Rare Diseases
🇺🇸 FDA Page 4FDA news and analysis for pharmaceutical BD, investment, and market access · 12 articles on this page
Gain critical insights into global orphan drug development & rare disease market trends. Stay ahead in pharma R&D, investment, and regulatory strategy.
Showing 37–48 of 81 articles
Regeneron Announces Free Gene Therapy Otarmeni for Rare Hearing Loss in U.S. Government Agreement
Adagrasib Phase 3 Trial Results: Key Insights on KRAS G12C NSCLC
Trevi Therapeutics Schedules Q1 2026 Earnings Call for Haduvio Chronic Cough Treatment Updates
Soligenix HyBryte Phase 3 FLASH2 Trial Shows Interim Results for Cutaneous T-Cell Lymphoma Treatment
BRC Therapeutics Receives FDA Clearance for BRC-002 Phase I Trial in Complex Regional Pain Syndrome
Vanda Pharmaceuticals' Imsidolimab Shows Efficacy for Generalized Pustular Psoriasis in NEJM Evidence Study
FDA Biosimilar Approval Pathway: Key Regulatory Changes & Market Impact 2026
Cellenkos Receives FDA Clearance for CK0801 Phase 2 Trial in Aplastic Anemia
AstraZeneca's SAPHNELO Autoinjector Approved by FDA for Self-Administration in Lupus Treatment
Intellia Therapeutics Submits FDA Application for Lonvo-Z, First CRISPR Gene Therapy for Hereditary Angioedema
Intellia Therapeutics Achieves First-Ever Positive Phase 3 Results for In Vivo Gene Editing with Lonvoguran Ziclumeran in Hereditary Angioedema
OSE Immunotherapeutics' Pegrizeprument (VEL-101) Receives FDA Orphan Drug Designation for Heart Transplant Rejection Prevention