REGENXBIO Announces Positive Topline Results From Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202

Regenxbio's Duchenne gene therapy clears phase 3 hurdle

Regenxbio is pitching at FDA approval in 2027 for its Duchenne muscular dystrophy gene therapy RGX-202, a potential rival to Sarepta/Roche's Elevidys.

Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission

The company hopes to prove that its treatment is more effective, and safer, than Sarepta Therapeutics’ Elevidys.

REGENXBIO Announces Positive Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202

/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III...

With new data, Regenxbio to seek FDA approval of Duchenne gene therapy

Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA leadership will have a “mandate on rare disease flexibility.”

REGENXBIO to push for accelerated approval of DMD gene therapy

REGENXBIO touted data from the Phase III portion of a study that showed higher benefit that Elevidys.The post REGENXBIO to push for accelerated approval of DMD gene therapy appeared first on Clinical Trials Arena.

REGENXBIO Stock Plunges as Duchenne Gene Therapy Win Runs Into Safety Questions

REGENXBIO shares fell 37% to $6.31 after its Duchenne muscular dystrophy gene therapy, RGX-202, met the main goal in a late-stage trial. Investors reacted to two serious adverse events and uncertainty over FDA approval. The company reported 93% of patients showed microdystrophin expression at 12 weeks. REGENXBIO said it will seek accelerated approval and targets a 2027 launch. The post REGENXBIO Stock Plunges as Duchenne Gene Therapy Win Runs In…