Catalent inks deal to manufacture Sarepta's DMD gene therapy

CDMO giant Catalent inked a deal with Sarepta Therapeutics to manufacture what looks to become the first gene therapy to treat Duchenne muscular dystrophy (DMD).

In November, the FDA gave its thumbs-up to Sarepta’s biologics license application (BLA) that was filed in September seeking accelerated approval of delandistrogene moxeparvovec (SRP-9001) that, if given a final signoff by the regulatory agency, would be the first gene therapy used to treat the condition.

The therapy is designed to deliver the microdystrophin-encoding gene into muscle tissue that triggers production of the microdystrophin protein. Patients with DMD have a mutation in the DMD gene and are unable to produce the protein on their own, leading to a progressive loss of muscle strength.

In addition to the supply agreement for SRP-9001, the deal calls for Catalent to support multiple gene therapy candidates in Sarepta’s pipeline for limb-girdle muscular dystrophy (LGMD), the company said in a Jan. 5 press release. Financial terms of the contract weren’t disclosed.

“Our partnership with the Sarepta team spans nearly a decade across multiple programs and modalities (and) we look forward to working together to manufacture these potentially life-changing and life-saving products for patients diagnosed with DMD and LGMD,” Alessandro Maselli, Catalent’s president and chief executive, said in the release.

In early November, Sarepta issued $1.2 billion in 1.25% convertible senior notes due in 2027 to help bankroll a rollout of the treatment. The company was targeting a goal of raising $2.1 billion in cash and cash equivalents.