Drugs
FDA has granted Johnson & Johnson a breakthrough therapy designation for nipocalimab and its use for treating adults living with moderate-to-severe Sjögren's disease (SjD), a debilitating and chronic autoantibody disease. Nipocalimab is said to be the only investigational therapy to win breakthrough status for SjD. The
Drugs
AbbVie says its two Phase 2 EMPOWER trials investigating emraclidine as a once-daily, oral monotherapy treatment for adults with schizophrenia who are experiencing an acute exacerbation of psychotic symptoms did not meet their primary endpoint. Specifically, the trials did not show a statistically significant reduction (improvement) in the change
Drugs
FDA has released a recent untitled letter that was sent to Brookfield Medical/Surgical Supplies, an outsourcing facility in Brookfield, CT, due to GMP issues cited during a May inspection. The four inspection observations in the letter were: * failure to follow procedures designed to prevent microbiological contamination of drug products
Drugs
FDA has accepted for review an Unicycive Therapeutics 505 (b) (2) NDA for oxylanthanum carbonate (OLC) and its use in treating hyperphosphatemia patients with chronic kidney disease (CKD) on dialysis. The agency has set a 6/28/2025 user fee review action target date. Unicycive says OLC is designed to
Drugs
Allogene Therapeutics is reporting encouraging response rate data for its investigational ALLO-316 in advanced renal cell carcinoma (RCC), but the results also showed three patient deaths (Grade 5 adverse events). The results are from the Phase 1 TRAVERSE trial of ALLO-316’s potential as an “off-the-shelf”
Drugs
Advocacy group Public Citizen is criticizing FDA for dragging its feet in removing “ineffective, albeit safe, over-the-counter oral formulations of phenylephrine from the U.S. market.” On 11/7, FDA issued a proposed rule to remove the ingredient found in cough and cold medicines, either alone or in
Drugs
Dizal Pharmaceutical has submitted an NDA for sunvozertinib for treating locally advanced or metastatic non-small cell lung cancer patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins). The submission is based on data from the WU-KONG1 Part B study that demonstrated sunvozertinib had a statistically
Drugs
FDA has extended by three months its review of a Neurotech Pharmaceuticals BLA for NT-501 (revakinagene taroretcel) as a treatment for macular telangiectasia Type 2 (MacTel). The extension is needed to review additional data provided by the company in response to recent requests from the agency. The revised user
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says it appreciates FDA taking steps to increase the use of model-informed drug development (MIDD) approaches in regulatory decision-making, to understand how sponsors are using emerging MIDD approaches within medical product development, and to enhance interactions with sponsors regarding MIDD. Responding
Drugs
Public Citizen says a potential legislative initiative expected to be advanced in 2025 would be dangerous in exempting rare disease drugs from the requirement to be subject to randomized trials. In a statement, Public Citizen says the proposal is being developed by retired FDA CDER director, acting commissioner, and principal
Drugs
Sarepta says it is discontinuing the development of SRP-5051 (vesleteplirsen), an investigational, next-generation peptide conjugated phosphorodiamidate morpholino oligomer for treating patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. The decision was based on FDA feedback about the therapy’s risk-benefit, according to the
Drugs
FDA has released its analysis of FY 2023 institutional review board (IRB) violations based on the form FDA-483s that were issued. The report says that broadly written regulations do not always convey the specific details of the observed violations. The agency says it reviewed and sub-categorized FDA-483
Drugs
FDA has approved Caris Life Sciences’ MI Cancer Seek and its use as a companion diagnostic (CDx) to identify cancer patients who may benefit from treatment with targeted therapies. “The assay includes one pan-cancer and five tumor-specific indications for numerous FDA-approved therapies,” the company says. “MI Cancer
Drugs
FDA has released the form FDA-483 issued following a 9/3-9/18 inspection at the Staska Pharmaceuticals outsourcing facility in Bennet, NE. The nine inspection observations were: * failure to thoroughly review any unexplained discrepancy and the failure of a batch or any of its components to meet any
Drugs
Three stakeholder organizations are asking FDA to revise the Risk Evaluation and Mitigation Strategy (REMS) for the psychiatric drug clozapine to reduce its barriers to treatment for many patients. In its comment for a joint meeting of the Drug Safety and Risk Management Advisory Committee and the Psychopharmacologic Drugs Advisory
Drugs
An FDA 11/2-11/8/2023 Bioresearch Monitoring Program inspection at the Armstrong County Memorial Hospital Institutional Review Board (IRB) in Kittanning, PA., found “objectionable conditions” indicating that the IRB did not adhere to applicable statutory requirements on the protection of human subjects. A 10/29 Warning Letter emphasized
Drugs
In an NPR interview, FDA commissioner Robert Califf says the field of artificial intelligence will be so big that FDA couldn’t possibly hire enough people to regulate it alone. “It’s very important that we get the clinical community, the health systems, the professions, the people you see for
Drugs
Acadia Pharmaceuticals says it is selling its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million to an undisclosed buyer. The PRV was awarded to Acadia 3/2023 following FDA approval of the company’s Daybue (trofinetide) for treating Rett syndrome. The company says the sale proceeds will support
Drugs
FDA has extended by three months its review of an Organon supplemental NDA for Vtama (tapinarof) cream, 1%, for treating atopic dermatitis in adults and children two years of age and older. The revised user fee review action target date is 3/12/2025. During its review, FDA requested the
Drugs
An FDA review of records submitted by Shandong Boyuan Pharmaceutical Co. in Shandong, China, at the agency’s request, identified current good manufacturing practice (CGMP) deviations in the firm’s manufacturing of active pharmaceutical ingredients (APIs). A just-released 10/17 Untitled Letter says the specific deviations were: * failure to
Drugs
A 5/17-5/17 FDA inspection at the Analytical Food Laboratories contract testing laboratory in Grand Prairie, TX, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 10/10 Warning Letter says specific violations were: * failure to establish and follow required laboratory
Biologics
Four stakeholders — St. Jude Children’s Research Hospital, the National Organization for Rare Disorders (NORD), the Muscular Dystrophy Association (MDA), and the Biotechnology Innovation Organization (BIO) — are voicing support for an FDA proposal to create an FDA Rare Disease Innovation Hub (RDI Hub). The four also respond to FDA questions