FDA Approves Revolution Medicines’ RASONQUE for Metastatic Pancreatic Cancer | iPharmaCenter
The U.S. Food and Drug Administration has approved RASONQUE, also known as daraxonrasib, as a once-daily oral treatment for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for multiagent chemotherapy. The decision marks the first approval of a broad RAS-targeted medicine for this disease. RASONQUE belongs to a new class of RAS(ON) multi-selective and mutant-selective inhibitors designed to suppre
21 minutes ago3 min read
FDA Approves Johnson and Johnson’s IMAAVY for Warm Autoimmune Hemolytic Anemia | iPharmaCenter
The U.S. Food and Drug Administration has approved IMAAVY, also known as nipocalimab, for adults and children aged 12 years and older with warm autoimmune hemolytic anemia who are currently receiving or have previously received corticosteroids. The decision gives patients with this rare and potentially life-threatening blood disorder the first treatment specifically approved for warm autoimmune hemolytic anemia. Johnson and Johnson said the approval followed the agency’s prio
1 day ago2 min read
Regeneron's Pasatru wins FDA nod as second FOP therapy, first to cut flare-ups and new bone lesions
The US Food and Drug Administration has cleared Regeneron's Pasatru (garetosmab) for adults with fibrodysplasia ossificans progressiva, marking the second approved treatment for this ultra-rare condition and the first to show meaningful reductions in both new heterotopic ossification lesions and clinician-assessed flare-ups. What changed for FOP patients? Fibrodysplasia ossificans progressiva is a rare genetic disease in which soft tissues such as muscle, tendon and ligament
Aug 203 min read
FDA Clears Ultragenyx Gene Therapy for Glycogen Storage Disease Type Ia
The U.S. Food and Drug Administration has granted accelerated approval to Genglycos, a one-time gene therapy developed by Ultragenyx Pharmaceutical for adults and children aged eight years and older with glycogen storage disease type Ia. The approval makes Genglycos the first FDA-authorized treatment for this rare inherited metabolic disorder. It is also designed to address the genetic cause of the disease rather than focusing only on managing its symptoms. What causes glycog
Aug 193 min read
FDA Grants Accelerated Approval to Zenbexus Combination for Relapsed Multiple Myeloma | iPharmaCenter
The US Food and Drug Administration has granted accelerated approval to Zenbexus, also known as iberdomide, in combination with daratumumab and hyaluronidase plus dexamethasone for adults with multiple myeloma previously treated with at least one regimen containing both a proteasome inhibitor and an immunomodulatory medicine. The approval is based on findings from the Phase 3 EXCALIBER-RRMM study, which evaluated the Zenbexus combination in patients with relapsed or refractor
Aug 142 min read