Shares of Sarepta SRPT were down 9.4% on Apr 13 after a Stat News article claimed that the FDA was initially against approving the company’s biologics license application (“BLA”) seeking accelerated approval for SRP-9001 to treat Duchenne muscular dystrophy (“DMD”).
Per the 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 official of the agency Peter Marks, who also happens to be an advocate for faster gene-therapy approvals, shifted the FDA’s path toward discussing the BLA at an advisory committee meeting. Per the FDA website, Marks is the current director of the FDA’s Center for Biologics Evaluation and Research (“CBER”).
Earlier this week, Sarepta confirmed that the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee meeting on the SRP-9001 BLA is scheduled next month on May 12. This committee meeting is scheduled just weeks before the expected PDUFA date of May 29, 2023, set by the FDA for the SRP-9001 BLA.
If the article’s contents are true, it puts a question mark on the therapy’s merits claimed by Sarepta, based on which the filing for SRP-9001 was initiated.
Per a Reuters report, the FDA denied making any statements stating that it cannot comment on any unapproved product, while Sarepta is yet to confirm the same.
If approved by the FDA, 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.
Shares of Sarepta have declined 3.8% so far this year compared to the industry’s 3.1% fall.
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SRP-9001 is being developed by Sarepta in partnership with Roche RHHBY. Sarepta and Roche entered into a licensing agreement in 2019 to develop SRP-9001 for DMD. Per the agreement, Roche has exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets
The BLA filing, made to the FDA last September, is based on data from multiple studies in a clinical development program evaluating SRP-9001 in DMD. Last June, 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 times. The time points vary from one-, two- and four years post-treatment.
Sarepta is evaluating SRP-9001 in an ongoing pivotal phase III study, EMBARK, which will serve as a confirmatory study seeking full approval for the therapy in DMD.
Currently, 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
Sarepta Therapeutics, Inc. Price
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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 Novo Nordisk NVO and Ligand Pharmaceuticals LGND, 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 Novo Nordisk’s 2023 earnings per share have increased from $4.20 to $4.51. During the same period, the earnings estimates per share for 2024 have risen from $4.90 to $5.26. Shares of Novo Nordisk are up 23.9% in the year-to-date period.
Earnings of Novo Nordisk 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 3.00%. In the last reported quarter, Novo Nordisk’s earnings beat estimates by 2.47%.
In the past 60 days, estimates for Ligand Pharmaceuticals’ 2023 earnings per share have increased from $3.30 to $4.15. During the same period, the earnings estimates per share for 2024 have risen from $3.10 to $4.57. Shares of Ligand Pharmaceuticals are up 13.0% in the year-to-date period.
Earnings of Ligand Pharmaceuticals beat estimates in one of the last four quarters while missing the mark on three other occasions. On average, the company’s earnings witnessed a negative surprise of 10.07%. In the last reported quarter, Ligand Pharmaceuticals’ earnings beat estimates by 10.57%.
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