Sarepta Therapeutics,, Inc. - SRPT STOCK NEWS

Sarepta Therapeutics announced that the FDA Cellular, Tissue and Gene Therapies Advisory Committee voted 8-6 in favor of accelerated approval for SRP-9001, a gene therapy designed to treat Duchenne muscular dystrophy (DMD). The therapy aims to deliver a gene coding for a shortened, functional form of dystrophin to muscle cells, addressing the underlying cause of DMD.

The committee's decision was based on non-clinical evidence and data from studies 101, 102, and 103, along with an integrated analysis comparing functional results to a propensity-score-weighted external control. While not binding, this vote will be considered by the FDA in its final decision, with a regulatory action date set for May 29, 2023.

SRP-9001 is currently under priority review by the FDA. Sarepta plans to commercialize the therapy in the United States upon approval, while Roche will handle distribution outside the U.S. as part of a partnership formed in December 2019.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) will release its first quarter 2023 financial results after market close on May 2, 2023. A conference call to discuss these results will be hosted at 4:30 p.m. E.T. The event will be available via webcast on the investor relations section of Sarepta's website.

The company focuses on precision genetic medicine for rare diseases, holding leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs). It boasts a pipeline of over 40 programs in development, driven by its Precision Genetic Medicine Engine employing gene therapy, RNA, and gene editing technologies.

Sarepta Therapeutics (NASDAQ: SRPT) announced the date of the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee meeting for the SRP-9001 biologics license application (BLA) on May 12, 2023. This virtual meeting is critical as it precedes the regulatory action date of May 29, 2023, for the investigational gene therapy targeting Duchenne muscular dystrophy.

CEO Doug Ingram expressed anticipation in showcasing the evidence of SRP-9001’s transformative potential. The therapy aims to deliver functional dystrophin components to muscle tissue, essential for patients suffering from Duchenne muscular dystrophy. Sarepta plans to commercialize SRP-9001 in the U.S. post FDA approval and has partnered with Roche to enhance global access to this promising therapy.

Sarepta Therapeutics (NASDAQ:SRPT) announced the granting of equity awards to 18 new employees as part of its 2014 Employment Commencement Incentive Plan, approved by the Compensation Committee on March 31, 2023. The awards include options to purchase 11,075 shares of common stock at an exercise price of $137.83 per share, and 5,700 restricted stock units (RSUs). The options will vest over four years, while the RSUs will vest yearly on anniversaries of the grant date. Sarepta focuses on precision genetic medicine aimed at rare diseases, with over 40 programs in development.

Sarepta Therapeutics (NASDAQ:SRPT) announced that the FDA's Office of Therapeutics will hold an advisory committee meeting regarding the SRP-9001 biologics license application (BLA) before the regulatory action date of May 29, 2023. This meeting signifies a shift from previous communications and focuses on evaluating the evidence that SRP-9001 can predict clinical benefits, essential for potential accelerated approval. CEO Doug Ingram expressed readiness to present comprehensive data supporting the therapy's transformative potential. A related conference call is scheduled for 4:30 p.m. ET today.