FDA Grant

Ikena Oncology, Inc. (NASDAQ: IKNA) received Fast Track designation from the U.S. Food and Drug Administration (FDA) for IK-930, in patients with unresectable NF2-deficient malignant pleural mesothelioma (MPM). IK-930 is currently being investigated in a Phase 1 clinical trial for patients with...

Paratek Pharmaceuticals, Inc. (NASDAQ: PRTK) received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its lead drug NUZYRA (omadacycline) for the treatment of pulmonary Nontuberculous Mycobacterial (NTM) disease caused by both Mycobacterium avium complex (...

Regulus Therapeutics Inc. (NASDAQ: RGLS) received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its lead drug RGLS8429 in the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). RGLS8429 is being investigated in a Phase 1 single-ascending dose...

The United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Kazia Therapeutics’ (NASDAQ: KZIA) Paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer. Paxalisib is being investigated in a...

The United States Food and Drug Administration (FDA) has granted Fast Track Designation to Kintara Therapeutics’ (NASDAQ: KTRA) lead drug candidate VAL-083 for the treatment of patients with newly-diagnosed unmethylated glioblastoma (GBM). Kintara is currently advancing VAL-083 AGILE...

Eledon Pharmaceuticals (NASDAQ: ELDN) received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its drug candidate Tegoprubart for the Prevention of Allograft Rejection in Pancreatic Islet Cell Transplantation. Tegoprubart is an anti-CD40L antibody with high affinity for...

Vertex Pharmaceuticals (NASDAQ: VRTX) received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its drug candidate inaxaplin (VX-147) for the treatment of patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS). The FDA grant was supported by the...

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to BioCryst Pharmaceuticals's (NASDAQ: BCRX) lead drug candidate BCX9250 for the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP). Fibrodysplasia ossificans...

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PDS Biotechnology's (NASDAQ: PDSB) lead drug candidate PDS0101 in combination with KEYTRUDA (pembrolizumab) for the treatment of patients with recurrent or metastatic HPV16-positive head and neck cancer....

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to Midatech Pharma PLC's (NASDAQ: MTP) lead drug candidate MTX110 for the treatment of patients with recurrent glioblastoma (rGBM). The company is planning to begin recruitment in the phase 1 study evaluating MTX110...

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Kymera Therapeutics (NASDAQ: KYMR) lead drug candidate KT-333 for the treatment of patients with Peripheral T-cell Lymphoma (PTCL). The company said that KT-333 is a first-in-class degrader of the...