Drugs
UCB is announcing positive data from a Phase 3 clinical trial evaluating fenfluramine as an adjunctive treatment for CDKL5 Deficiency Disorder (CDD). The trial met both its primary and key secondary endpoints in patients with the rare and severe form of epilepsy, according to the company. The primary outcome was
Drugs
The U.S. Court of Appeals for the D.C. Circuit has upheld a lower court’s decision for FDA that a “clinically superior drug is not the same as a drug that is otherwise the same.” The appeals court reaffirmed that FDA acted correctly in determining that Avadel CNS
Drugs
Achieve Life Sciences has submitted an NDA for cytisinicline for treating nicotine dependence as part of smoking cessation in adults. The submission is based on data from two placebo-controlled Phase 3 trials, ORCA-2 and ORCA-3. “In both studies, cytisinicline administered for either six or 12 weeks, alongside
Drugs
FDA has issued safety-related labeling updates for two bile acid transport inhibitors — Mirum Pharmaceuticals’ Livmarli (maralixibat) and Ipsen Biopharmaceuticals’ Bylvay (odevixibat) — to emphasize increased risks of liver injury and bleeding associated with their use. The changes are based on new data from expanded clinical trials and postmarketing experience, an
Drugs
FDA has published a guidance, Antibacterial Therapies for Patients with an Unmet Medical Need for the Treatment of Serious Bacterial Diseases—Questions and Answers, to help sponsors in the clinical development of new antibacterial drugs to treat serious bacterial diseases, particularly those caused by antimicrobial drug-resistant organisms, in patients
Drugs
Altimmune is announcing mixed topline results today from its Phase 2b IMPACT trial evaluating pemvidutide in patients with metabolic dysfunction-associated steatohepatitis (MASH), a progressive liver disease. The study showed that pemvidutide led to significant improvements in disease resolution and weight loss, with promising signals of anti-fibrotic activity, according
Devices
FDA has posted a draft guidance entitled “Unique Device Identifier (UDI) Requirements for Combination Products.” The document discusses how the agency’s UDI requirements apply to combination products with device constituent parts. It also outlines requirements, recommendations, and best practices for UDI labeling and submission of information to the Global
Biologics
Former CBER director Peter Marks says the FDA announcement of a new “evidence-based approach to Covid-19 vaccination” is a “dramatic departure” not just from previous agency vaccine policies but also from the deliberate and measured way in which the agency previously made such policy, with open public discussion
Drugs
FDA has published a guidance, Conducting Remote Regulatory Assessments—Questions and Answers, to help enhance the industry’s understanding of such assessments, thus facilitating the process for FDA conducting the assessments. The document says the term Remote Regulatory Assessment (RRA) is used to describe a category of activities for which
Devices
FDA has released a final guidance entitled Cybersecurity in Medical Devices: Quality System Considerations and Content of Premarket Submissions. The document applies to devices with cybersecurity considerations, including but not limited to those that have a device software function or that contain software (including firmware) or programmable logic, and it
Drugs
FDA has granted regenerative medicine advanced therapy designation for enGene Holdings’ detalimogene voraplasmid, the company’s lead investigational therapy to treat high-risk, Bacillus Calmette-Guérin-unresponsive, non-muscle invasive bladder cancer with carcinoma in situ. A company statement describes detalimogene as a novel non-viral gene-based immunotherapy for
Drugs
Viatris says it will submit an NDA later this year for its MR-141 (phentolamine ophthalmic solution 0.75%) for treating presbyopia based on just-released data from the Phase 3 VEGA-3 trial. Presbyopia is a progressive vision condition affecting older adults that reduces their ability to focus on
Drugs
A 3/4-3/6 FDA inspection at the Sterling Distributors wholesale distribution facility in Coral Springs, FL, found significant violations of Drug Supply Chain Security Act (DSCSA) requirements. A 6/5 Warning Letter says the specific violations were: * engaging in wholesale distribution of prescription drugs without being appropriately licensed;
Drugs
FDA has released a final guidance entitled “Early Lyme Disease as Manifested by Erythema Migrans: Developing Drugs for Treatment.” The document provides FDA’s current recommendations regarding the development of drugs to support an indication for the treatment of early Lyme disease as manifested by erythema migrans (EM). The guidance
Drugs
FDA has approved an updated label for Eli Lilly’s Amyvid (florbetapir F 18 injection) for brain imaging to estimate amyloid plaque density in patients with cognitive impairment being evaluated for Alzheimer’s disease and other causes of cognitive decline. The label update includes: * Revising how Amyvid can be used
Biologics
FDA has required and approved updates to the prescribing information for Pfizer’s Comirnaty Covid-19 mRNA vaccine and Moderna’s Spikevax Covid-19 mRNA vaccine to include new safety information about the risks of myocarditis and pericarditis following their administration. The warning on myocarditis and pericarditis in the vaccines’
Drugs
The staff of the online newsletter Global Biodefense raises a concern that FDA’s recent action stopping clinical trials that involve shipping participants’ biological samples to laboratories in China and other “hostile” countries can cripple biomedical innovation and progress in the U.S. “The stated concern is that overseas sample
Drugs
FDA has granted Syndax Pharmaceuticals a priority review of its supplemental NDA for Revuforj (revumenib) as a treatment for relapsed or refractory (R/R) acute myeloid leukemia (AML) with mutant NPM1 (mNPM1) — the most common genetic alteration found in adult AML patients. The agency has set a 10/25 user
FDA Policy/General
Former Rep. John Doolittle (R-CA) says the restructuring underway at FDA, while well-intentioned, risks “undermining the very innovation and domestic capacity the president seeks to promote.” Writing for The Hill, Doolittle, who served on committees with FDA oversight, says the elimination of 3,500 full-time FDA employees,
Devices
FDA has approved an InspireMD PMA for its CGuard Prime Carotid Stent System for treating carotid artery stenosis. The device is described as a novel mesh-covered stent designed to improve patient safety through sustained embolic protection. The device’s approval is supported by data from the company’s C-
Devices
Cook Medical has recalled (Class 1) its Beacon Tip 5.0 Fr Angiographic Catheter because it may experience tip separation. The issue was identified through field complaints, with users reporting tip separation occurring both prior to and during patient contact, a recall notice says. “If an affected product is used,
Drugs
Nektar Therapeutics is reporting promising results from a mid-stage clinical trial of its experimental therapy rezpegaldesleukin for patients with moderate-to-severe atopic dermatitis. The therapy met all primary and key secondary endpoints in a 16-week analysis of the global Phase 2b REZOLVE-AD study, it says. The