how long the suspension will last.

Elevidys remains under regulatory review in the EU, where it was filed for approval in June of last year by Roche, which holds marketing rights outside the US. The two companies had already taken the decision to stop supplying it for the treatment of non-ambulatory DMD patients, the group in which two deaths from acute liver failure have been recorded.

Sarepta chief executive Doug Ingram lamented the need to call a halt to shipments, saying that "individuals with Duchenne are losing muscle daily and in need of disease-modifying options," but added that "it is important for the patients we serve that Sarepta maintains a productive and positive working relationship with the FDA."

While the regulator cannot force a company to withdraw a product from sale, it can order a recall of shipments, and there had been speculation that may have been the next step if Sarepta had continued to resist voluntary action.

"Sarepta remains committed to transparency and patient safety and will continue to provide timely updates to patients, families, healthcare providers, and the broader Duchenne community as additional information becomes available," said the biotech.

The FDA asked for a pause after the death of a third patient who had been treated with LGMD candidate SRP-9004 in a clinical trial. In the wake of that fatality, Sarepta announced it was pausing development of its gene therapy pipeline, along with sweeping job cuts, and a refocusing of its R&D efforts around small interfering RNA therapies.

After news of the impasse between the company and the regulator emerged, one healthcare facility involved in treating patients with Elevidys, Children's Hospital Los Angeles, announced it would suspend all dosing of the gene therapy, which might suggest the company is facing some pushback from its certified provider network.