Regulatory Ultragenyx wins FDA approval for first treatment targeting root cause of GSDIa pharminent August 20, 2026
Regulatory Regeneron wins FDA approval to challenge Ipsen for rare bone disease market pharminent August 20, 2026
Regulatory FDA grants accelerated approval to Ultragenyx’s Genglycos gene therapy pharminent August 20, 2026
M&A / Deals BioMarin spends up to $490m for Alesta’s bone disease candidate pharminent August 19, 2026
M&A / Deals Sentynl bets up to $475M in licensing deal for Mereo’s rare lung disease asset pharminent August 12, 2026
Pipeline Epicrispr lands $90M to advance epigenetic editing drug for rare muscle disease pharminent August 11, 2026
Pipeline BioMarin axes asset from $270M Inozyme takeover, ending run in rare metabolic indication pharminent August 10, 2026
Clinical Data Intellia finds genetic suspect for liver safety signals with ATTR gene therapy pharminent August 7, 2026
Regulatory Novartis receives European Commission approval for Itvisma for spinal muscular atrophy pharminent July 2, 2026
M&A / Deals Ipsen to buy Swiss biotech Memo Therapeutics in potential $800M deal pharminent July 1, 2026
Regulatory Sarepta secures early 2027 FDA decision for Duchenne drugs, pushing past confirmatory fail pharminent July 1, 2026
Clinical Data BridgeBio’s achondroplasia pill shows ‘compelling’ growth and body proportionality benefit pharminent June 29, 2026
M&A / Deals Ionis grants Recordati rights to zilganersen for Alexander disease pharminent June 26, 2026
Regulatory FDA’s uniQure, REGENXBIO reversals could bolster other near-term rare disease applications pharminent June 25, 2026
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
