Drugs
FDA has issued Saol Therapeutics a complete response letter (CRL) on its NDA for SL1009 (sodium dichloroacetate oral solution) for treating an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). There are about 1,000 children in the U.S. with PDCD, a rare and life-threatening genetic disorder
Drugs
FDA has launched a new “green list” import alert aimed at preventing unapproved and potentially unsafe GLP-1 drug ingredients from entering the U.S. The move comes as demand surges for drugs such as semaglutide and tirzepatide, which are FDA-approved for weight management and Type 2 diabetes. The
Drugs
FDA has granted CERo Therapeutics fast track status for its lead investigational compound, CER-1236, for treating acute myeloid leukemia. A first-in-human Phase 1/1b study, currently underway, is designed to evaluate the safety and preliminary efficacy of CER-1236 in patients with acute myeloid leukemia that is
Drugs
FDA’s recent use of ad hoc “expert panels” in place of traditional advisory committees risks undermining scientific rigor and public trust, according to an online opinion piece in STAT by Johns Hopkins Bloomberg School of Public Health’s drug safety expert G. Caleb Alexander. In recent months, the agency
Drugs
FDA says an evaluation of reports contained in its vaccines’ adverse event database shows there have been Covid-19 vaccine-related deaths in children and a “proper investigation” is underway that will generate a releasable report in the coming weeks. In a 9/4 appearance on The Lead with Jake
Drugs
BioNTech and Duality Biologics say their experimental antibody-drug conjugate, trastuzumab pamirtecan (BNT323/DB-1303), met its primary endpoint in a pivotal Phase 3 trial for patients with HER2-positive metastatic or unresectable breast cancer. The China-based study compared trastuzumab pamirtecan to the approved therapy trastuzumab emtansine (T-DM1)
Drugs
FDA has extended by three months its review of Agios Pharmaceuticals’ supplemental NDA seeking to expand the use of Pyrukynd (mitapivat) to adults with thalassemia. The extension, which was triggered by a Risk Evaluation and Mitigation Strategy (REMS) proposal that the company recently submitted, pushes back the user fee review
Drugs
FDA has granted breakthrough therapy designation to Lilly’s olomorasib in combination with the anti-PD-1 therapy Keytruda (pembrolizumab) for the first-line treatment of patients with unresectable advanced or metastatic non-small cell lung cancer with a KRAS G12C mutation and PD-L1 expression at or under 50%
Drugs
FDA has released the form FDA-483 issued following a 7/14-7/19 inspection at GenoGenix, a Boca Raton, FL, outsourcing facility. The 14 inspection observations were: * failure to include validation of the process in procedures designed to prevent microbiological contamination of drug products purporting to be sterile; * failure
Drugs
Sanofi says its investigational antibody amlitelimab met all primary and key secondary endpoints in a late-stage trial of patients with moderate-to-severe atopic dermatitis, reinforcing its potential as a first-in-class therapy with dosing as infrequent as four times a year. In the global COAST 1 Phase
Drugs
FDA has said it does not plan to convene an advisory committee to discuss the Aquestive Therapeutics NDA for its Anaphylm (dibutepinephrine) sublingual film to treat severe allergic reactions. A company statement says the PDUFA action date for the NDA remains 1/31/2026. The company says Anaphylm is a
Drugs
FDA says that going forward, complete response letters (CRLs) will be released promptly after they are issued to sponsors. In an announcement, the agency also said it had released 89 previously unpublished CRLs issued from 2024 to the present, associated with pending or withdrawn applications. “This is a milestone day
Drugs
FDA has posted a complex innovative trial case study that evaluated the benefit and risk of a higher dose than previously evaluated in a clinical trial of a drug to treat alopecia areata. The case study says the agency considered these trial design features innovative: * using external and synthetic placebo
Drugs
A 3/10-3/19 FDA inspection at the Amneal Pharmaceuticals drug manufacturing facility in Gujarat, India, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/27 Warning Letter says the specific violations were: * failure to thoroughly investigate any unexplained discrepancy or failure of
Drugs
FDA has granted Boehringer Ingelheim a breakthrough therapy designation for Hernexeos (zongertinib) tablets for the first-line treatment of adults with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) mutations. The designation is supported by data from the ongoing Beamion-LUNG 1 trial, which
Drugs
FDA has launched its Rare Disease Evidence Principles (RDEP) process to “provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient populations with significant unmet medical need and that are driven by a known genetic defect.” An agency statement says that
Drugs
FDA has accepted for review an Achieve Life Sciences’ NDA for cytisinicline, an investigational treatment for nicotine dependence. The submission has been assigned a user fee review action target date of 6/20/2026. The NDA is based on data from more than 2,000 clinical trial participants enrolled in
Drugs
Drug developers are using artificial intelligence (AI) technologies for discovery and safety testing to get faster and cheaper results and help reduce the dependence on animal testing. That’s the view of several experts interviewed by Reuters for a story on AI-driven drug development. “Within the next three to
Drugs
A 2/24-3/5 FDA inspection at Chromatography Institute of America, doing business as Compounder’s International Analytical Laboratory in Castle Rock, CO, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs and significant deviations from CGMP for active pharmaceutical ingredients. An 8/20 Warning
Drugs
FDA has published its 2025 annual forecast for planned OTC drug monograph activities. The non-binding list shows planned monograph activities the agency intends to initiate over the next three years. Topics of planned monograph activities listed in the 2025 forecast are: * risks associated with codeine-containing cough medicine; * pediatric
Drugs
Merck’s investigational oral PCSK9 inhibitor enclicitide decanonate met all its primary and secondary endpoints in the Phase 3 CORALreef Lipids trial evaluating the drug to treat adults with hypercholesterolemia on a moderate or high-intensity statin. A company statement says treatment with enlicitide resulted in statistically significant and clinically
Drugs
FDA has released the form FDA-483 with four observations from a 7/29-8/8 inspection at AnazaoHealth, a Las Vegas, NV, outsourcing facility. The four inspection observations were: * failure to establish adequate written procedures for production and process controls designed to assure that drug products have the identity,