Drugs
A BMJ investigation asks whether FDA broke its own rules requiring substantial evidence that a drug is effective when it approved in 7/2019 Merck’s antibiotic Recarbrio (relebactam and imipenem-cilastin). “Despite the absence of any clinical studies to provide substantial evidence of its effectiveness, the agency approved the
Drugs
Viking Therapeutics says top-line results from its Phase 2b clinical trial of VK2809 in patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH) show that it met the primary endpoint — statistically significant reductions in liver fat content from baseline to Week 12 as compared with placebo. NASH is a liver
Drugs
Pharmaceutical Research and Manufacturers of America says it appreciates FDA providing the updated guidance and recognizing the “advances in technology” that require additional recommendations. “In general,” it says, “we believe the draft guidance provides useful information to sponsors and other regulated entities regarding FDA’s electronic signatures to be trustworthy,
Drugs
FDA has cleared a Waypoint Orthopedics 510(k) for the Waypoint GPS, a smart bone awl for use during pedicle screw pilot hole drilling. The device is designed to provide operators with additional information via visual feedback through its proprietary color-sensing technology. The visual feedback is displayed wirelessly to
Drugs
FDA has issued a final guidance entitled “Reporting and Mitigating Animal Drug Shortages.” The document provides recommendations on the information sponsors should submit to CVM to report and mitigate animal drug shortages. The agency notes that while reporting animal drug shortages is voluntary, receiving information about shortages allows it to
Drugs
FDA has granted Globus Medical a humanitarian device exemption to allow the marketing of the Reflect Scoliosis Correction System, which the company says is designed to correct progressive scoliosis in young patients while preserving motion, maintaining stability, and allowing for future modulated growth. “Unlike rigid metal rods for fusion, Reflect
Drugs
The U.S. Supreme court has declined to hear a closely watched appeal of a lower court’s ruling in a so-called “skinny label” case won by GlaxoSmithKline (GSK) against Teva over a generic form of GSK’s heart drug Coreg (carvedilol). The question at issue was: If a
Drugs
FDA’s Pulmonary-Allergy Drug Advisory Committee 5/11 voted to recommend approval for ARS Pharmaceuticals’ neffy (epinephrine) nasal spray for treating severe allergic reactions in adults and children. If approved, neffy would become the first needle-free epinephrine product. Panel members voted 16 to 6 to back approval for
Drugs
FDA has granted Pyxis Oncology an orphan drug designation for PYX-201 in pancreatic cancer. The company says subject dosing is already underway in a Phase 1 trial known as PYX-201-101. Preliminary data from the trial is expected in early 2024. PYX-201 is described by Pyxis as
Drugs
A bipartisan group of U.S. Senators and House members are urging FDA to step up its regulatory activities to improve rare disease product development and reviews. In a 5/2 letter, the lawmakers ask FDA to “establish an agency task force to, first, identify areas of strength and areas
Drugs
FDA has approved an Otsuka Pharmaceutical and Lundbeck supplemental NDA for Rexulti (brexpriprazole) oral tablets for treating agitation associated with dementia due to Alzheimer's disease. Approval was based on data from two 12-week, randomized, double-blind, placebo-controlled studies. “In both studies, patients who received 2 mg
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says it appreciates FDA efforts to meet its PDUFA 7 commitment to discuss negative control methods in real-world data studies and their potential to enhance and modernize the agency drug safety system. The comments came in response to the FDA 3/8
Drugs
The Council for Responsible Nutrition (CRN) petitioned FDA 3/9 to reconsider how it has been invoking the drug preclusion clause of the Dietary Supplement Health and Education Act (DSHEA). CRN says (includes a link to the petition) that the agency has been and will likely continue to be misapplying
Drugs
FDA has approved expanded Magnetic Resonance Imaging (MRI) labeling for Abbott’s Eterna spinal cord stimulation (SCS) system to include new leads that are magnetic resonance (MR)-conditional. Abbott says the expanded labeling “means people with chronic pain can undergo MRI scans within the approved outlined conditions and have a
Drugs
FDA has published a Study Data Technical Conformance Guide incorporating a prior guidance, Providing Regulatory Submission in Electronic Format — Standardized Study Data. The guide provides specifications, recommendations, and general considerations on how to submit standardized study data using FDA-supported data standards located in the FDA Data Standards Catalog. The
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AdvaMed says it “strongly supports FDA’s efforts to provide recommendations regarding requirements in clinical investigations for when electronic systems, electronic records, and electronic signatures are considered trustworthy, reliable, and generally equivalent to paper records and handwritten signatures.” The association’s comments responded to an FDA draft Q&A
Drugs
FDA says it is requiring sponsors of amphetamine and methylphenidate products used to treat attention deficit/hyperactivity disorder and other disorders to update and standardize their prescribing information to clearly inform patients, caregivers, and healthcare professionals of the risks associated with the medications. An online notice says the required updates
Drugs
CDER director Patrizia Cavazzoni says the Center has published a discussion paper, Using Artificial Intelligence and Machine Learning in the Development of Drug and Biological Products, to initiate a discussion with interested parties in the medical products development community on using artificial intelligence/machine learning (AI/ML) in drug and
Drugs
The FDA Cellular, Tissue, and Gene Therapies Advisory Committee meets 5/12 to consider a Sarepta BLA seeking accelerated approval for its SRP-9001 (delandistrogene moxeparvovec), an adeno-associated virus vector-based gene therapy product, to treat ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the
Drugs
Sens. Debbie Stabenow (D-MI) and Tina Smith (D-MN) and Rep. Jamie Raskin (D-MD) have introduced the Pharmaceutical Research Transparency Act to “increase transparency and make drug companies disclose their research and development expenses.” A statement issued by Stabenow says the bill follows a Government Accountability Office report
Drugs
Advisory committee members 5/10 voted 17 to 0 to recommend the approval of a Laboratoire HRA Pharma NDA seeking a prescription-to-nonprescription switch of its Opill, a norgestrel tablet 0.075 mg oral contraceptive pill. Overwhelmingly, panel members dismissed FDA concerns outlined in a briefing document released in
Drugs
FDA has published an immediately effective guidance, Testing of Glycerin, Propylene Glycol, Maltitol Solution, Hydrogenated Starch Hydrolysate, Sorbitol Solution, and Other High-Risk Drug Components for Diethylene Glycol and Ethylene Glycol, to alert drug manufacturers, compounders, repackers, and suppliers to the potential health hazard of glycerin and other high-risk