Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to all patients who have the muscle-wasting disease. Analysts say prospects of the therapy, Elevidys, rest on FDA willingness to exercise flexibility it has already shown to rare disease drugmakers, including Sarepta.
Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.
Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.
The FDA decision gives Sarepta Therapeutics its third approved drug for Duchenne muscular dystrophy. The accelerated approval requires the biotech to conduct additional clinical testing to confirm the drug’s benefit.
Despite a strongly critical complete response letter - which the FDA sent in August but did not make public until Tuesday - the drugmaker successfully appealed and won approval for the drug, Vyondys 53, in December, to the surprise of many.
An analyst wrote that the deal would create value for Sarepta, which has a gene therapy in Phase II development for Duchenne muscular dystrophy, SRP-9001, but lacks the resources to commercialize it abroad.
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
The FDA had previously rejected Sarepta's application for Vyondys 53 in August, citing infections and kidney toxicity in preclinical studies. However, some analysts saw the move as a 'slap on the wrist' for the controversial approval of Sarepta's first DMD drug.
Several FDA approvals - including ones going against AdCom votes - have attracted controversy and a perception of lowered standards at the agency. However, some see a long-standing institutional tension between patient protection and speedy approval, with the latter having a net positive effect.
In a note to investors, SVB Leerink analyst Joseph Schwartz suggested that the 'questionable' approval of Sarepta's Exondys 51 may have played a role in the FDA's decision to turn down golodirsen.
The decision stood in contrast with the agency's highly controversial approval of Sarepta's previous DMD drug, Exondys 51. The company's shares fell sharply in after-hours trading.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
While there were also bright spots - like CMS' decision on CAR-T therapy - several biopharma companies experienced unrelated setbacks this week that caused their shares to tank.
Although the agency goes against about one-fifth of AdCom recommendations, a study published Sunday only found one reliable variable predicting when that would happen.
Data from the drugmaker's Phase Ib study showed patients responding, but an analyst wrote that it appears less potent and to have higher toxicity than that of its competitor, Sarepta Therapeutics.
Sarepta Therapeutics gains rights to University of Florida-resident startup's programs for central nervous system diseases.
Checkpoint Pharmaceuticals wants to advance a drug that boosts immune activity to make checkpoint inhibitors work better.
