News

Shuttle Pharma FDA Orphan-Drug Nears Patient Enrollment Milestone for Phase 2 Clinical Trial
Shuttle Pharmaceuticals Holdings, Inc. (Nasdaq: SHPH) (“Shuttle Pharma” or the “Company”), a discovery and development stage ...
Rare Disease Industry Awaits FDA Chief’s New Drug Pathway Plan
FDA Commissioner Marty Makary’s proposal to roll out a new drug approval pathway is heightening hope for the rare disease ...
The Manila Times21h
FDA Provides Seven Years of Orphan-Drug Exclusive Approval for Ryoncil®
Mesoblast's RYONCIL ® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA approved mesenchymal ...
Investor's Business Daily on MSN6h
Biohaven Plunges On A Surprise FDA Twist For Its Rare-Disease Drug
Biohaven shares plunged Thursday after the Food and Drug Administration unexpectedly delayed the review date for its rare-disease drug.
US FDA Orphan Drug Rare Disease Market Clinical Trials Drug Sales Insight 2030
Kuick Research Report Gives Detailed Insight On More Than 1400 FDA Orphan Designated Drugs And Highlights More Than USD 190 Billion Opportunity</p ...
Targeted Oncology3d
THE001, Novel Sarcoma Treatment, Earns FDA Orphan Drug Designation
THE001, a thermosensitive doxorubicin liposome for soft tissue sarcomas, received FDA orphan drug designation, offering ...
Pharmabiz16h
US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II
US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II: Rockville, Maryland Thursday, May 15, 2025, 1 ...
The Pharma Letter1d
Kaerus wins two FDA designations for KER-0193 in Fragile X syndrome
UK-based Kaerus Bioscience, a biopharma created by European investment firm Medicxi for the development of therapeutics for ...
Business Wire1d
Cure Rare Disease Receives Orphan Drug Designation from FDA for Investigational Therapy Targeting Spinocerebellar Ataxia Type 3 (SCA3)
“Receiving Orphan Drug Designation ... exemption from certain FDA application fees, and eligibility for seven years of marketing exclusivity if the drug is approved. Cure Rare Disease’s ...
Pharmabiz3d
Saudi FDA grants orphan drug designation to IASO Bio’s BCMA-targeted CAR-T cell therapy product, Equecabtagene Autoleucel
Saudi FDA grants orphan drug designation to IASO Bio’s BCMA-targeted CAR-T cell therapy product, Equecabtagene Autoleucel: Shanghai, Nanjing Monday, May 12, 2025, 16:00 Hrs [IST ...