Drugs
FDA has deferred action on an Amicus Therapeutics BLA for cipaglucosidase alfa, the biological component of AT-GAA in late-onset Pompe disease, due to the agency’s inability to conduct a required manufacturing site inspection in China before the PDUFA action date. A company announcement says the inability to
Drugs
Pfizer says it will file by year end a BLA for its investigational bivalent respiratory syncytial virus (RSV) vaccine after stopping enrollment in a Phase 3 study that it says showed favorable results. In the study, the vaccine was administered to pregnant participants to protect their infants from RSV after
Drugs
FDA has released a final guidance, “S1B(R1) Addendum to S1B Testing for Carcinogenicity of Pharmaceuticals; International Council for Harmonization; Guidance for Industry,” which outlines a “weight of evidence” approach for assessing human carcinogenic risk in drugs. The International Council for Harmonization document is part of FDA’s and other
Drugs
FDA has released a draft guidance entitled “Measuring Growth and Evaluating Pubertal Development in Pediatric Clinical Trials.” The document provides recommendations on the most appropriate methods for measuring and recording growth and evaluating pubertal development for evaluating safety. The agency notes that it does not address use of growth or
Drugs
Bristol Myers Squibb (BMS) has reported that its COMMANDS Phase 3 study evaluating Reblozyl (luspatercept-aamt) met its primary endpoint in first-line treatment of anemia in adult patients with myelodysplastic syndromes who require red blood cell transfusions. The endpoint was the significant and clinically meaningful improvement in red blood
Drugs
FDA is relaxing advertising and promotion restrictions placed on six Covid-19 emergency use authorizations (EUA) after determining that “there may be benefit in clarifying the condition” for “EUA sponsors that wish to include in promotional materials information about the safety and efficacy data that supported the EUA’s issuance.
Drugs
FDA has posted a seven-item Form FDA-483 from an inspection of New England Life Care (dba Advanced Compounding Solutions) earlier this year. During the inspection, the agency cited the firm’s failure to thoroughly review any unexplained discrepancy and the failure of a batch or any of its
Drugs
FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 10/26 to recommend approval of GlaxoSmithKline’s (GSK) anemia drug daprodustat for use in treating chronic kidney disease (CKD) in adult patients on dialysis, but it did not support approval in those who are not on dialysis. Panel members voted
Drugs
CDER director Patrizia Cavazzoni says Don Ashley is retiring after more than 29 years in federal service, including the last six years as director of CDER’s Office of Compliance (OC). Cavazzoni says Ashley transformed OC into a higher-performing organization with a well-defined strategic focus and wide-ranging
Drugs
FDA principal deputy commissioner Janet Woodcock told a Council for Federal Cannabis Regulation Webinar that the agency is exploring what flexibilities it might have as part of President Biden’s order for a marijuana scheduling review. But Woodcock cautioned that while FDA will lead the scientific and medical evaluation of
Drugs
FDA has made available a draft guidance entitled “Clostridioides difficile Infection (CDI): Developing Drugs for Treatment, Reduction of Recurrence, and Prevention.” The document says clinical trials for drugs to treat CDI should be randomized, double-blinded, and controlled. “Sponsors should use an active control in trials for CDI treatment and
Drugs
Three FDA officials say the agency is continuing to work with stakeholders to advance treatment options for children. Writing in an FDA Voice post, the three say FDA is working to: * improve the timeliness of pediatric medical product development; * engage patients and their families in clinical research; and * collaborate with
Drugs
FDA medical reviewers say they are unable to establish a substantive risk/benefit relationship for using Y-mAbs Therapeutics’ I-omburtamab in pediatric patients with CNS/leptomeningeal metastases from neuroblastoma. The reviewers raised their concerns in a briefing paper released in advance of a 10/28 meeting of the Oncologic
Drugs
GSK has abandoned plans to seek FDA approval for otilimab, an investigational monoclonal antibody targeting granulocyte-macrophage colony-stimulating factor (GM-CSF) as a treatment for moderate to severe rheumatoid arthritis (RA) in patients who did not respond to earlier treatments. The company says it based its decision on data
Drugs
FDA has published the Fiscal Year 2023 GDUFA Science and Research Priority Initiatives that were developed in collaboration with industry and the public. The document lists eight areas encompassing scientific challenges that the generic drug industry and the FDA generic drug program identified as being significant over the next five
Drugs
FDA has postponed an 11/18 joint meeting of its Nonprescription Drugs Advisory Committee and Obstetrics, Reproductive and Urologic Drugs Advisory Committee. Panel members were scheduled to discuss a Laboratoire HRA Pharma supplemental NDA for Opill (norgestrel, 0.075 mg) and a proposed indication for a nonprescription use as a
Drugs
FDA has accepted for priority review a Seres Therapeutics BLA for its investigational oral microbiome therapeutic SER-109 for preventing recurrent C. difficile infection (rCDI). The agency has set a 4/26/2023 user fee review action target date. SER-109 comprises purified Firmicutes spores for treating rCDI, which the
Drugs
The International Council for Harmonization (ICH) has released a presentation outlining its finalized guidance entitled “A Selective Approach to Safety Data Collection in Specific Late-Stage Pre-Approval or Post-Approval Clinical Trials (E19).” The guideline provides recommendations on the use of selective safety data collection that may be applied
Drugs
AstraZeneca has reported positive high-level results from the SERENA-2 Phase 2 trial and the CAPitello-291 Phase 3 trial of new breast cancer drugs. The company says the SERENA-2 trial showed that AZ’s next-generation oral selective estrogen receptor degrader camizestrant met the primary endpoint of
Drugs
A 3/28-4/20 FDA inspection at the Nephron SC drug manufacturing facility in West Columbia, SC, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 10/11 Warning Letter says the firm also failed to submit an NDA Field Alert Report to the
Drugs
FDA says it is postponing a 10/31 Endocrinologic and Metabolic Drugs Advisory Committee meeting that was scheduled to review and vote on an Ipsen Biopharmaceuticals NDA for palovarotene capsules, indicated for preventing heterotopic ossification in adults and children with fibrodysplasia ossificans progressiva (see earlier announcement). The meeting is being
Drugs
A 4/21-4/27 FDA inspection at the Advanced Cosmetic Research Laboratories drug manufacturing facility in Chatsworth, CA, found significant violations of current good manufacturing practice (CGMP) regulations. A 9/26 Warning Letter says the agency has not received a detailed response from the firm for corrective actions to