Rare Disease

• M&A / Deals

Neurocrine to acquire Prader-Will drug in $2.9B Soleno buyout

pharminent April 6, 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

• Regulatory

FDA approves new high dose Spinraza regimen for SMA

pharminent April 1, 2026

• M&A / Deals

Biopharma M&A Heats Up, Rare Diseases Win Three Approvals, Wave Crashes

pharminent April 1, 2026

• M&A / Deals

Biogen uplifts rare disease pipeline with $5.6bn Apellis buyout

pharminent April 1, 2026

• Clinical Data

PepGen Loses Half of Share Price on Mixed Data in Rare Muscle-Wasting Disease

pharminent March 31, 2026

• Market Access

UCB brings first therapy for rare disease TK2d to EU

pharminent March 31, 2026

• Regulatory

AZ considers filings for long-acting Strensiq follow-up

pharminent March 31, 2026

• Regulatory

Biogen Bounces Back With FDA Nod for High-Dose Spinal Muscular Atrophy Drug

pharminent March 30, 2026

• Commercial

The Rare Journey Needs a Special Pharmacy Partner

pharminent March 27, 2026

• Regulatory

FDA clears Denali drug in ‘clear step’ for rare disease biotechs

pharminent March 26, 2026

• Clinical Data

Maze tumbles despite positive data for kidney disease drug

pharminent March 25, 2026

• Clinical Data

Beam posts positive data on base editing treatment for AATD

pharminent March 25, 2026

• Clinical Data

Sarepta sees early success with RNAi drugs from Arrowhead

pharminent March 25, 2026