Drugs
FDA has extended by three months its review of a Cytokinetics NDA for aficamten for treating patients with obstructive hypertrophic cardiomyopathy (oHCM). The agency said the review extension is required to conduct a full review of the company’s proposed Risk Evaluation and Mitigation Strategy (REMS), according to the company.
Drugs
FDA has approved a Satsuma Pharmaceuticals 505(b)(2) NDA for Atzumi (dihydroergotamine (DHE)) nasal powder for the acute treatment of migraine with or without aura in adults. The NDA was resubmitted 11/2024 after addressing chemistry, manufacturing, and controls considerations cited in a 1/2024 complete response letter, according
Drugs
Reps. Alexandria Ocasio-Cortez (D-NY) and Nydia Velázquez (D-NY) are calling on HHS secretary Robert F. Kennedy, Jr., to reverse the reduction in force that forced the FDA San Juan, Puerto Rico, medical products laboratory to close. In a statement (contains a link to their letter to Kennedy)
Drugs
Immunic says “positive” data from the Phase 2 CALLIPER trial of nuclear receptor related 1 (Nurr1) activator vidofludimus calcium (IMU-838) in patients with progressive multiple sclerosis (PMS) showed the therapy reduced the relative risk of 24-week confirmed disability worsening (24wCDW) events by 20% compared to placebo. “Further analyses
Drugs
Gilead Sciences has agreed to pay $202 million to settle a U.S. government civil fraud lawsuit and resolve claims that the company offered and paid kickbacks (e.g., honoraria payments, meals, and travel expenses) to healthcare practitioners who spoke at or attended Gilead speaker events to induce them to
Drugs
FDA has approved a Johnson & Johnson BLA for Imaavy (nipocalimab-aahu), a human FcRn-blocking monoclonal antibody for treating generalized myasthenia gravis. The company describes the therapy as an immunoselective therapy designed to “substantially reduce immunoglobulin G (IgG), including harmful IgG autoantibodies, without additional detectable effects on other adaptive
Drugs
Boehringer Ingelheim has released new findings from its Beamion LUNG-1 trial, highlighting the potential of zongertinib as a breakthrough oral therapy for patients with advanced non-small cell lung cancer (NSCLC) harboring HER2 mutations. The trial focused on patients with advanced NSCLC who had previously received treatment and whose
Biologics
FDA commissioner Martin Makary says there will be no agency-wide reorganization. Interviewed by Jeremy Faust for an Inside Medicine Substack post, Makary said some staff asked for a reorganization and he rejected the proposal. “We are consolidating travel office, information technology, and some other things that make sense for
Drugs
Senator Josh Hawley (R-Mo.) has sent a 4/28 letter to FDA commissioner Marty Makary asking that FDA review a new study allegedly revealing greater safety risks than the agency currently acknowledges for the abortion drug mifepristone. Hawley questioned Makary’s recent remarks about the agency having “no plans
Drugs
A 10/15-10/25/2024 FDA inspection at the Cosco International drug manufacturing facility in Marietta, GA, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 4/14 Warning Letter says the specific violations were: * failure to establish adequate written procedures for
Drugs
FDA says it will miss a 4/29 user fee review action extension on a Stealth BioTherapeutics NDA for elamipretide, a first-in-class mitochondria-targeted therapeutic for treating Barth syndrome. The review was extended in January to allow FDA to review supplemental information provided in response to recent requests
Drugs
FDA says it purchased from Amazon external analgesic products intended to be used before sensitive cosmetic procedures. A just-released 4/17 Warning Letter says the products are unapproved new drugs that are misbranded. The letter says the products are marketed as, among other things, external analgesics to be used
Drugs
A 10/16-11/4/2024 FDA inspection at the BEO Pharmaceuticals drug manufacturing facility in Brooksville, FL, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 4/9 Warning Letter says specific violations were: * failure to test samples each component for identity
Drugs
Pfizer says the pivotal Phase 3 CREST trial of sasanlimab, an investigational anti-PD-1 monoclonal antibody (mAb), in combination with standard of care (SOC) Bacillus Calmette-Guérin (BCG) met its primary endpoint as an induction therapy with or without maintenance in patients with BCG-naïve, high-risk non-muscle
Drugs
Johnson & Johnson says data from the Phase 2b SunRISe-1 study evaluating TAR-200, an intravesical gemcitabine releasing system, for patients with certain types of bladder cancer. “These first results show the promise of TAR-200 in this patient population with more than an 80% disease-free survival (DFS)
Drugs
FDA has issued Telix Pharmaceuticals a complete response letter on its NDA for Pixclara (floretyrosine F18), a positron emission tomography agent for imaging gliomas, a rare and life-threatening brain cancer. The letter says the NDA cannot be approved in its current form and that additional confirmatory clinical evidence is
Drugs
FDA has placed a clinical hold on VYNE Therapeutics’ Phase 1b study evaluating VYN202 for treating moderate-to-severe plaque psoriasis. The action was based on a recent observation of testicular toxicity in dogs from a non-clinical toxicology study with VYN202, according to the company. VYNE says it has
Drugs
A Northern Texas federal judge has denied a preliminary injunction sought by industry trade group Outsourcing Facilities Association to delay FDA enforcement actions against compounding pharmacies that make copies of Novo Nordisk’s weight-loss drug Wegovy (semaglutide) and the diabetes medication Ozempic (semaglutide). FDA earlier allowed compounding pharmacies to
Drugs
FDA is likely to see up to a 25%, if not greater, reviewer exodus over the coming months as about 600 reviewers have currently recused themselves from working on product evaluations while they look for employment in industry or outside of the federal government. “These are people who are looking
Drugs
Several anonymous current and former FDAers say the reductions in force the agency has experienced have led to key databases not being maintained and updated, meaning that healthcare professionals are unable to get basic information about drugs they prescribe. The staffers spoke with Axios for an online article. A laid-
Drugs
FDA has approved an Akeso Biopharma BLA for penpulimab-kcqx, indicated for adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC). The approval covers penpulimab in combination with platinum-based chemotherapy (either cisplatin or carboplatin) and gemcitabine as a first-line treatment for adults with recurrent or metastatic NPC.
Drugs
FDA has granted BrainChild Bio a breakthrough therapy designation for its investigational CAR T-cell therapy, BCB-276, for treating diffuse intrinsic pontine glioma (DIPG), a rare and fatal pediatric brain tumor. The company says the designation was granted based on encouraging clinical data from the Phase 1 BrainChild-03