FDA Considers Market Withdrawal of Sarepta's Duchenne Gene Therapy Amid Safety Concerns
The U.S. Food and Drug Administration (FDA) is contemplating the potential market withdrawal of Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy, Elevidys, following reports of multiple patient deaths linked to liver toxicity. This development marks a significant setback for the biotech company and raises concerns about the safety of gene therapies using adeno-associated virus (AAV) vectors.
FDA Signals Stronger Action on Elevidys
FDA Commissioner Martin Makary told Bloomberg News that the agency is assessing whether Elevidys should remain on the U.S. market. This statement comes in the wake of three patient deaths associated with Sarepta's gene therapy platform, including two teenagers treated with Elevidys and a 51-year-old man who received an experimental therapy for limb-girdle muscular dystrophy.
An FDA spokesperson confirmed to industry publications that a federal official is "taking a hard look at pulling it from the market." Additionally, the agency plans to request that Sarepta voluntarily halt all shipments of Elevidys, expanding beyond the previous restriction on non-ambulatory patients.
Sarepta's Troubled Week
The news caps off a tumultuous week for Sarepta Therapeutics. On Wednesday, the company announced a major restructuring, including the layoff of approximately 500 employees and a pivot from gene therapy to its siRNA platform assets. The company also disclosed that the FDA would require a new black box warning on Elevidys for liver risks.
However, Sarepta faced criticism for failing to disclose the third patient death during Wednesday's investor call. CEO Douglas Ingram defended the company's decision, stating that the death was "neither material nor relevant" to the information shared during the restructuring announcement.
Safety Concerns and Regulatory Scrutiny
Elevidys, approved by the FDA in June 2023, was hailed as the first gene therapy for Duchenne muscular dystrophy. However, its approval was controversial, with former Center for Biologics Evaluation and Research (CBER) director Peter Marks overruling negative opinions from FDA review teams.
The treatment's safety profile has come under increasing scrutiny following reports of acute liver failure in treated patients. Sarepta has been working on developing new regimens of immune-suppressing drugs, including sirolimus, to mitigate liver toxicity risks.
The recent developments have had a significant impact on Sarepta's stock, which plummeted more than 35% following the news of potential FDA action. The broader biotech market, as measured by the XBI index, also experienced a decline, reflecting investor concerns about the future of gene therapies using AAV vectors.
As the FDA continues its investigation and deliberations, the pharmaceutical industry watches closely, recognizing that the outcome could have far-reaching implications for the development and regulation of gene therapies.
References
- Sarepta Craters as FDA Considers Requesting Halt to Elevidys Shipments
The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a stop to all shipments of the Duchenne muscular dystrophy therapy Elevidys following a third patient death linked to the underlying platform.
- FDA to ask Sarepta to stop shipping Duchenne gene therapy
The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the therapy, Elevidys.
- FDA mulls Elevidys market withdrawal following 3rd death after a Sarepta gene therapy
Although Sarepta initially appeared to have avoided the worst possible outcome for Elevidys—market withdrawal—thanks to the gene therapy’s new black box warning, the FDA is signaling stronger misgivings about the approved drug in the wake of a third patient death from an investigational treatment.
- FDA mulls Elevidys market withdrawal following 3rd death after a Sarepta gene therapy
Although Sarepta initially appeared to have avoided the worst possible outcome for Elevidys—market withdrawal—thanks to the gene therapy’s new black box warning, the FDA is signaling stronger misgivings about the approved drug in the wake of a third patient death from an investigational treatment.
Explore Further
What steps is Sarepta Therapeutics taking to address the liver toxicity issues associated with Elevidys?
How might the potential market withdrawal of Elevidys impact the competitive landscape of gene therapies for Duchenne muscular dystrophy?
What specific clinical data led to the FDA's initial approval of Elevidys despite safety concerns?
Are there alternative gene therapies for Duchenne muscular dystrophy available on the market, and how do they compare to Elevidys?
What potential impact could the FDA’s decision have on the future regulatory process for gene therapies using AAV vectors?