Drugs
FDA has denied as unwarranted a 3/1/2024 petition submitted by Aimed Alliance asking that the agency issue a guidance with an interpretation and a clear position statement about drug importation activities conducted by alternative funding programs. Specifically, the petition said the proposed guidance should address: * whether third-party
FDA Policy/General
Agios Pharmaceuticals says it plans to seek accelerated approval for mitapivat in sickle cell disease, following a pre-submission meeting with FDA. At the meeting, FDA advised the company to include a confirmatory clinical trial as part of an accelerated approval pathway submission. Agios says it has already submitted a
Drugs
FDA has issued a safety alert warning that patients taking Amgen’s Tavneos (avacopan) for a rare autoimmune condition may face a risk of severe liver injury, including cases that have resulted in death. The agency says it has identified postmarketing reports of drug-induced liver injury (DILI) in patients
FDA Policy/General
FDA is seeking industry and public input on how to expand the use of digital health technologies (DHT) in clinical trials, as part of a broader push to modernize drug and biologic development and regulatory decision-making. DHTs — ranging from wearable sensors and smartphone apps to implantable or ingestible devices
FDA Policy/General
Scholar Rock has resubmitted its BLA for apitegromab, an investigational therapy for children and adults with Spinal Muscular Atrophy (SMA), following alignment with FDA on manufacturing and supply issues that previously delayed approval. The resubmission follows a 9/2025 complete response letter that cited inspection findings at a contract manufacturing
Biologics
A novel “off-the-shelf” CAR-T cell therapy developed by researchers at Washington University School of Medicine in St. Louis has received an FDA breakthrough therapy designation. The therapy, known as WU-CART-007 (soficabtagene geleucel), is designed to treat T-cell acute lymphoblastic leukemia and T-cell lymphoblastic
Drugs
While FDA has been taking a more aggressive approach to publicizing its criticisms of drugmakers, the shift is drawing both praise from investors and concern across the pharmaceutical industry, according to a 3/30 analysis by PharmaVoice. The agency’s evolving stance, which is part of the Trump administration’s
Drugs
FDA inspection records show that some generic drug manufacturers are routinely failing to carry out basic safety and quality checks, raising concerns about the integrity of medications reaching U.S. patients, according to a new report published March 30 by the MedShadow Foundation. The analysis, authored by Emma Yasinski, found
Biologics
CBER has issued four untitled letters in a single coordinated enforcement action, targeting promotional materials for leading T-cell therapies and signaling heightened scrutiny of how cutting-edge biologics are marketed. The letters fit into a wider FDA enforcement trend. Since late 2025, the agency has significantly increased scrutiny of
Drugs
FDA has accepted for priority review a Praxis Precision Medicines NDA for relutrigine, an investigational therapy targeting rare genetic epilepsies. The agency set a user fee review action target date of 9/27. Relutrigine is being developed to treat developmental and epileptic encephalopathies (DEEs) caused by mutations in the SCN2A
Drugs
FDA has approved a higher-dose regimen of Biogen’s Spinraza (nusinersen) for treating spinal muscular atrophy, giving patients and clinicians a new dosing option aimed at improving outcomes in the rare neuromuscular disorder. The decision is based on data from the Phase 2/3 DEVOTE study and expands on
Drugs
Merck says new Phase 3 data show its investigational cholesterol drug enlicitide significantly lowered “bad” cholesterol compared with commonly used non-statin pills, positioning the therapy as a potential oral alternative to injectable PCSK9 inhibitors. Results from the CORALreef AddOn trial, presented at the American College of Cardiology Annual Scientific
Biologics
FDA has granted Rocket Pharmaceuticals accelerated approval for its gene therapy Kresladi (marnetegragene autotemcel) for children with severe Leukocyte Adhesion Deficiency-I (LAD-1), marking the first approved treatment targeting the underlying genetic cause of the disease. The therapy’s approval covers pediatric patients with biallelic mutations in the ITGB2
Drugs
FDA has issued an untitled letter to STAQ Pharma of Ohio, citing significant manufacturing, quality control, and labeling violations that could put patients at risk. The just-posted 12/5/2025 letter follows an FDA inspection conducted in May at the company’s Columbus, OH facility, which is registered as
Devices
FDA has issued a final guidance titled Incorporating Voluntary Patient Preference Information (PPI) Over the Total Product Life Cycle. The document was mandated under the medical device user fee program to provide insight and address common questions for those interested in the voluntary use of PPI in regulatory submissions. The
FDA Policy/General
An opinion piece published by The Washington Times’ editorial board is drawing attention to growing criticism of FDA commissioner Marty Makary, arguing that his agency leadership reflects ideology overtaking scientific judgment. The editorial takes aim at recent remarks by Makary and personnel decisions within the agency, raising concerns about their
Devices
FDA has cleared a Medtronic 510(k) for its Stealth AXiS surgical system in cranial and ear, nose and throat procedures, extending the reach of a platform the company is positioning as a unified navigation and robotics hub across surgical specialties. The latest clearance follows an earlier nod for spine
Devices
Erbe USA has recalled (Class 1) its flexible cryoprobes due to their potential to rupture during activation, which could result in severe injury or death. To date, there have been five serious injuries linked to the malfunction, FDA says. According to Erbe USA, the issue stems from insufficient adhesive applied
Drugs
A new CDER guidance recap podcast explains the newly published FDA draft guidance on the use of Bayesian methodology in clinical trials of drug and biological products. On the podcast, CDER Office of Biostatistics master mathematical statistician James Travis says the guidance is important because, as the number of proposals
Drugs
At the 2/26 PDUFA 8 reauthorization meeting, FDA said it would consider an industry counterproposal to the agency’s America First fee incentive proposal. Meeting minutes indicate that industry representatives said FDA’s proposed three-time fee differential would be “extremely punitive rather than an incentive for substantial changes
Drugs
In an FY 2025 GDUFA Science and Research Report, GDUFA Science and Research Program joint directors Iilun Murphy and Michael Kopcha say extended GDUFA-funded research on bioequivalence (BE) risk assessments and Biopharmaceutics Classification System classification supported the FY 2025 publication of over 800 product-specific guidances that aligned FDA’
FDA Policy/General
University of Southern California Price School of Public Policy associate professor Genevieve Kanter says policymakers should consider conflict of interest policies to be part of a broader strategy of building public trust by being more trustworthy as an agency. “This includes providing comprehensive scientific justification for decisions, being transparent about