FDA Approves Takeda’s MIMRYLO for Polycythemia Vera | iPharmaCenter
The U.S. Food and Drug Administration has approved MIMRYLO, also known as rusfertide, for the treatment of erythrocytosis in adults with polycythemia vera, a rare and chronic blood cancer. The decision introduces the first hepcidin mimetic medicine authorized for this condition, offering patients a new mechanism of action beyond the therapies currently available. A Long-Standing Challenge in PV Management Polycythemia vera affects an estimated 90,000 people in the United Stat
2 days ago3 min read
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
5 days 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
6 days 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