Sarepta SRPT announced that an FDA advisory committee narrowly recommended approving the company’s biologics license application (“BLA”) seeking accelerated approval for SRP-9001 to treat Duchenne muscular dystrophy (“DMD”). The therapy has been developed in collaboration with Roche RHHBY.
The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8:6, with the majority recommending the BLA’s approval. This narrow voting is likely due to the FDA’s concerns on SRP-9001. Per the agency’s briefing documents issued last week, the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 will benefit DMD patients.
Last month, a news article issued by a third-party claimed that the FDA was initially against approving Sarepta’s SRP-9001 BLA. Per this article, some of the FDA’s staff members reached a “non-binding conclusion” that SRP-9001 BLA should be rejected. However, the intervention of a top agency official Peter Marks, who also happens to be an advocate for faster gene-therapy approvals, shifted the FDA’s path toward discussing the BLA at the CTGTAC meeting. Per the FDA website, Marks is the current director of the FDA’s Center for Biologics Evaluation and Research (“CBER”).
A final decision on the BLA is expected before May 29, 2023. Though advisory committee recommendations are not binding on the FDA, they are taken into consideration before arriving at a final decision.
Trading of Sarepta’s shares were halted on May 12 ahead of the CTGTAC decision. In the year so far, the stock has declined 7.2% compared with the industry’s 6.2% fall.
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TheBLA is supported by data from multiple studies in a clinical development program evaluating SRP-9001 in DMD. Last year, Sarepta and Roche announced new functional data across these studies, demonstrating that treatment with SRP-9001 led to functional improvements in individuals suffering from DMD compared with a propensity-weighted external control group multiple time. The time points vary from one, two and four years post-treatment.
If the FDA were to follow the CTGTAC’s recommendation, SRP-9001 will be the first gene therapy for DMD patients. The therapy is also expected to generate a billion dollars in revenue for Sarepta. The gene therapy has been granted Fast Track, Rare Pediatric Disease (RPD) and orphan drug designations by the FDA.
Sarepta is also evaluating SRP-9001 in an ongoing pivotal phase III EMBARK study, which will serve as a confirmatory study seeking full approval in DMD indication.
SRP-9001 has been developed by Sarepta in partnership with Roche, as part of a licensing agreement entered in 2019. Per the agreement, Roche has exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets.
Sarepta’s commercial portfolio consists of three RNA-based PMO therapies targeting DMD — Exondys 51, Vyondys 53 and Amondys 45. These drugs can potentially address nearly a third of all patients with DMD in the United States.
Other than SRP-9001, Sarepta is also developing SRP-5051 (vesleteplirsen), its next-generation exon-skipping pipeline candidate for treating DMD patients with skipping exon 51. This February, management also started an early-stage study (VOYAGENE) evaluating its other gene therapy candidate, SRP-9003, in patients with Limb-girdle muscular dystrophy (LGMD) type 2E/R4
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Zacks Rank & Stocks to Consider
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include ANI Pharmaceuticals ANIP and Ocuphire Pharma OCUP, each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2023 earnings per share have increased from $2.42 to $2.61. Shares of ANI Pharmaceuticals are up 3.6% in the year-to-date period.
Earnings of ANIP Pharmaceuticals beat estimates in each of the last four quarters, delivering an average earnings surprise of 68.64%. In the last reported quarter, ANI Pharmaceuticals’ earnings beat estimates by 244.12%.
In the past 60 days, estimates for Ocuphire Pharma’s 2023 loss per share have improved from 29 cents to 24 cents. During the same period, the loss estimates per share for 2024 have narrowed from 86 cents to 81 cents. Shares of Ocuphire Pharma have surged 70.8% in the year-to-date period.
Earnings of Ocuphire Pharma beat estimates in three of the last four quarters while missing the mark on one occasion. On average, the company’s earnings witnessed a surprise of 23.85%. In the last reported quarter, Ocuphire Pharma’ earnings beat estimates by 42.34%.
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