Sarepta Gets Thumbs Down From Regulators Regarding Elevidys Gene Therapy

On Friday, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) and Roche Holdings AG (OTC:RHHBY) are collaborating to develop and commercialize gene therapy.

Also Read: Elevidys Halt Sparks Analyst Downgrades—Sarepta Races To Revise Label

The CHMP opinion is based on data from the largest and broadest gene therapy clinical programme in DMD to date, including results from the pivotal Phase 3 EMBARK study, which showed that treatment with Elevidys provided sustained stabilisation or slowing of disease progression and a consistent and manageable safety profile in ambulatory patients.

To date, more than 900 individuals with DMD, 760 of whom are ambulatory, have been treated with Elevidys in clinical and real-world settings.

While the primary endpoint was not met in EMBARK after one year, Elevidys showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo.

Longer-term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other Elevidys studies that showed clinically meaningful improvements across key measures of motor function.

On Monday, Sarepta said it will voluntarily and temporarily pause all U.S. shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, effective July 22.

According to a media report on Thursday, a senior FDA official had stated that the Center for Biologics Evaluation and Research would require additional clinical data to validate Elevidys’ safety profile before returning to the market.

William Blair says it’s unclear what more the FDA wants before allowing Elevidys shipments to restart. However, requiring Sarepta to run another trial in ambulatory patients would be unusual, since Elevidys already has full approval for that group and there have been no deaths among those patients.

Analyst Sami Corwin pointed out that three patient deaths linked to severe liver damage after receiving Novartis AG’s (NYSE:NVS) Zolgensma were reported in the FDA’s adverse event database.

However, the FDA did not stop shipments of that drug, which suggests its cautious approach may be limited to Sarepta's products. While there’s a chance the current pause could be lifted by the end of the year—especially for younger patients who can still walk—ongoing uncertainty about how long the pause will last and what steps are needed to resume treatment may keep pressure on the stock.

On Wednesday, Sarepta partner Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) said Sarepta has recently experienced setbacks in products and programs unrelated to those licensed from Arrowhead. 

Arrowhead believes it is on track to earn the first $100 million soon and the remaining $200 million by the end of the year. 

Arrow added should Sarepta fail to make either the $100 million or $200 million near-term payment associated with enrollment of ARO-DM1 cohorts, Arrowhead would have the right to terminate the agreement with respect to ARO-DM1.

Price Action: SRPT stock is down 7.53% at $11.91 at the last check on Friday.

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