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
FDA Policy/General
A “well-reasoned” recent Supreme Court opinion in a case involving FDA “is a welcome source of reassurance at a time when President Donald Trump is waging an unprecedented attack on regulatory agencies.” That’s the view of George Washington University School of Law professor Richard Pierce, writing in The
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
FDA Policy/General
Reps. Greg Murphy (R-NC), Angie Craig (D-MN), Nick Begich (R-AK), and Hillary Scholten (D-MI) have introduced the No Handouts for Drug Advertisements Act, a bill that would prohibit tax deductions for the annual $6 billion in expenses related to direct-to-consumer (DTC) advertising of prescription
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
Biologics
FDA has approved Abeona Therapeutics’ Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, an autologous cell-based gene therapy for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a debilitating genetic skin disease. The BLA resubmission was filed 11/2024 following a 4/2024 complete response
Devices
Attorneys Melissa Bianchi and Jodi Scott (Hogan Lovells) say medical device companies can learn from a recent FDA Warning Letter to Exer Labs citing a failure to obtain PMA or 510(k) clearance for its Exer Scan mobile AI-based screening tool. The letter also cited good manufacturing practice violations
Biologics
A Memorial Sloan Kettering Cancer Center clinical trial has demonstrated that immunotherapy alone can allow people with MMRd (mismatch repair deficiency) cancers to avoid surgery and preserve their quality of life, according to a cancer center statement. The statement says the Phase 2 trial showed that 80% of patients with
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)
Biologics
Novavax and HHS are having to clarify that FDA is requesting a post-approval study and not a clinical trial that must be conducted prior to approval of the company’s Covid-19 vaccine, which is currently available under an emergency use authorization(EUA). The company’s pending BLA is
Devices
Medtronic has submitted a PMA for its Hugo robotic-assisted surgery (RAS) system for a urologic indication. The submission is based on data from the company’s Expand URO clinical study, which met both primary safety and effectiveness endpoints. “The prospective, multi-center, single-arm IDE study included 137 patients
FDA Policy/General
FDA has given BlackfinBio the green light to conduct a Phase 1/2 clinical trial of its novel adeno-associated virus gene therapy, BFB-101, in children with hereditary spastic paraplegia, Type 47 (SPG47). Previously, the agency awarded the company an orphan drug designation and rare pediatric disease designation for
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
FDA Policy/General
HHS secretary Robert F. Kennedy Jr. earlier this week vowed to restore FDA’s and other health agencies’ Freedom of Information Act (FOIA) offices that were gutted earlier this month under White House-mandated staff cuts. The removal of FOIA offices appeared to go against previous Kennedy promises of “radical
Devices
FDA has granted Orchestra BioMed a breakthrough device designation for its atrioventricular interval modulation (AVIM) therapy, a pacemaker that uses conduction pacing to reduce blood pressure in patients with increased 10-year atherosclerotic cardiovascular disease risk, preserved left ventricular systolic function, and uncontrolled hypertension, despite the use of anti-hypertensive