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FDA has released a new draft guidance entitled Oncology Therapeutic Radiopharmaceuticals (RPTs): Dosage Optimization During Clinical Development, highlighting the need for more tailored strategies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
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
Drugs
FDA has approved a Precigen BLA for Papzimeos (zopapogene imadenovec-drba), which the agency describes as a “first-of-its-kind non-replicating adenoviral vector-based immunotherapy for the treatment of adult patients with recurrent respiratory papillomatosis (RRP).” The rare chronic disease is caused by persistent human papillomavirus (HPV) 6
Drugs
Pfizer says a Phase 3 study of its investigational sickle cell disease drug inclacumab failed to meet the primary endpoint. THRIVE-131 is evaluating inclacumab, a P-selectin inhibitor, in 241 patients aged 16 and older with sickle cell disease who experienced between two and 10 vaso-occlusive crises (VOCs)
Drugs
In an 8/14 open letter to HHS secretary Robert F. Kennedy, Jr. and FDA commissioner Martin Makary, the Multidisciplinary Association for Psychedelic Studies (MAPS) is calling for approval for MDMA-assisted therapy for those living with treatment-resistant trauma. The letter notes that one year ago, FDA issued a
Drugs
President Donald Trump has issued an Executive Order calling for actions to replenish the Strategic Active Pharmaceutical Ingredients Reserve (SAPIR) that was created during his first term and then, he says, was depleted by the Biden administration’s lack of effort to ensure domestic sources for essential medicines. The order
Drugs
Schrödinger has discontinued the clinical development program for SGR-2921, its CDC7 inhibitor being evaluated in a Phase 1 dose-escalation study in patients with relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes. “Despite early evidence of monotherapy activity observed in the Phase 1 study, based on the
Drugs
FDA has granted Halda Therapeutics a Fast Track designation for HLD-0915, which the company describes as a drug candidate for treating metastatic castration-resistant prostate cancer (mCRPC). In preclinical prostate cancer models, the therapy showed tumor shrinkage, prostate-specific antigen declines, and activity in drug-resistant disease, it says.
Drugs
An 8/12 petition submitted by Foley & Lardner on behalf of a client asks FDA to not approve any ANDA submission for a generic version of Nova Nordisk’s Ozempic (semaglutide injection) unless specified conditions are met. The petition says FDA should ensure that: * the generic drug-device product
Drugs
FDA has published a CDER Manual of Policies and Procedures, Equal Voice Collaboration and Regulatory and Policy Decision-Making in CDER, describing the principles behind the Center’s Equal Voice and how the process is used for collaborative review and regulatory and policy decision-making. The MAPP says CDER uses
Drugs
Texas attorney general Ken Paxton has filed suit against Eli Lilly, charging that the company bribed and illegally induced healthcare providers to prescribe 14 of its most profitable drugs, including the GLP-1 medications Mounjaro and Zepbound (both tirzepatide). A statement (contains a link to the suit) from Paxton’s
Drugs
The Justice Department says two men have been charged as part of its investigation into illegal importation and distribution of misbranded sildenafil, an active ingredient in FDA-approved erectile dysfunction drugs. A department statement says that Jitender Behl of East Greenwich, RI, imported hundreds of thousands of pills from India
Drugs
Ocular Therapeutix says that it has received a written agreement from FDA regarding its registrational trial design under a Special Protocol Assessment for evaluating Axpaxli in the treatment of non-proliferative diabetic retinopathy (NPDR). “This formal agreement ensures direct FDA alignment with our proposed approach for NPDR and provides us
Drugs
FDA is announcing a 12/5 Public Workshop on “Pediatric Drug Development Safety and New Approach Methodologies” to discuss new approach methods that can address pediatric developmental safety during drug development. FDA says the meeting comes at a critical time for pediatric drug development. “An accumulation of knowledge from pediatric
Drugs
HHS officials say the agency is investing in On Demand Pharmaceuticals’ Pharmacy on Demand modular manufacturing unit to make the U.S. the most competitive nation in the world for drug manufacturing. An agency statement describes an 8/13 visit made by HHS to On Demand Pharmaceuticals’ new centralized production
Drugs
FDA has issued a Manual of Policies and Procedures (MAPP) detailing how the CDER Office of Pharmaceutical Quality (OPQ) will facilitate and expedite the development, time to submission, assessment, and marketing approval of NDAs and BLAs for products that address unmet medical needs. The document helps fulfill agency commitments on