By Christy Santhosh and Mrinalika Roy (Reuters) -Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as the company faces intense
Sarepta's Gene Therapy Faces EU Rejection Amid Regulatory Challenges
By Christy Santhosh and Mrinalika Roy
(Reuters) -Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as the company faces intense regulatory scrutiny after two recent patient deaths tied to the treatment.
Shares of the Cambridge, Massachusetts-based company slumped 11%. They have lost 88.8% so far this year.
The EU committee's negative opinion for Elevidys is yet another major setback for the company, which was directed by the U.S. health regulator earlier this month to stop all shipments of the gene therapy — the only one approved by it for treating Duchenne muscular dystrophy (DMD).
Sarepta had already halted dosing in non-ambulatory patients, meaning those unable to walk independently, and later complied with the U.S. Food and Drug Administration's request to pause all shipments, along with Roche — its partner outside the U.S.
"While we are disappointed by CHMP's (Committee for Medicinal Products for Human Use) negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne," said Sarepta's research and development head, Louise Rodino-Klapac.
DMD is primarily seen in males, affecting 1 in 5,000 boys born worldwide. The average life expectancy for someone with the disorder is 22 years.
William Blair analyst Sami Corwin said the fall in Sarepta shares reflected the reduced revenue from potential future milestone and royalty payments from Roche.
"While we believe there is a possibility the pause on Elevidys shipments in the U.S. is lifted before year-end specifically for young and ambulatory patients... the uncertainty will continue to pressure stock."
Roche said on Friday it would continue its dialogue with the European Medicines Agency to explore a potential path forward to make Elevidys available in the EU.
The benefit-risk profile remains positive in the ambulatory patients, the Swiss drugmaker said.
Sarepta said it continues to work with the FDA in the U.S. to address recent safety concerns related to Elevidys.
(Reporting by Christy Santhosh in Bengaluru; Editing by Shilpi Majumdar)
