Biologics
FDA has extended until the fourth quarter the review of two Regeneron Pharmaceuticals regulatory submissions for Eylea HD (aflibercept) injection 8 mg regulatory submissions because information submitted to address a recent inspection was deemed a major amendment. One submission was a chemistry, manufacturing and controls prior-approval supplement for the
Drugs
B. Braun Medical has recalled (Class 1) one lot each of its Lactated Ringers Injection USP 1,000 mL and 0.9% sodium chloride injection USP 1,000 mL due to the presence of particulate matter inside the containers. B. Braun has received several complaints and identified the potential for
Drugs
A 2/24-2/28 FDA inspection at the Anhui Hanbon Daily Chemical Co., Ltd., drug manufacturing facility in Huainan Shi, Anhui Sheng, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/11 Warning Letter says the specific violations were: * failure to test
FDA Policy/General
FDA has extended by 90 days its review of a Regenxbio’s BLA for RGX-121 (clemidsogene lanparvovec), an investigational gene therapy for Hunter syndrome, a rare and life-threatening genetic disorder. The agency’s extension pushes back the user fee review action target date from 11/9 to 2/
Drugs
FDA has issued PTC Therapeutics a complete response letter (CRL) on its NDA for vatiquinone and its use for treating Friedreich’s ataxia, calling for additional evidence of the drug’s effectiveness. The CRL said PTC’s submission did not demonstrate “substantial evidence of efficacy” and that another well-controlled
Drugs
FDA has placed a clinical hold on a BiomX Phase 2b trial of BX004, an investigational phage therapy for cystic fibrosis (CF). The agency action does not relate to the BX004 drug itself, but instead concerns a third-party nebulizer device used to deliver the treatment, the company says. BiomX
Biologics
Viking Therapeutics is reporting mixed data from a Phase 2 study for its oral obesity drug VK2735, with patients losing up to 12.2% of body weight over 13 weeks. Despite the encouraging efficacy, shares of the biotech tumbled more than 35% at one point as investors focused on high
Devices
FDA has published a guidance, Marketing Submission Recommendations for a Predetermined Change Control Plan for Artificial Intelligence-Enabled Device Software Functions, recommending that such a plan describe the planned artificial intelligence-enabled device software function (AI-DSF) modifications, the associated methodology to develop, validate, and implement those modifications, and an
Drugs
FDA has denied a 12/4/2022 petition submitted by Ferring Pharmaceuticals asking the agency to reconsider its denial of a 2/10/2022 petition asking that FDA require ANDA sponsors citing Ferring’s Firmagon (degarelix) as the reference-listed drug to conduct in vivo bioequivalence studies and update the
Drugs
FDA has published a guidance, Emerging Scientific and Technical Information on Leachable NDBA and Other Small-Molecule Nitrosamines in Infusion Bags, asking manufacturers and applicants to obtain better data and applicants to report data in their applications so the agency can better understand the emerging issue of the detection of
Devices
FDA has cleared a NeuroOne Medical Technologies 510(k) to market its OneRF Trigeminal Nerve Ablation System for use in procedures to create radiofrequency lesions for treating pain, or for lesioning nerve tissue for functional neurosurgical procedures. “This clearance leverages the company’s proprietary OneRF Ablation System RF generator platform
Devices
A 3/14-4/1 FDA inspection at Les Encres LLC in Lenoir City, TN, found multiple violations in the firm’s work as a specification developer of Les Encres absorbable polydioxanone surgical suture devices. A just-released 7/30 Warning Letter says Quality System regulation violations were: * failure to
Drugs
FDA has granted Altimmune a fast track designation for pemvidutide and its use for treating alcohol use disorder. The company is currently evaluating pemvidutide in the Phase 2 RECLAIM trial. The primary endpoint is a reduction in the number of heavy drinking days, with secondary measures including improvements in World
Drugs
FDA has released a new draft guidance entitled Oncology Therapeutic Radiopharmaceuticals (RPTs): Dosage Optimization During Clinical Development, highlighting the need for more tailored strategies to determine safe and effective dosages. RPTs are drugs that deliver radiation systemically to treat cancer and share properties of both external beam radiation therapy (EBRT)
Drugs
Stealth BioTherapeutics has resubmitted its NDA for elamipretide for treating Barth syndrome, a rare and life-threatening mitochondrial disease. The filing follows an FDA complete response letter (CRL) in May, which requested additional information but did not require new clinical efficacy data, according to the company. It says the submission
Drugs
The U.S. Court of Appeals for the DC Circuit has ruled that FDA improperly rejected Vanda Pharmaceuticals’ request for a hearing on its supplemental NDA that was previously under consideration for sleep drug Hetlioz (tasimelteon) and its proposed use for jet lag In February, the agency denied a Vanda
FDA Policy/General
Conflicts of interest among federal vaccine advisors had fallen to historic lows well before HHS secretary Robert F. Kennedy Jr. abruptly dismissed the Centers for Disease Control and Prevention’s vaccine advisory committee earlier this summer, according to new research from the USC Schaeffer Center for Health Policy & Economics.
Devices
Tandem Diabetes Care has recalled (Class 1 device correction) for certain t:slim X2 insulin pumps to address a potential speaker-related issue that can trigger an error resulting in a discontinuation of insulin delivery. “The error, which appears as a Malfunction 16 alarm to the user, will stop insulin
Drugs
FDA is announcing a two-day (9/18-19) virtual public workshop to address methodological challenges in collecting and using patient experience data to inform drug development and regulatory decision-making. The “Patient-Focused Drug Development: Workshop #2” is the second in a series mandated by the user fee program
Drugs
FDA has granted Novo Nordisk accelerated approval for a new Wegovy (semaglutide) indication for treating metabolic dysfunction-associated steatohepatitis (MASH) in adults with moderate to advanced liver scarring (fibrosis), but not with cirrhosis of the liver, in conjunction with a reduced calorie diet and increased physical activity, according to the
Drugs
FDA has approved a Tonix Pharmaceuticals NDA for Tonmya (cyclobenzaprine HCl sublingual tablets), a once-daily, non-opioid drug for treating fibromyalgia in adults. Tonmya is taken at bedtime and uses a sublingual formulation designed for rapid absorption. In clinical trials, the drug was shown to significantly reduce daily pain
Drugs
FDA has extended until 10/3 its review of a clinical hold response from Neurizon Therapeutics’ NUZ-001, its lead experimental treatment for amyotrophic lateral sclerosis (ALS). The agency’s standard review period for such responses is 30 days, but Neurizon said the delay stems from FDA-wide staffing cuts