Drugs
FDA has granted Opna Bio an orphan drug designation for its investigational treatment OPN-2853 (zavabresib), a small-molecule bromodomain and extra-terminal motif inhibitor, for myelofibrosis, which is characterized by progressive scarring of the bone marrow and leading to impaired blood cell production and symptoms that can include severe
Drugs
Nearly half of drug shortage announcements led to protective purchasing intended to safeguard patient access, according to a report in the United States Pharmacopeia online Quality Matters newsletter. While protective purchasing is intended to preserve patient access, the report says, it can have the unintended consequence of deepening supply disruptions
Drugs
FDA has released the form FDA-483 issued following an 11/13-11/21/2025 inspection at the Sato Pharmaceutical over-the-counter sterile and non-sterile drug manufacturing facility in Hachioji, Tokyo, Japan. The 10 specific inspection observations were: * failure to thoroughly review any unexplained discrepancy and the failure
Drugs
IntraBio is reporting positive results from a pivotal Phase 3 clinical trial showing that levacetylleucine significantly improved neurological symptoms in patients with ataxia-telangiectasia, a rare and progressive neurodegenerative disease with no approved treatments. The trial met its primary endpoint, the company says, demonstrating a statistically significant and clinically meaningful
Drugs
FDA says it will start accepting applications on 2/1 for the PreCheck Pilot Program, which it says is intended to strengthen the domestic pharmaceutical supply chain by increasing regulatory predictability, facilitating the construction of U.S. manufacturing sites, and streamlining aspects of pharmaceutical manufacturing facility assessments before a specific
Drugs
Four stakeholder organizations are voicing basic support for an FDA draft guidance, Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies. Pharmaceutical Research and Manufacturers of America (PhRMA) says it supports a “totality of the evidence” approach to determining whether
Drugs
Cogent Biosciences says FDA has agreed to accept the company’s planned NDA for cancer drug bezuclastinib under the agency’s Real-Time Oncology Review program, based on positive late-stage trial results in patients with gastrointestinal stromal tumors (GIST). The company will seek approval for the therapy and its
Drugs
FDA is touting upcoming actions to encourage moving more medications from prescription-only status to over-the-counter (OTC) availability, a shift the agency says could lower drug prices, reduce unnecessary emergency room visits, and streamline access to common treatments. In an interview with The Daily Wire, FDA commissioner Marty
Drugs
FDA says its ImportShield Program, formerly known as the Nationalized Entry Review Program, increased processing speed by 66%, increased monthly volume capacity by 33%, and reduced staff hours by 20%, all in its first four months of operation (since 8/2025). An agency statement says the new program “streamlines FDA’
Devices
Advanced Medical Technology Association (AdvaMed) is urging FDA to narrow and clarify a draft guidance on quality management system information, warning that the current proposal could add unnecessary burden and confusion to the premarket review process. In just-posted comments, the group said FDA’s recent draft guidance on Quality
Drugs
Novo Nordisk has submitted to FDA its second supplement to its nomination of semaglutide products (Ozempic and Wegovy) to the list of drug products that present demonstrable difficulties for compounding under the Federal Food, Drug, and Cosmetic Act (FFDCA). “This supplement adds to the body of evidence that demonstrates the
Drugs
A 12/17/2024-1/10/2025 FDA Bioresearch Monitoring Program inspection of the Temecula, CA, Prodrome Sciences USA clinical investigation of Omega-3 plasmalogens found objectionable conditions indicating that the company did not adhere to laws and regulations governing the conduct of clinical investigations and the protection of human
Drugs
FDA has released a draft guidance outlining how drug developers can use minimal residual disease (MRD) and complete response (CR) as primary endpoints in multiple myeloma trials to support accelerated approval, a move that could further speed access to new treatments in a disease area where outcomes have steadily improved.
Biologics
Moderna and Merck say that updated five-year follow-up data show that their personalized mRNA cancer vaccine candidate, intismeran autogene, significantly reduced the risk of melanoma recurrence or death when used in combination with Merck’s immunotherapy Keytruda (pembrolizumab), compared with Keytruda alone, according to the companies. In a
Biologics
FDA has asked Capricor Therapeutics for a full clinical study report from the company’s Phase 3 HOPE-3 trial as part of its ongoing BLA review for Deramiocel, a proposed cell therapy for Duchenne muscular dystrophy. The request follows the agency’s review of topline HOPE-3 data that
Drugs
FDA has published for review and comment an International Council for Harmonization guidance, The Common Technical Document for the Registration of Pharmaceuticals for Human Use: Quality, M4Q(R2), establishing the location and structure of quality information for registration applications of all medicinal products for human use. The document says it
Drugs
A 6/16-6/24/2025 FDA inspection at the Integrity Partners Group’s Chemisphere drug manufacturing facility in St. Louis, MO, identified four current good manufacturing practice (CGMP) regulation violations in the firm’s production of finished drugs. A just-released 12/15/2025 Warning Letter says the specific
Biologics
Valneva says it has voluntarily withdrawn its BLA for Ixchiq, the company’s chikungunya vaccine, based on FDA safety-related restrictions on the product. In 8/2025, CBER suspended Valneva’s BLA due to serious safety concerns that the vaccine could be causing chikungunya-like illness in vaccine recipients. The
Drugs
FDA has accepted for priority review a Celcuity NDA for gedatolisib in patients with hormone receptor–positive, HER2-negative, PIK3CA wild-type advanced breast cancer. The company says the submission is based on data from the PIK3CA wild-type cohort of Celcuity’s Phase 3 VIKTORIA-1 trial, which evaluated
Drugs
FDA inspectors cited Oklahoma-based outsourcing facility Qualgen for a wide range of manufacturing and quality control failures during a recent inspection, including inadequate controls to prevent cross-contamination of hazardous drugs and repeated lapses previously flagged by the agency. According to an FDA Form 483 issued at the conclusion
Drugs
The U.S. Supreme Court has agreed to hear a patent-infringement dispute between generic drugmaker Hikma Pharmaceuticals USA, Inc. and brand-name company Amarin to decide whether generic drugmakers may face liability for inducement of patent infringement when they market a drug under a so-called “skinny label” — i.
Drugs
Former FDA oncology drug leader Richard Pazdur said concerns surrounding a controversial fast-track drug review initiative under commissioner Marty Makary reflect deeper problems inside the agency, including politicization of regulatory decisions, diminished transparency, and disregard for scientific staff — issues that ultimately led to his departure from the agency late