Drugs
FDA says it is launching the Commissioner’s National Priority Voucher (CNPV) program under which drug developers may redeem vouchers to participate in an agency program that shortens the review time from about 10-12 months to 1-2 months following submission of a final drug application. An FDA notice
Drugs
Aldeyra Therapeutics has resubmitted an NDA for reproxalap to treat the signs and symptoms of dry eye disease. The submission addresses an agency complete response letter sent in April that said the NDA “failed to demonstrate efficacy in adequate and well-controlled studies in treating ocular symptoms associated with dry
Drugs
A 1/16-1/24 FDA inspection at Zhejiang Huahai Pharmaceutical Co., Ltd., in Zhejiang Province, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 6/6 Warning Letter says the specific violations were: * failure to clean, maintain, and, as appropriate for the nature
Drugs
The HHS Office of Inspector General (OIG) semiannual report covering the period 10/1/2024 to 3/31 says the OIG concluded that FDA’s use of its accelerated approval pathway raised concerns in three of 24 drugs reviewed. The report says OIG identified instances in which FDA (1) approved
Drugs
A ProPublica investigative report cites at least 20 instances in which FDA identified significant problems at drug manufacturing firms in India, imposed import bans on the firm’s products, and then secretly allowed the companies to continue to ship drugs to the U.S. Focusing initially on Sun Pharma, the
Drugs
Sarepta Therapeutics says it has stopped shipping its gene therapy Elevidys (delandistrogene moxeparvovec‑rokl) to non‑ambulatory Duchenne muscular dystrophy (DMD) patients and paused dosing in a key confirmatory trial after a second recipient died from acute liver failure. Both fatal cases involved non‑ambulatory individuals with DMD, the company
Drugs
A 12/5-12/11/2024 FDA inspection at the Kenil Healthcare Private Limited drug manufacturing facility in Gujarat, India, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 6/12 Warning Letter says the specific violations were: * failure to clean, maintain, and, as appropriate
Drugs
FDA has approved updated labeling on Pfizer’s s Talzenna (talazoparib) in combination with Xtandi (enzalutamide) for men with metastatic castration-resistant prostate cancer (mCRPC). “The FDA approved updated labeling with the inclusion of final overall survival (OS) data for the combination’s existing indication for the treatment of adults
Drugs
Roche says it is moving its Parkinson’s drug prasinezumab into a Phase 3 trial after two mid‑stage trials and their open‑label extensions hinted at a disease‑slowing effect in early‑stage disease. Prasinezumab, a monoclonal antibody, is designed to target aggregated alpha-synuclein. A 2022 Phase 2
Drugs
Milestone Pharmaceuticals has submitted its response to a March-issued FDA complete response letter on the company’s NDA for Cardamyst (etripamil) nasal spray and its use to convert acute episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults. PSVT is described as a sudden, rapid, and irregular
Drugs
Compliance and product testing firm Qalitex Laboratories has issued a warning to dietary supplement and wellness product sellers on Amazon, highlighting a growing overlap between FDA’s enforcement and Amazon’s compliance policies. The industry advisory outlines how Amazon has increasingly aligned its internal compliance standards with FDA regulations — a
Drugs
AbbVie says its Phase 3 VERONA clinical trial evaluating venetoclax in combination with azacitidine for newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) did not meet the primary endpoint of extending overall survival. The trial showed a hazard ratio of 0.908 and a p-value of 0.3772, indicating
Drugs
Incyte is reporting the first clinical data from two studies evaluating the safety, tolerability and efficacy of INCA033989, a novel, first-in-class targeted monoclonal antibody in patients with mutant calreticulin (mutCALR)-expressing myeloproliferative neoplasms (MPNs). The studies evaluated the safety and efficacy of INCA033989 in patients with essential thrombocythemia
Drugs
FDA has told KalVista Pharmaceuticals that it will not meet a 6/17 user fee review action target data for its NDA for sebetralstat, indicated for treating hereditary angioedema (HAE). The agency said the goal date would not be met due to heavy workload and limited resources, according to the
Drugs
FDA has approved Merck’s Keytruda (pembrolizumab) as the first anti‑PD‑1 therapy that can be given both before and after surgery for adults with resectable, locally advanced head and neck squamous‑cell carcinoma whose tumors express PD‑L1. The approval allows the drug to be used as a
Drugs
FDA has placed a clinical hold on a Rein Therapeutics Phase 2 (RENEW) trial of LTI-03 to request additional non-clinical information. The company says LTI-03 is a novel, multi-pathway Caveolin-1-related peptide being developed for treating patients with idiopathic pulmonary fibrosis (IPF). “Importantly, to date
Drugs
FDA has released a final guidance entitled “ANDAs: Pre-Submission Facility Correspondence (PFC) Related to Prioritized Generic Drug Submissions.” The document describes and provides recommendations on the content, timing, and assessment of a PFC for a priority ANDA. It also provides information regarding FDA’s assessment process for a PFC.
Drugs
A recent FDA pre-license inspection of Capricor Therapeutics’ San Diego manufacturing facility for Deramiocel, the company’s lead cell therapy candidate with a BLA under agency review for its use in treating Duchenne Muscular Dystrophy (DMD) concluded with a Form 483 containing several observations, the company says, adding that
Drugs
FDA has approved a UroGen Pharma NDA for Zusduri (mitomycin intravesical solution) for adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. Approval was based on data from the ENVISION trial (NCT05243550) that evaluated patients’ complete response (CR) at three months and duration of response (DoR)
Drugs
FDA has granted Sumitomo Pharma America a fast track designation for nuvisertib (TP-3654) and its use in treating patients with intermediate or high-risk myelofibrosis (MF). The company describes nuvisertib as an oral, highly selective inhibitor of PIM1 kinase, which demonstrated clinical activity, including symptom and spleen responses correlating
Drugs
A 1/7-1/10 FDA inspection at the Amish Origins Management drug manufacturing facility in Worland, WY, found significant violations of current good manufacturing practice (CGMP) regulations in its manufacturing of unapproved and misbranded external analgesic and sunscreen products. A 5/28 Warning Letter says specific CGMP violations were:
Drugs
FDA has published reports on the real-world evidence submissions to CBER and CDER in FY 2024, as required under the PDUFA VII reauthorization. The summary report (includes links to the CBER and CDER statistics pages) says the reporting intends to describe submissions to FDA containing real-world data to