Biologics
FDA has granted Pfizer traditional approval for its previously accelerated-approved Tivdak (tisotumab vedotin-tftv) for recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. Approval was based on efficacy data from the innovaTV 301 (NCT04697628), an open-label, active-controlled trial that enrolled 502 patients with
Biologics
FDA has released a draft guidance entitled “Considerations for the Use of Human- and Animal-Derived Materials in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products.” The document says that the use of human- and animal-derived materials to manufacture cellular and gene therapy products and
Biologics
FDA has posted a draft guidance entitled “Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products.” The document provides recommendations for determining the appropriate cell safety testing to support a BLA or IND. “Cell safety testing should be based on a risk analysis that considers
Biologics
The top priorities at CBER this year include updating the Center’s tissue guidance to reduce the risk of infectious disease transmission, applying the accelerated approval pathway to rare disease gene therapies, providing general guidance on platform technologies and a guidance on platform technologies specifically for genome editing, according to
Biologics
FDA has accepted for priority review a GSK supplemental BLA for Jemperli (dostarlimab) for combination use with standard-of-care chemotherapy (carboplatin and paclitaxel) to expand treatment to all adult patients with primary advanced or recurrent endometrial cancer, including those with mismatch repair proficient/microsatellite stable tumors. Currently, the drug
Biologics
FDA has issued Abeona Therapeutics a complete response letter (CRL) on a a BLA for prademagene zamikeracel (pz-cel), requesting additional information to satisfy chemistry manufacturing and controls (CMC) requirements before the submission can be approved. The submission seeks the therapy’s approval for use in treating patients with recessive
Biologics
FDA has approved Alvotech and Teva Pharmaceuticals’ Selarsdi (ustekinumab-aekn) injection for subcutaneous use, a biosimilar to Johnson & Johnson’s Stelara, for treating moderate to severe plaque psoriasis and for active psoriatic arthritis in adults and pediatric patients six years and older. “Being able to develop the biosimilar in
Biologics
FDA has accepted for review a GSK BLA for its 5-in-1 meningococcal ABCWY (MenABCWY) vaccine candidate. The user fee review action target date is 2/14/2025. The vaccine combines the antigenic components of the company’s two established meningococcal vaccines — Bexsero (meningococcal Group B vaccine) and Menveo
Biologics
Enlivex Therapeutics says there were mixed results from its Phase 2 study of Allocetra in patients with sepsis, in which 120 patients enrolled. The company says it is developing Allocetra as a universal, off-the-shelf cell therapy designed to reprogram macrophages into their homeostatic state. “Diseases such as solid
Biologics
Four Hogan Lovells attorneys say a discussion paper published by CBER, CDER, CDRH, and the Office of Combination Products on artificial intelligence (AI) and medical products aims to provide greater transparency on how the agency’s Centers are collaborating to safeguard public health while fostering responsible and ethical innovation. The
Biologics
Gritstone Bio reports mixed data from an ongoing, signal-seeking Phase 2 portion of a Phase 2/3 study evaluating Granite, a personalized neoantigen cancer vaccine for front-line use in metastatic microsatellite-stable colorectal cancer (MSS-CRC). “Circulating tumor DNA (ctDNA) analysis over several months of treatment shows the
Biologics
Eisai has submitted a supplemental BLA for monthly Leqembi (lecanemab-irmb) intravenous maintenance dosing in Alzheimer’s disease (AD) patients with mild cognitive impairment or mild dementia stage of disease (early AD). The submission is based on modeling of observed data from the Phase 2 study (Study 201) and its
Biologics
FDA has approved a Medexus Pharmaceuticals supplemental BLA for Ixinity [coagulation factor IX (recombinant)] for the on-demand, prophylactic, and perioperative treatment of pediatric patients under 12 years of age with hemophilia B. With this approval, the intravenous recombinant factor IX therapeutic is now approved for use in all patients
Biologics
The Biotechnology Innovation Organization (BIO) says an FDA 2023 draft guidance on potency assurance for cellular and gene therapy (CGT) products “addresses a wide range of CGT product development considerations related to potency assurance. The scope helpfully includes both investigational and licensed products, and the different phases of development.” BIO
Biologics
Mesoblast says it will refile next quarter its BLA for remestemcel-L for treating pediatric steroid-refractory acute graft versus host disease (SR-aGVHD) following favorable guidance from FDA regarding the company’s clinical data. On 8/4/2023, the agency issued a second complete response letter that requested more
Biologics
FDA has issued Regeneron Pharmaceuticals two complete response letters (CRLs) on its BLA for odronextamab in relapsed/refractory (R/R) follicular lymphoma (FL) and in R/R diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. “The only approvability issue is related to the enrollment
Biologics
CBER has launched a Web page describing its approach to artificial intelligence and machine learning (AI/ML) for biological and other products regulated by the Center. The page says the Center has participated in public workshops and discussions organized by FDA and other national and international organizations to better understand
Biologics
FDA has approved an Orchard Therapeutics BLA for Lenmeldy (atidarsagene autotemcel), a one-time, single-dose gene therapy indicated for treating children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. “It
Biologics
Six House members from California and Arizona, five Republicans and one Democrat, are asking FDA commissioner Robert Califf to “reconsider expediently the addition of coccidioidomycosis, also known as Valley Fever, to the current list of tropical diseases that qualify for a priority review voucher as directed in House Report 117-
Biologics
FDA has approved Hugel’s Letybo (botulinum toxin), a neurotoxin for treating moderate-to-severe glabellar (frown) lines in adults. Approval was based on data from three Phase 3 trials that enrolled more than 1,000 subjects in the U.S. and Europe (BLESS I NCT02677298, BLESS II NCT02677805 and
Biologics
CBER has cited Oregon Medical Centers in an untitled letter over claims it is making on social media sites Facebook and Youtube about its cellular product derived from a human umbilical cord. The letter takes issue with the medical facility referring to its product as “regenerative medicine,” “regenerative cells,” “regenerative
Biologics
FDA is affirming its regulation of “fresh allograft heart valves” in a just-posted letter to healthcare professionals. The letter addresses recent questions the agency has received about the regulatory status of the allograft heart valves used in a procedure commonly referred to as “partial heart transplantation,” which involves transplantation