Regulatory, Setbacks

Sarepta halts its golodirsen Duchenne MD study in UK after patient experiences adverse event

Sarepta said this morning that it has halted dosing its Duchenne muscular dystrophy drug golodirsen at all its UK sites after one of the patients in the trial experienced an adverse event. But the biotech swiftly indicated that it doesn’t expect the halt to last for long.

The news was first reported by EP Vantage writer Jacob Plieth, who noted chatter about the dosing halt on Facebook citing issues with rhabdomyolysis side effects — a breakdown of muscle tissue that triggers severe pain.

Doug Ingram, Sarepta CEO

Sarepta’s shares $SRPT slid 6.5% in pre-market trading as the report circulated.

In a statement sent around after the EP Vantage story hit, Sarepta said that UK trial rules required a stoppage at those sites, even though an independent review board had looked over the safety data and concluded that the study could continue. And it is — outside of the UK. The company sent around a statement saying:

Patients enrolled in the study at UK study sites have temporarily stopped dosing due to UK specific stopping rules, triggered by one serious adverse event (SAE) that could possibly be related to the investigational drug product.

The biotech is now seeking a green light to get dosing started again in the UK.

As Sarepta noted, rhabdomyolysis is not an uncommon symptom of DMD, which causes boys to steadily weaken before it kills them. It’s a rare, deadly disease, and Sarepta has touted early evidence that the drug can produce small quantities of dystrophin, hoping that the FDA will see enough cause to offer an early OK.

That’s a tall order, though, after Sarepta won a controversial approval for Exondys51 that required Janet Woodcock to carry it past a gamut of skeptics at the agency who had been harshly critical of the biotech’s development efforts.

Here’s the full statement from Sarepta:

Study 4045-301 (ESSENCE) is a global, randomized double-blind, placebo-controlled study evaluating efficacy and safety in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exons 45 or 53. Patients enrolled in the study at UK study sites have temporarily stopped dosing due to UK specific stopping rules, triggered by one serious adverse event (SAE) that could possibly be related to the investigational drug product. The study remains blinded and the adverse event observed is consistent with those seen in patients with DMD. The safety data from all patients in the ESSENCE trial were reviewed by an independent Data Monitoring Committee (DMC). The DMC deemed that dosing could continue for all patients. However, based on the UK specific stopping rules of the study, the Medicine and Healthcare products Regulatory Agency (MHRA) required that dosing stop at all UK sites. Sarepta is currently submitting an amendment to the MHRA. Following approval from the MHRA, dosing can be reinitiated at the UK sites.


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