The U.S. Food and Drug Administration (FDA) approved Camzyos (mavacamten) capsules as the first treatment for children with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Camzyos was first approved for adults in 2022 and is now approved for both adults and pediatric patients weighing at least 30 kg (approximately 66 lbs) …
FDA has approved Gazyva (obinutuzumab) injection to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or steroid-dependent, cFDA has approved Gazyva (obinutuzumab) injection to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or …
FDA Approves Drug to Treat Idiopathic Nephrotic Syndrome in Patients 2 Years and Older Read More »
The U.S. Food and Drug Administration (FDA) has approved Pasatru (garetosmab-grts) to reduce new heterotopic ossification (bone formation outside the skeleton) and reduce clinician-assessed disease flare-ups in adults with fibrodysplasia ossificans progressiva (FOP). Pasatru is the second drug approved for patients with this very rare disease.
On September 24, 2026, the Food and Drug Administration approved belzutifan (Welireg, Merck & Co., Inc.) in combination with lenvatinib (Lenvima, Eisai Inc.) for adults with advanced renal cell carcinoma with a clear cell component (ccRCC) following a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
On September 23, 2026, the Food and Drug Administration approved lirafugratinib (Lyrfigtu, Elevar Therapeutics, Inc.), a kinase inhibitor, for the treatment of adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement.
The Committee recommended restricting and better defining the approved uses of the medicines. The medicines can now only be used to relieve symptoms of specific conditions leading to nerve damage in one or more nerves (mononeuropathies and polyneuropathies), but no longer for neuritis, polyneuritis, polyradiculoneuropathy and myasthenic syndrome. They can also be used for the symptomatic …
EMA concludes review of ipidacrine-containing medicines Read More »
EMA’s human medicines committee (CHMP) has concluded that the benefits of sodium oxybate syrup and oral solution (175 mg/ml) continue to outweigh their risks when used to treat acute alcohol withdrawal syndrome and to support long‑term abstinence in people with alcohol dependency. However, due to limitations and uncertainties in the data, additional studies are required …
The Food and Drug Administration’s Innovative Science and Technology Approaches for New Drugs (ISTAND) program has accepted a letter of intent (LOI) for a wearable sensor-derived digital outcome measure intended to quantify foot drop severity and its impact on gait in adult patients with Charcot-Marie-Tooth disease type 1A (CMT1A), a progressive, degenerative disease. This LOI …
FDA’s Rare Disease Endpoint Advancement (RDEA) Pilot Program is accepting proposals for this quarter until September 30, 2026.
