Gene Therapy UniQure to file gene therapy for approval, reflecting major shifts at FDA pharminent June 17, 2026
Clinical Data Rhythm’s obesity drug scores ‘better than expected’ weight loss in rare genetic disease pharminent June 15, 2026
Clinical Data Novartis’ $12B Avidity buy pays dividends with Phase 1/2 muscular dystrophy win pharminent June 12, 2026
M&A / Deals Beren pockets $300m to take Niemann-Pick disease therapy to market pharminent June 11, 2026
Regulatory Disc’s FDA meeting sets ‘clear path’ for embattled rare blood disease drug pharminent June 10, 2026
Clinical Data Otsuka energizes IgA neuropathy space with new Phase 3 Voyxact data pharminent June 5, 2026
M&A / Deals Travere beefs up rare kidney disease portfolio with up to $1.1B China deal pharminent June 3, 2026
M&A / Deals Servier to acquire Edgewise’s muscular dystrophy business for $2.65bn pharminent June 2, 2026
Clinical Data BioMarin bounces back as Voxzogo performs ‘bigger than expected’ in new indication pharminent May 21, 2026
Clinical Data Beam one-ups Wave as both show promise of genetic editing for AATD pharminent May 20, 2026
Clinical Data Relay doubles the bar, outpacing Novartis with a 60% response in rare disease pharminent May 19, 2026
Clinical Data Relay drug shows early promise against rare blood vessel diseases pharminent May 19, 2026
Clinical Data BioMarin suffers another blow to rare disease portfolio in Phase 3 flop pharminent May 19, 2026
Other 225: An Interview With Craig Martin, Founder and CEO of the Orphan Therapeutics Accelerator pharminent May 18, 2026
Commercial Pfizer deals extend patent life for a top-selling rare disease drug pharminent April 28, 2026
Regulatory Intellia Initiates Rolling BLA Submission for In Vivo CRISPR Therapy in Hereditary Angioedema pharminent April 28, 2026
Clinical Data Ionis’ antisense drug stabilizes ultra-rare disease ahead of FDA decision pharminent April 22, 2026
Regulatory FDA seeks permanent future for rare pediatric priority review vouchers pharminent April 9, 2026
