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.
The U.S. Food and Drug Administration has approved Aqneursa (levacetylleucine) to treat ataxia in adults and children with ataxia-telangiectasia weighing at least 15 kg (about 33 pounds). Aqneursa was previously approved in 2024 to treat the neurological symptoms of Niemann-Pick disease type C, another rare neurological condition.
On September 18, 2026, the Food and Drug Administration approved imlunestrant (Inluriyo) in combination with abemaciclib (Verzenio), for adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine …
On October 1, 2026, FDA will begin accepting requests from drug and biological product sponsors to participate in the fifth and final year of the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program.
The U.S. Food and Drug Administration (FDA) issued a draft strategy document “BsUFA III Future Needs for the Development of Interchangeable Products” BsUFA III Future Needs for the Development of Interchangeable Products” based on the scientific workshop held on September 19, 2025, and comments submitted to the public docket following the workshop.
