FDA Designation: THE-630 to Treat Gastrointestinal Tumors
Theseus Pharmaceuticals announced the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for THE-630 to treat patients with advanced gastrointestinal stromal tumors (GIST). GIST is the most common sarcoma of the gastrointestinal tract, and...
Azafaros: ODD for AZ-3102 to Treat GM2 Gangliosidosis
Azafaros has announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102 to treat GM2 gangliosidosis, including Tay-Sachs disease and Sandhoff disease. GM2 gangliosidosis, a group of rare genetic disorders that...
FDA Approval: Prophylactic Treatment for Type 3 VWD
The U.S. Food and Drug Administration (FDA) has approved Takeda Pharmaceutical’s VONVENDI to reduce the frequency of bleeding episodes in patients with severe Type 3 von Willebrand disease (VWD). VWD is the most common inherited bleeding disorder that is caused by a...
ALX Oncology: Evorpacept Receives ODD to Treat GC
The U.S. Food and Drug Administration (FDA) has granted an orphan drug designation (ODD) for ALX Oncology’s evorpacept, a next-generation CD47 blocker, to treat patients with gastric cancer and gastroesophageal junction cancer. Gastric cancer and gastroesophageal...
Immunocore: FDA Clearance of Uveal Melanoma Drug KIMMTRAK
The U.S. Food and Drug Administration (FDA) has granted approval of Immunocore’s KIMMTRAK (tebentafusp-tebn) to treat adults who are HLA-A*02:01-positive for unresectable or metastatic uveal melanoma (mUM). Uveal melanoma is a rare and aggressive cancer of the eye, in...
Imara IND Clearance: Tovinontrine (IMR-687) to Treat HFpEF
The U.S. Food and Drug Administration (FDA) has cleared Imara’s Investigational New Drug (IND) application for tovinontrine (IMR-687) to treat heart failure with preserved ejection faction (HFpEF). HFpEF (diastolic heart failure) is a form of heart failure that occurs...