Novartis scores FDA nod for game-changing SMA treatment

Itvisma is a one-time, fixed-dose therapy designed to tackle the genetic root cause of SMA

FDA slaps gene therapy Elevidys with boxed warning after fatal liver failures

Elevidys came under scrutiny in June when the FDA disclosed two fatal cases in non-ambulatory boys who developed dangerously high liver enzyme levels

Rafa Laboratories lands $186 million BARDA contract to develop life-saving trauma drug

Landmark studies have shown that rapid TXA administration can significantly reduce mortality

Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions

New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris

Novartis to acquire Avidity Biosciences for $12 billion

The acquisition will follow the planned separation of Avidity’s early-stage precision cardiology programs into a new, independent company

Moderna’s mRNA-4359 shows promising early results in resistant Melanoma at ESMO 2025

mRNA-4359 advances to phase 2 following encouraging results in checkpoint inhibitor-resistant Melanoma

FDA approves Zydus’ Deflazacort oral suspension, 22.75 mg/mL

Deflazacort oral suspension is indicated for treating Duchenne Muscular Dystrophy in patients 5 years of age and older

DINAMIQS inaugurates new cGMP viral vector manufacturing facility in Switzerland

The new site enables end-to-end production of viral vector gene therapies

Sarepta refuses to comply with FDA request to stop shipping gene therapy Elevidys

FDA requests Sarepta Therapeutics to suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths

Teijin Pharma receives approval for additional indication for XEOMIN in Japan