Targeted Oncology6d
FDA Grants Orphan Drug Designation to PEP-010 in Pancreatic Cancer
The FDA granted an orphan drug designation to PEP-010 for the treatment of patients with metastatic pancreatic cancer.
ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA ...
CURE10d
Bexobrutideg Receives Orphan Drug Designation in Waldenström Macroglobulinemia
Bexobrutideg, a bioavailable agent, was granted orphan drug designation by the FDA for Waldenström macroglobulinemia, a type ...
Palatin's Oral MC4R Agonist PL7737 Receives FDA Orphan Drug Designation for Obesity Due to Leptin Receptor Deficiency
Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing first-in-class medicines based on ...
Marvel to File for Orphan Drug Designation with The U.S. FDA for MB-204 as a Treatment for Rett Syndrome
Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc.
Targeted Oncology8d
FDA Grants Orphan Drug Designation to HLX22 for Gastric Cancer Treatment
The FDA granted orphan drug designation to HLX22 for gastric cancer, advancing global phase 3 trials to improve first-line ...
Korro Bio gets Orphan Drug Designation from FDA for its genetic disorder treatment
Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...
Business Insider11d
Nurix Announces U.S. FDA Orphan Drug Designation Granted to Bexobrutideg (NX-5948) for the Treatment of Waldenström Macroglobulinemia
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström Macroglobulinemia First-in-class Bruton’s tyrosine kinase (BTK) degrader NX-5948 ...
PEP-Therapy receives FDA Orphan Drug Designation for PEP-010 for treatment of Pancreatic Cancer
Therapy receives FDA Orphan Drug Designation for PEP-010for treatment of Pancreatic CancerParis (France), March 17, 2025 – ...
Rhythm Pharmaceuticals Receives Orphan-Drug Designation in Japan for Obesity Treatment
Rhythm Pharmaceuticals received an orphan-drug designation from Japan's Ministry of Health, Labour and Welfare for its treatment for a certain type of obesity.
Palatin Technologies, Inc.: Palatin's Oral MC4R Agonist PL7737 Receives FDA Orphan Drug Designation for Obesity Due to Leptin Receptor Deficiency
Initiated IND-enabling toxicology program IND submission planned for 4Q25; Clinical data expected in 1H26 CRANBURY, N.J., March 25, 2025 /PRNewswire/ -- Palatin ...