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.
The Food and Drug Administration has approved an additional warning in labeling for Kybella (deoxycholic acid) injection regarding adverse reactions associated with use of the drug outside of the submental fat region (fat under the chin, known as a “double chin”) and recommends against this unapproved use. The agency approved this revised labeling after its …
The U.S. Food and Drug Administration toThe U.S. Food and Drug Administration today announced the final design of the Expedited Investigational New Drug (IND) Pilot. Currently, first-in-human clinical trials may take up to two years to complete in the United States. The same types of trials are completed much faster in China and Australia, threatening America’s …
FDA Launches Expedited IND Pilot, Begins Accepting Applications Read More »
The U.S. Food and Drug Administration today approved Isembyld (apitegromab-mstn) injection to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving an SMN2-targeted treatment. Isembyld is the first therapy approved for SMA that directly targets muscle loss, working alongside existing treatments to address a …
FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy Read More »
