Sarepta Therapeutics excels in rare disease therapies using RNA, gene editing, and gene therapies, with significant revenue growth driven by ELEVIDYS gene therapy approval. Despite missing the primary endpoint, ELEVIDYS hit key secondary endpoints, leading to FDA approval for all DMD patients ages 4 and older. Now with a label that covers roughly 80% of the DMD population, Sarepta is positioned for explosive growth in the DMD market.
Following the label expansion, Sarepta's (SRPT) Elevidys is approved to treat all DMD patients aged four years and older, regardless of ambulation status.
Sarepta Therapeutics NASDAQ: SRPT is leading the game in precision genetic medicine and biotechnology. Sarepta Therapeutics' stock price has witnessed a surge of over 40% after receiving expanded FDA approval for its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys.
Sarepta Therapeutics Inc (NASDAQ:SRPT) shares soared almost 35% after the US Food and Drug Administration (FDA) approved the expanded use of its gene therapy Elevidys for patients with Duchenne muscular dystrophy (DMD). DMD is a rare genetic condition which causes weakness and the wasting away of the body's muscles, with symptoms including trouble walking and running, falling frequently, fatigue and learning disabilities or difficulties.
Sarepta Therapeutics (NASDAQ: SRPT ) stock jumped about 30% overnight after the Food and Drug Administration (FDA) approved one of its gene therapies for children with Duchenne muscular dystrophy (DMD). Peter Marks, director of the Center for Biologics Evaluation and Research, overruled three research teams and his top lieutenants in making the decision.
Sarepta Therapeutics (SRPT) shares soared in premarket trading Friday, a day after the Food and Drug Administration (FDA) allowed expanded use of the biotech's treatment for a rare muscle disease in children, which especially affects boys.
Sarepeta Therapeutics Inc.'s stock soared 38% in premarket trade Friday, after the biotech said the U.S. regulator has approved an expanded indication of its treatment for the rare muscle disorder Duchenne muscular dystrophy.
The biopharmaceutical company says the drug was approved for ambulatory and non-ambulatory people, who are at least 4 years old, and with a confirmed mutation in the DMD gene.
Thursday, the FDA approved labeled indication for Sarepta Therapeutics Inc's SRPT Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.
Sarepta Therapeutics shares surged about 36% in premarket trading on Friday as an expanded use approval opened up a bigger market in the U.S. for its gene therapy for patients with Duchenne muscular dystrophy (DMD).
Sarepta Therapeutics (NASDAQ: SRPT), a pharmaceutical company that describes itself as specializing in “precision genetic medicine for rare diseases”, saw its stock price shoot up by 36% in after-hours trading last night. But why? Ground-breaking medical breakthrough Toward the end of the trading day yesterday, Sarepta announced that it had obtained approval from the U.S.
Sarepta rocketed late Thursday after the FDA approved its Duchenne muscular dystrophy gene therapy for all patients age 4 and older.