Drugs
FDA has accepted for review a Merck NDA for its investigational once-daily HIV treatment combining doravirine and islatravir (DOR/ISL). The two-drug regimen is intended for adults with HIV-1 infection who are already virologically suppressed on existing antiretroviral therapy. The agency has set a user fee review
Drugs
The House Energy and Commerce Committee Health Subcommittee has scheduled a 7/16 public hearing titled “Legislative Proposals to Maintain and Improve the Public Health Workforce, Rural Health, and Over-the-Counter Medicines.” Energy and Commerce Committee chair Brett Guthrie (R-KY) and Health Subcommittee chairman-designate Morgan Griffith (R-
Drugs
FDA has approved an updated dosing schedule for Eli Lilly’s Alzheimer’s therapy Kisunla (donanemab-azbt), aimed at reducing the risk of a known side effect called amyloid-related imaging abnormalities with edema (ARIA-E). Approved 7/2024 as a once-monthly infusion therapy targeting amyloid plaques in the
Drugs
A 12/3-12/6/2024 FDA inspection at the Dynamic Blending Specialists drug manufacturing facility in Vineyard, UT, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 6/15 Warning Letter says the specific violations were: * failure to conduct at least one
Drugs
A 3/11-3/14 FDA inspection at the Libby Laboratories drug manufacturing facility in Berkeley, CA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 6/27 Warning Letter says the specific violations were: * failure to conduct for each batch of drug product, appropriate
Drugs
Rhythm Pharmaceuticals is announcing positive topline results from its Phase 2 trial evaluating bivamelagon (formerly LB54640) in patients with acquired hypothalamic obesity. The investigational oral melanocortin-4 receptor (MC4R) agonist achieved statistically significant and clinically meaningful reductions in body mass index at 14 weeks of treatment, the company says. Rhythm
Drugs
A Washington state federal judge has reversed his earlier injunction and determined that FDA legally restricted the availability of mifepristone and misoprostol, used in combination for a chemical abortion. Courthouse News Service reports that Washington, Oregon, and 15 other blue states plus Washington, DC, had sued to maintain access to
Drugs
FDA has granted Trethera a fast track designation for TRE-515 for treating prostate cancer. TRE-515 is currently in Phase 1 clinical trials for solid tumors. It is described by the company as a first-in-class clinical-stage drug that inhibits deoxycytidine kinase, a rate-limiting enzyme in
Drugs
More than 50 biotechnology industry executives and investors have sent a letter to FDA commissioner Marty Makary, urging the agency to reject efforts to restrict access to the abortion medication mifepristone. The letter supports a recent citizen petition by three major medical organizations — the American College of Obstetricians and Gynecologists,
Drugs
FDA says a new analysis of human pregnancy data compiled from the use of endothelin receptor antagonist (ERA) medicines for 20 years found that REMS requirements about embryofetal toxicity (EFT) risk are no longer needed. A notice says the agency believes that labeling alone is adequate to communicate the risk,
Drugs
An 11/4-11/15/2024 FDA inspection at the Exela Pharma Sciences outsourcing drug manufacturing facility in Lenoir, NC, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 7/2 Warning Letter says the specific violations were: * failure to thoroughly investigate any unexplained discrepancy
Drugs
Representing 39 additional health advocacy organizations, the Association for Accessible Medicines has written to the Republican and Democratic leaders of the Senate HELP Committee and the House Energy and Commerce Committee to voice support for S. 1954, the Biosimilar Red Tape Elimination Act. The letter says the bill would remove
Drugs
A Federation of American Scientists (FAS) online paper calls for an interagency approach to increase transparency in validating surrogate endpoints used in FDA drug approvals. While FDA regularly updates its Table of Surrogate Endpoints That Were the Basis of Drug Approval or Licensure, FAS says, the table does not include
Drugs
Taiho Pharmaceutical is reporting disappointing data from its Phase 3 extension study of TAS-205 (pizuglanstat) in patients with Duchenne muscular dystrophy (DMD) (REACH-DMD) that showed no significant difference in the mean change from baseline to 52 weeks in the time to rise from the floor, which was the
Drugs
A new FDA clinical trial has found that cannabidiol (CBD) at doses typical of common consumer use may cause liver enzyme elevations in otherwise healthy adults — raising new concerns about the safety of unregulated CBD products widely available in the U.S. The study, published this week in JAMA Internal
Drugs
FDA has issued a complete response letter (CRL) to Unicycive Therapeutics for its NDA for oxylanthanum carbonate (OLC) to treat hyperphosphatemia in dialysis patients with chronic kidney disease. A company statement says the letter cited deficiencies previously identified at a third-party manufacturing vendor unrelated to OLC. There were no
Drugs
FDA has given accelerated approval to Dizal (Jiangsu Pharmaceutical Co., Ltd.) for its Zegfrovy (sunvozetinib) for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on
Drugs
A 9/3-9/18/2024 FDA inspection at Staska Pharmaceuticals, an outsourcing facility in Bennet, NE, found that the drug products produced failed to meet the legal conditions necessary for drugs produced by an outsourcing facility to qualify for exemptions from certain provisions of the Federal Food, Drug, and
Drugs
FDA has approved a KalVista Pharmaceuticals NDA for Ekterly (sebetralstat) for treating acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. The approval comes after FDA told KalVista it would not meet a 6/17 user fee review action target data for the
Drugs
FDA has accepted for priority review a Denali Therapeutics’ BLA for tividenofusp alfa and its use for treating Hunter syndrome. The agency has set a user fee review action target date of 1/5/2026. Tividenofusp alfa is described by the company as a next-generation enzyme replacement therapy designed
FDA Policy/General
Ten current and former FDA staff members and leaders who spoke anonymously with the investigative journalism newsroom ProPublica say agency inspectors continue to suffer from the impact of significant staff cuts and reorganizations, even as the Trump administration officially denies that the cuts had an impact on inspections. In its
Drugs
FDA has accepted and granted priority review for an sBLA seeking approval to update the label for Merck’s Winrevair (sotatercept-csrk) based on the Phase 3 ZENITH trial. A company statement says Winrevair was approved in 2024 to treat adults with pulmonary arterial hypertension (PAH) to increase exercise capacity,