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Vega Therapeutics Receives FDA Orphan Drug Designation for VGA039 for the Treatment of von Willebrand Disease

Vega Therapeutics, Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, announced that the U.S. Food and Drug Administration (FDA)...

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Rocket Pharmaceuticals, Inc,  a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need,...

FDA Approves AYVAKIT as the First and Only Treatment for Indolent Systemic Mastocytosis

Blueprint Medicines Corporation announced that the U.S. Food and Dru Administration (FDA) has approved AYVAKIT (avapritinib) for the treatment of adults with indolent systemic mastocytosis (ISM)....

EPKINLY Approved by U.S. FDA as the First and Only Bispecific Antibody to Treat Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma

AbbVie announced that the U.S. Food and Drug Administration (FDA) has approved EPKINLY, as the first and only T-cell engaging bispecific antibody for the...

FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa

The U.S. Food and Drug Administration approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients...
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