Drugs
FDA reviewers will ask an advisory committee 10/31 whether Vertex Pharmaceuticals and CRISPR Therapeutics need to conduct additional studies on their sickle cell disease (SCD) gene therapy exagamglogene autotemcel (exa-cel) to obtain a better safety assessment. According to briefing documents released 10/27 in advance of the meeting,
Drugs
Senator Marco Rubio (R-FL) has introduced a bill entitled Further Strengthening America’s Supply Chains and National Security Act to reduce America’s dependence on China for pharmaceuticals and to address related supply chain risks that lead to shortages of critical drugs. “The COVID-19 pandemic highlighted how vulnerable
Drugs
FDA has approved a Coherus BioSciences and Junshi Biosciences BLA for Loqtorzi (toripalimab-tpzi) in combination with cisplatin and gemcitabine for the first-line treatment of adults with metastatic or recurrent locally advanced nasopharyngeal carcinoma (NPC), and as monotherapy for treating adults with recurrent, unresectable, or metastatic NPC with disease
Drugs
FDA is seeking public comment on the usefulness of biosimilarity statements in the product labeling. In an online post, CDER Office of Therapeutic Biologics and Biosimilars director Sarah Yim says FDA is recommending that all labeling for biosimilars, including interchangeable biosimilars, include a biosimilarity statement, rather than both a biosimilarity
Drugs
FDA is advising healthcare providers and the public about the possible risks that probiotic products containing live bacteria or yeast pose to hospitalized preterm infants. An agency news release (contains links to related materials) says the agency is concerned that such products are being illegally sold to treat or prevent
Drugs
CDER’s Office of Prescription Drug Promotion (OPDP) has launched a new Web page to display untitled letters issued for drug advertising and promotion violations. The Web page consolidates all OPDP untitled letters issued since 2017 onto a single page. Previously, they were displayed on separate Web pages based on
Drugs
Three former senior Intarcia Therapeutics employees who were closely involved in the development of the company’s ITCA 650 (exenatide in DUROS device), indicated for improving glycemic control in adults with Type 2 diabetes, say CDER made “erroneous statements” in a “misleading” presentation to the 9/21 meeting of the
Drugs
FDA has published a draft guidance, Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities, describing how the agency requests and conducts voluntary remote interactive evaluations at facilities where drugs are manufactured, processed, packed, compounded, or held, and at drug facilities covered under the agency’s bioresearch monitoring program.
Drugs
A Florida public broadcasting station says documents filed by state officials in a Florida federal court indicate it is unlikely FDA will meet its previously stated intention to rule by 10/31 on a Florida proposal to import some drugs from Canada for use in government programs such as Medicaid,
Drugs
FDA has accepted for review an Akebia Therapeutics NDA resubmission for vadadustat as a treatment for anemia due to chronic kidney disease in adult patients on dialysis. The agency has classified the submission as a Class 2 response and set a 3/27/2024 review action target date. A 3/
Drugs
BioXcel Therapeutics says an independent third-party audit of data integrity at one TRANQUILITY II Phase 3 trial site of the company’s BXCL501 for the acute treatment of agitation associated with dementia reported positive findings. “The team of auditors did not identify any findings that they believe impact the
Drugs
FDA commissioner Robert Califf posted a positive online post-trip report on his recent official visit to India. He says he made the trip “to strengthen our collective efforts to advance the development and availability of medical products that both our countries and the rest of the world need.” While
Drugs
FDA has published a draft guidance, Topical Dermatologic Corticosteroids: In Vivo Bioequivalence, to assist applicants who submit ANDAs for the products. The document has recommendations for in vivo studies to demonstrate the bioequivalence of topical corticosteroids. When finalized, the draft will replace a 6/1995 guidance, FDA says. It says
Drugs
FDA has released the form FDA-483 issued following a 6/26-7/4 inspection at UCB Farchim SA, a drug substance and bulk drug product manufacturer in Bulle, Fribourg, Switzerland. The four inspection observations were: * quality unit oversight over quality control operations is deficient, including laboratory and production electronic
Drugs
Attorney Dara Katcher Levy (Hyman, Phelps & McNamara) says an FDA 10/23 draft guidance on communications from firms to healthcare providers about scientific information on unapproved uses of approved/cleared medical products represents a shift in the agency’s approach. Writing in her firm’s online FDA Law Blog,
Drugs
FDA has accepted for review an AstraZeneca supplemental BLA that seeks approval for a self- or caregiver-administered option for FluMist Quadrivalent, a needle-free nasal spray. The submission is based on data from a usability study that the company says “confirmed that individuals over 18 years of age could
Drugs
FDA has cleared an NKGen Biotech IND for its SNK01 NK cell therapy to treat Alzheimer’s disease. The company says SNK01 is an autologous, non-genetically modified NK cell product that has enhanced cytotoxicity and activating receptor expression. An approved trial of 30 patients with moderate Alzheimer’s disease
Drugs
FDA has granted Shanghai-based Zhimeng Biopharma an orphan drug designation for its small-molecule KCNQ2/3 selective opener compound (CB03) for treating amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease). The company says a Phase 1 study evaluating the safety, tolerability, and pharmacokinetics of CB03 in
Drugs
FDA has granted Genentech accelerated approval for an expanded use for Rozlytrek (entrectinib) in treating pediatric patients older than one month with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result
Drugs
FDA has approved a new indication for Servier Pharmaceuticals’ Tibsovo (ivosidenib) to treat adults with relapsed or refractory (R/R) myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH-1) mutation as detected by an FDA-approved test. An agency statement says this is the first targeted therapy approved for
Drugs
FDA has released a revised https://www.fda.gov/regulatory-information/search-fda-guidance-documents/communications-firms-health-care-providers-regarding-scientific-information-unapproved-uses entitled “Communications From Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products — Questions and Answers.” The documents is
Drugs
FDA has issued Panacea Biotec Pharma a nine-item Form FDA-483 after inspecting the firm’s Himachal Pradesh, India manufacturing 10/3-12 and documenting GMP deficiencies. The company says that the inspection’s findings are “related to improvements in existing procedures and are addressable.” According to the FDA-