AstraZeneca Reports the CHMP Positive Opinion for Klygefa to Treat Generalised Myasthenia Gravis

Shots: The CHMP has recommended Klygefa(gefurulimab; SC, QW) as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adults who are AChR antibody-positive; filings are under review in the US, China & other countries Opinion was supported by the P-III (PREVAIL) trial assessing Klygefavs PBO in 260 gMG adults for…

ByRidhi RastogiSeptember 21, 2026

Teva Reports the US FDA’s NDA Submission for Ecopipam to Treat Pediatric Tourette Syndrome

Shots: The US FDA has received an NDA for ecopipam, a selective dopamine D1 receptor antagonist, for the treatment of pediatric Tourette syndrome NDA was supported by the P-III trial data showing that ecopipam significantly delayed time to relapse vs PBO in pediatric Tourette syndrome pts who responded to treatment during the open-label treatment period;…

ByRidhi RastogiJune 19, 2026

Sarepta Therapeutics Reports the MHLW’s Conditional & Time-Limited Approval of Elevidys for Duchenne Muscular Dystrophy (DMD)

Shots: Japan's MHLW has granted conditional & time-limited approval to Elevidys for DMD pts (3–8yrs.) with no exon 8/9 deletions in the DMD gene & who are negative for anti-AAVrh74 Abs Approval was based on extensive clinical data, incl. 2yr. global P-III (EMBARK) trial showing motor function benefits vs external controls; Part 2 data was…

ByRidhi RastogiMay 14, 2025

AstraZeneca Reports the CHMP Positive Opinion for Klygefa to Treat Generalised Myasthenia Gravis

Teva Reports the US FDA’s NDA Submission for Ecopipam to Treat Pediatric Tourette Syndrome

Sarepta Therapeutics Reports the MHLW’s Conditional & Time-Limited Approval of Elevidys for Duchenne Muscular Dystrophy (DMD)

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