Drugs
FDA has accepted for review a supplemental NDA for Nucala (mepolizumab) and its use as an add-on maintenance treatment for patients with chronic obstructive pulmonary disease with an eosinophilic phenotype. The agency has set a user fee review action target date of 5/7/2025. The monoclonal antibody was
Drugs
FDA has approved a Cumberland Pharmaceuticals supplemental NDA for a simplified dosing regimen of its Acetadote (N-acetylcysteine for injection) product. Acetadote is an intravenous formulation of N-acetylcysteine indicated to prevent or lessen liver injury after ingestion of potentially toxic quantities of acetaminophen. “The newly approved dosing regimen simplifies
Drugs
Merck says the Phase 3 KEYLYNK-001 trial evaluating Keytruda (pembrolizumab) plus chemotherapy followed by maintenance with Lynparza (olaparib), with or without bevacizumab, as a first-line treatment for people with BRCA non-mutated advanced epithelial ovarian cancer met its primary endpoint of progression-free survival. However, the company says
Drugs
Agios says it has filed a supplemental NDA for an expanded indication for its Pyrukynd (mitapivat) for treating adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The pyruvate kinase activator is currently approved for treating hemolytic anemia in adults with pyruvate kinase deficiency. The submission
Drugs
AbbVie is announcing “positive topline results” from its pivotal Phase 3 TEMPO-2 trial evaluating tavapadon as a flexible-dose monotherapy in early Parkinson's disease. The investigational D1/D5 dopamine receptor partial agonist is being evaluated as a once-daily treatment for Parkinson’s. Based on the results,
Drugs
Chimerix says that based on FDA feedback it plans to submit an NDA before the end of the year seeking accelerated approval for dordaviprone for treating recurrent H3 K27M-mutant diffuse gliomas. The aggressive and potentially lethal gliomas affect over 2,000 patients annually in the U.S, according to
Drugs
FDA has posted an 11-item Form FDA-483 from a 10/2023 Fresenius Kabi Oncology inspection at the company’s Pradesh, India manufacturing facility. Significant GMP deficiencies cited in the form are: * Procedures designed to prevent microbiological contamination of drug products purporting to be sterile are not established and
Drugs
FDA has released a final guidance entitled “Standardized Format for Electronic Submission of NDA and BLA Content for the Planning of Bioresearch Monitoring (BIMO) Inspections for CDER Submissions.” The document describes the electronic submission of certain data and information in standardized formats, which would include all pivotal studies used to
Drugs
Dozens of patient advocacy groups have signed a letter urging congressional leaders to pass before the end of the 118th Congress the Creating Hope Reauthorization Act (H.R. 7384/S. 4583), which would reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program for at least five years. Noting that
Drugs
FDA has published a draft guidance, Expedited Program for Serious Conditions—Accelerated Approval of Drugs and Biologics, with information on the agency’s policies and procedures for accelerated approval as well as threshold criteria generally applicable to concluding that a drug is a candidate for accelerated approval. The document also
Drugs
FDA has approved AstraZeneca’s Imfinzi (durvalumab) to treat adults with limited-stage small cell lung cancer whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy. A company statement says Imfinzi is the only approved immunotherapy regimen for that cancer diagnosis. The approval was based on
Drugs
Rayner Surgical is petitioning FDA to apply specified restrictions on any ANDA referencing or relying on Omidria (phenylephrine and ketorolac injection) 1%/3%. The 11/27 petition specifically asks that the agency: * decline to accept or approve any ANDA referencing Omidria for a proposed generic product that lacks a buffer;
Drugs
Protara Therapeutics is reporting encouraging data from the Phase 2 open-label ADVANCED-2 trial of its cell-based TARA-002 in high-risk non-muscle invasive bladder cancer patients with carcinoma in situ who are Bacillus Calmette-Guérin (BCG)-unresponsive or BCG-naïve. “The complete response (CR) rate across
Drugs
FDA has granted EpicentRx a fast-track designation for its AdAPT-001 and its combination use with checkpoint inhibitors nivolumab or atezolizumab in treating recurrent or refractory advanced or metastatic soft tissue sarcoma (STS) with disease progression after at least one prior line of therapy. The designation was based on
Drugs
FDA has granted Merus accelerated approval for Bizengri (zenocutuzumab-zbco) for adults with advanced, unresectable, or metastatic non-small cell lung cancer (NSCLC) and pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. Last month, FDA extended by three months its
Drugs
Researchers at FDA’s National Center for Toxicological Research (NCTR) have found that drug interactions with UDP-glucuronosyltransferase (UGT) enzymes are a strong predictor of drug-induced liver injury (DILI) across different drug types. An agency statement says NCTR conducted the study with scientists from Elsevier’s Life Sciences. “This
Drugs
DifGen Pharmaceuticals is petitioning FDA to not approve an ANDA for prednisolone ophthalmic solution 1% without the inclusion of long-term stability studies conducted under refrigerated conditions. The 11/26 petition specifically asks the agency to: * require generic applicants to conduct long-term stability studies that specifically demonstrate the product’
Drugs
A 4/1-4/5 FDA Bioresearch Monitoring Program inspection at a clinical trial site operated by neurologist Han Phan in Atlanta, GA, found that she did not adhere to applicable laws and regulations governing the conduct of clinical investigations. A 12/3 Warning Letter says Phan failed to ensure
Drugs
Eli Lilly’s weight-loss drug Zepbound (tirzepatide) demonstrated superiority over Novo Nordisk’s Wegovy (semaglutide) in the head-to-head SURMOUNT-5 phase 3b clinical trial, according to the company. Over 72 weeks, participants taking Zepbound saw an average weight loss of 20.2%, outperforming the 13.7% weight
Drugs
FDA has granted Stoke Therapeutics a breakthrough therapy designation for zorevunersen and its use in treating Dravet syndrome with a confirmed mutation in the SCN1A gene. The designation is based on clinical data from the Phase 1/2a and open-label extension studies, which demonstrated zorevunersen’s “substantial and sustained
Drugs
An FDA review of records submitted by Zhejiang Uniquality Nursing Products Technology Co., Ltd., doing business as Zhejiang YouQuan Care Products in Zhejiang Sheng, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 11/12 Warning Letter says specific violations were: * failure
Drugs
Roivant says its Kinevant Sciences unit is discontinuing its development of namilumab for treating sarcoidosis after a Phase 2 study failed to show treatment benefit. The Phase 2 RESOLVE-Lung study (NCT05314517) “failed to meet the primary endpoint of proportion of subjects with a rescue event during the double-blind