Ionis Wins First Ever FDA Approval for an Alexander Disease Treatment, Marking a Turning Point for a Disease With No Prior Options
Regulators in the United States have given the green light to a new spinal injection therapy built to slow one of the rarest and most devastating disorders of the nervous system, handing patients and families their first real treatment option after decades with none. The Food and Drug Administration cleared Zanvastro, known by its scientific name zilganersen, for use in both children and adults living with Alexander disease, a genetic condition so uncommon that it touches rou
10 minutes ago3 min read
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
5 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
Aug 273 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
Aug 262 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