Granted Both Orphan Drug & Fast Track designations for Amezalpat (TPST-1120) for the treatment of patients with Hepatocellular Carcinoma (HCC)• Announced Agreement with Roche to Support Advancement ...

The FDA granted an orphan drug designation to PEP-010 for the treatment of patients with metastatic pancreatic cancer.

ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA ...

Received a "Study May Proceed” letter from the FDA to evaluate amezalpat in combination with atezolizumab (TECENTRIQ®) and ...

Bexobrutideg, a bioavailable agent, was granted orphan drug designation by the FDA for Waldenström macroglobulinemia, a type ...

Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030. The 2025 ...

The FDA granted orphan drug designation to HLX22 for gastric cancer, advancing global phase 3 trials to improve first-line ...

Soleno's shares soar in the premarket hours following the FDA approval of Vykat XR (diazoxide choline) for treating ...

Initiated IND-enabling toxicology program IND submission planned for 4Q25; Clinical data expected in 1H26 CRANBURY, N.J., March 25, 2025 /PRNewswire/ -- Palatin ...

The FDA's Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or ...

Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...