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
M&A / Deals Neurocrine Nabs Rare Obesity Disorder Drug in ‘Surprising’ $2.9B Soleno Deal pharminent April 6, 2026
Policy / Pricing Rare Disease Leaders Call for Regulatory Clarity as FDA Balances Urgency With Rigor pharminent April 2, 2026
M&A / Deals Biopharma M&A Heats Up, Rare Diseases Win Three Approvals, Wave Crashes pharminent April 1, 2026
Clinical Data PepGen Loses Half of Share Price on Mixed Data in Rare Muscle-Wasting Disease pharminent March 31, 2026
Regulatory Biogen Bounces Back With FDA Nod for High-Dose Spinal Muscular Atrophy Drug pharminent March 30, 2026
Regulatory FDA clears Denali drug in ‘clear step’ for rare disease biotechs pharminent March 26, 2026
