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
Devices
FDA has cleared an Exactech 510(k) for the Equinoxe Scapula Reconstruction System for treating acromial and scapular spine fractures. The device allows orthopedic surgeons to treat scapular fractures using several different techniques, including single and dual plating, regardless of placement of any particular reverse total shoulder arthroplasty implant design.
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
Devices
AirLife and Vyaire are recalling (Class 1) certain infant heated wire circuits due to the risk that accessory adapters may unintentionally disconnect during setup or use when the circuit reaches operating temperature. “Use of the affected product may cause serious adverse health consequences, including lack of oxygen to the body
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
FDA Policy/General
FDA needs to expand its reliance on real-world clinical data to monitor the safety and effectiveness of approved medical products, a move that would augment data from initial clinical trials according to Ethics and Public Policy Center physician and bioethicist Aaron Kheriaty. Writing in an opinion piece in The
Devices
A 1/21-1/27 FDA inspection at Insightra Medical Inc. in Clarksville, TN, found multiple violations in the firm’s manufacture of the Double Pump and the Ultra Intra Aortic Balloon Pump (IABP) catheter kits. A just-released 6/3 Warning Letter says the IABP catheter kit is adulterated
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
Devices
FDA has approved an expanded indication for Insightec’s Exablate Neuro platform for staged bilateral pallidothalamic tractotomy treatment in patients living with advanced Parkinson's disease (PD). The device previously received approval for unilateral treatment of medication-refractory essential tremor in 2016, tremor-dominant PD in 2018, and unilateral
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
Devices
An FDA Early Alert says the agency is aware that Abiomed has issued a letter to affected customers recommending that all Automated Impella Controllers have updated instructions for use. The alert says users should be aware that the controller may not detect an Impella pump when connected. It says they
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