Drugs
FDA has granted breakthrough therapy designation to Vertex Pharmaceuticals’ VX-548 to treat acute and neuropathic pain. The company says it has reached agreement with FDA on the design of a pivotal Phase 3 trial that will open in the fourth quarter of this year. The Phase 3 program will
Drugs
An 11/29-12/13/21 FDA inspection at the Goodman Eye Center, San Francisco, CA, conducted as part of the agency’s Bioresearch Monitoring Program, found objectionable conditions associated with a clinical trial conducted at the site. A 7/14 Warning Letter emphasized these issues: * failing to submit an
Drugs
FDA has published a guidance, General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products, to assist sponsors of INDs, NDAs, and BLAs and their supplements who are planning to conduct clinical studies in neonatal populations. The document has recommendations for neonatal clinical pharmacology studies, whether they are
Drugs
The Department of Justice has opened a criminal investigation into whether Cassava Sciences manipulated research results for its experimental Alzheimer’s drug simufilam, Reuters reports exclusively, based on information from two sources. The news service says it was told those doing the investigation specialize in examining whether companies or individuals
Drugs
FDA has published a guidance, Cancer Clinical Trial Eligibility Criteria: Available Therapy in Non-Curative Settings, with recommendations for clinical investigators and sponsors about including patients who have not received available therapy for their cancer in clinical trials of drugs and biological products for the treatment of cancer in the
Drugs
FDA has approved an additional indication for GlaxoSmithKline’s Benlysta (belimumab) to treat children age 5 to 17 with active lupus nephritis who are receiving standard therapy. The company says the approval extends the current U.S. indication to include both lupus and active lupus nephritis for the intravenous formulation
Drugs
A 13/15/21-1/14/22 FDA inspection at the Bi-Coastal Pharma International drug manufacturing facility in Shrewsbury, NJ, found significant violations of current good manufacturing practice regulations for finished drugs. A 6/30 Warning Letter says specific violations were: * failing to establish and follow adequate written procedures
Drugs
A 1/10-1/21 FDA inspection at the Jost Chemical drug manufacturing facility in St. Louis, MO, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (APIs). A 7/18 Warning Letter says specific deviations were: * failure of the quality unit to ensure that
Drugs
Immuron says FDA has placed a clinical hold on an unidentified investigational drug being evaluated for treating campylobacter and enterotoxigenic escherichia coli. The Australian biopharmaceutical company, which is focused on developing and commercializing oral immunotherapeutic products for preventing and treating gut pathogens, says FDA has given feedback to the U.
Drugs
Avadel CNS Pharmaceuticals is suing FDA and HHS over an agency decision to require a patent statement for the company’s Lumryz NDA. The suit, filed in DC federal court, gives four reasons why FDA erred in requiring Avadel to file the statement for its REMS computerized system that FDA
Drugs
FDA has accepted and granted priority review to a Biogen NDA for tofersen, an investigational new drug intended to treat superoxide dismutase 1 amyotrophic lateral sclerosis. The agency set a PDUFA action date of 1/25/23 for the NDA. Biogen says it was informed that FDA plans to schedule
Drugs
FDA has granted a Taiho Oncology 2/10 petition asking that the agency not permit ANDA applicants citing Lonsurf (trifluridine and tipiracil) as the reference-listed drug to omit certain labeling information describing pharmacokinetics in and dosage reduction instructions for patients with severe renal impairment and other information from Taiho’
Drugs
Researchers from Cambridge Health Alliance and the University of Mississippi say that after FDA issued Warning Letters targeting specific dietary supplement products, 29% remained available for purchase, with most containing a prohibited ingredient. The researchers looked at FDA Warning Letters targeting the presence of BMPEA or DMHA in 31 supplements.
Drugs
Genentech says it is happy to see an FDA flexible approach to meeting the requirements for substantial evidence of effectiveness when faced with clinical trial challenges and the potential need for concomitant antibacterial drug therapy. The company commented on an FDA draft guidance Antibacterial Therapies for Patients With an Unmet
Drugs
Medexus Pharmaceuticals says its medac licensor has resubmitted its NDA for treosulfan, indicated for combination use with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT). The current submission includes updates to data files and supporting information in response to an FDA information request in May
Drugs
FDA has made available a draft guidance entitled “Real-Time Oncology Review (RTOR).” The review program was rolled out 2/2018 to promote earlier submission of topline results (i.e., efficacy and safety results from clinical studies before the study report is completed) and datasets, after database lock, to support
Drugs
FDA has accepted for priority review a Daiichi Sankyo and AstraZeneca supplemental BLA for Enhertu (fam-trastuzumab deruxtecan-nxki) for treating adult patients with unresectable or metastatic HER2 low breast cancer who have received a prior therapy in the metastatic setting. The agency has set a user fee review action
Drugs
FDA has released a draft guidance on the expanded use of remote regulatory assessments (RRAs) beyond the Covid-19 pandemic to assess an FDA-regulated establishment and its records to determine compliance in lieu of conducting a physical inspection. The agency notes that RRAs will be an additional regulatory tool
Drugs
CDER director Patrizia Cavazzoni says the potential use of “circulating tumor DNA (ctDNA)” as a potential endpoint to support an accelerated approval is very promising, but it requires more data to validate the biomarker for further regulatory uses. Speaking at a 7/19 Friends of Cancer Research fireside chat on
Drugs
FDA has posted a final guidance entitled “Failure to Respond to an ANDA Complete Response Letter Within the Regulatory Timeframe.” The document provides recommendations on actions an ANDA applicant can take after the issuance of a complete response letter (CRL) as well as the actions that FDA may take if
Drugs
FDA has issued a final guidance entitled “Orange Book Questions and Answers” that answers commonly asked questions it has received from interested parties. It covers topics such as the content and format of the Orange Book, petitioned ANDAs, drug products moving between the Active and Discontinued sections, and drug product
Drugs
FDA has made available a proposed rule on Revising the National Drug Code Format and Drug Label Barcode Requirements (Docket No. FDA-2021-N-1351) that outlines the agency’s adoption of 12-digit format for national drug codes (NDCs). The agency says it is running out of 10-digit