Drugs
FDA has granted Umoja Biopharma a fast track designation for its investigational therapy UB-VV111 for treating relapsed or refractory Large B-cell Lymphoma and Chronic Lymphocytic Leukemia following two or more prior therapies. The company describes UB-VV111 as an off-the-shelf, in vivo CAR T cell therapy
Drugs
FDA has issued a complete response letter to Fortress Biotech and its majority-owned subsidiary, Cyprium Therapeutics, regarding their NDA for CUTX-101, a copper histidinate therapy for treating Menkes disease in pediatric patients. The letter cited manufacturing and GMP concerns at the facility producing CUTX-101, according to the
Drugs
FDA has approved a Novartis NDA for Rhapsido (remibrutinib), a Bruton’s tyrosine kinase (BTK) inhibitor for treating adults with chronic spontaneous urticaria (CSU) who remain symptomatic despite antihistamine therapy. Taken as a twice-daily pill, Rhapsido is designed to block BTK, a protein that activates immune cells responsible for
Drugs
FDA has published the eight FY 2026 Generic Drug User Fee Act science and research priority areas. The list was developed after a 6/3-6/4 public workshop to gather input on the priorities, a notice says. The agency says the eight priority areas “encompass scientific challenges that the
Drugs
A Bioresearch Monitoring Program inspection at a clinical site operated by Pamela Den Besten, DDS, in San Francisco, CA, found that she failed to submit an IND for the conduct of a clinical investigation with an investigational new drug. A 9/19 Warning Letter says Den Besten conducted a study
Drugs
A 1/27-2/26 FDA inspection at the Persōn & Covey drug manufacturing facility in Glendale, CA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. The inspection also found that two of the firm’s drug products are legally considered to be unapproved new
Drugs
CDER director George Tidmarsh has questioned the use of surrogate endpoints and has publicly attacked Aurinia Pharmaceuticals’ Lupkynis (voclosporin), which was approved in 2021 for treating lupus nephritis. In an unprecedented and now-deleted LinkedIn post, Tidmarsh claimed that voclosporin has “significant toxicity” and has not been shown to have
Drugs
In the 16 months since its founding, the CDER Quantitative Medicine Center of Excellence (QM CoE) says it has achieved “significant accomplishments” in three strategic areas — enterprise planning and coordination, applied regulatory science, and education and ecosystem development. A QM CoE Progress and Priorities Update says the Center of Excellence
Drugs
FDA researchers say that while much more work is needed, the Office of New Drugs (OND) specialized term grouping system for adverse event analysis, known as OND Custom Medical Queries (OCMQ), has been useful in premarket drug safety evaluation. Writing in the open-source journal Drug Safety, the researchers say
Drugs
FDA has posted an International Council for Harmonization draft guideline entitled E20 Adaptive Designs for Clinical Trials that provides recommendations on using adaptive designs in confirmatory clinical trials. The guideline lays out principles to help sponsors ensure that such trials produce reliable and interpretable evidence to support benefit-risk assessments
Drugs
FDA released a draft guidance to help drugmakers design clinical trials for treatments targeting disseminated coccidioidomycosis, also known as Valley Fever, a fungal disease caused by Coccidioides species. The document outlines the agency’s current thinking on trial designs, patient populations, and efficacy endpoints for evaluating potential therapies. Coccidioidomycosis is
Drugs
Larimar Therapeutics has reported encouraging long-term data from its open-label study of nomlabofusp, an investigational therapy for Friedreich’s ataxia (FA), while confirming plans to seek accelerated FDA approval in the second quarter of 2026. The study, which is evaluating daily subcutaneous doses of nomlabofusp in adolescents and
Drugs
FDA is stepping up efforts to close persistent gaps in clinical trial reporting, warning that incomplete or delayed disclosures threaten public trust and scientific progress. An online post from CDER director George Tidmarsh notes that clinical trial transparency is “an ethical obligation under the Declaration of Helsinki” and a cornerstone
Drugs
AbbVie has filed an NDA for tavapadon, an investigational once-daily oral therapy for Parkinson’s disease, based on data from late-stage clinical trials that showed the treatment improved symptoms across different stages of the condition. Tavapadon is described by the company as a selective dopamine D1/D5 receptor
Drugs
FDA has approved a Crinetics NDA for Palsonify (paltusotine) for treating adults with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option. The rare endocrine disorder causes some bones, organs, and other tissue to grow bigger, according to an FDA release.
Drugs
FDA has issued Novartis Pharmaceuticals an untitled letter citing the company’s allegedly false or misleading direct-to-consumer television advertisement for Fabhalta (iptacopan), saying the ad downplays serious safety risks and exaggerates the drug’s benefits. Fabhalta, approved 12/2023, is a twice-daily oral treatment for paroxysmal nocturnal
Drugs
Hansa Biopharma says investigational imlifidase met the primary endpoint in a pivotal Phase 3 study in highly sensitized kidney transplant patients, setting up a potential U.S. regulatory filing later this year. The U.S.-based ConfIdeS trial enrolled adults with a calculated panel reactive antibody level of 99.9%
Drugs
FDA has published its Electronic Submission Gateway NextGen Unified Submission Portal Guide for Industry Users, outlining the functionalities, processes, and requirements for submitting regulatory information through the NextGen Unified Submission Portal (USP). The document says it is intended to facilitate a smooth transition to Electronic Submission Gateway NextGen, providing users
Drugs
FDA has approved an Eli Lilly NDA for Inluriyo (imlunestrant, 200 mg tablets), an oral estrogen receptor antagonist for treating adults with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2–), ESR1-mutated advanced or metastatic breast cancer (MBC) whose disease progressed after at least one line
Drugs
In a 9/19 letter to 20 Republican attorneys general, HHS secretary Robert F. Kennedy, Jr., and FDA commissioner Martin Makary say FDA is leading a review of the medication abortion drug mifepristone. Kennedy and Makary wrote in response to a 7/31 letter from the attorneys general raising concerns
Drugs
FDA says it has removed the REMS (Risk Evaluation and Mitigation Strategy) program for Sanofi’s thyroid cancer drug Caprelsa (vandetanib). An agency notice says Caprelsa will remain available with the same prescribing information, but healthcare providers will no longer need special certification or extra monitoring beyond standard clinical care.
Drugs
A 12/3-12/6/2024 FDA Bioresearch Monitoring Program inspection involving two clinical trial protocols at the University of Texas Health Science Center at San Antonio, with Dr. Ralph DeFronzo as clinical investigator, found “objectionable conditions” and a failure to follow the law and regulations governing the conduct of