Biologics
Constantly changing Covid-19 transmissible variants present a “major challenge” for current Covid vaccines, and continuing to develop variant-specific vaccine boosters is “inadequate as a long-term strategy for addressing Covid-19 in populations globally,” writes CDER director Peter Marks and two researchers from Brown University in a 1/
Biologics
FDA has extended by three months a Krystal Biotech BLA for B-Vec (beremagene geperpavec), a gene therapy indicated for treating patients with dystrophic epidermolysis bullosa (DEB), after it submitted manufacturing data pursuant to an agency information request. The topical gene therapy is designed to treat DEB at the molecular
Biologics
BioMarin Pharmaceutical is submitting to FDA additional long-term data (three years) as part of the ongoing BLA review for Roctavian (valoctocogene roxaparvovec), an investigational one-time gene therapy for treating adults with severe hemophilia A. The data submitted show the mean annualized bleed rate reduced by 80% from baseline
Biologics
CDER director Patrizia Cavazzoni is attempting to restore credibility within her Center by announcing the accelerated approval for a Biogen/Eisai BLA for Leqembi (lecanemab-irmb) for treating Alzheimer’s disease (AD), while also addressing previous controversies with the 2021 approval of Biogen’s Aduhelm (aducanumab). Leqembi is the second
Biologics
An FDA update says it is closely monitoring the emergence of the XBB.1.5 Omicron subvariant that is currently estimated to account for 28% of circulating Covid-19 variants in the U.S. “Because of its similarity to variants that are not neutralized by [AstraZeneca’s] Evusheld (e.g.
Biologics
Advocacy group Public Citizen is calling on FDA to postpone a 1/6-expected accelerated approval decision on Biogen/Eisai’s Alzheimer’s drug lecanemab until after an advisory committee reviews the data and votes on its risk-benefit profile. In a 1/5 letter from Public Citizen’s Health
Biologics
FDA has approved a Ferring Pharmaceuticals BLA for Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating adenoviral vector‑based gene therapy indicated for treating adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Approval
Biologics
FDA says it is holding a 1/26/2023 meeting of its Vaccines and Related Biological Products Advisory Committee to consider “whether and how the composition for primary doses of the currently available Covid-19 vaccines should be modified and how and whether the composition and schedule for booster doses
Biologics
Three Ropes & Gray attorneys say a potential split between the 9th and 11th Circuit Courts of Appeals over stem cell therapy regulation could “fundamentally alter FDA’s ability to regulate stem cell therapies.” Writing in an online Alert, the attorneys outline the two cases that are setting up the
Biologics
Moderna and Merck say their mRNA-4157/V940 investigational personalized mRNA cancer vaccine in combination with Keytruda (pembrolizumab) met its primary endpoint in the Phase 2b KEYNOTE-942 trial. A joint announcement says the vaccination demonstrated a significant and clinically meaningful reduction in the risk of disease recurrence or death
Biologics
Janssen Pharmaceutical has submitted a BLA for talquetamab for treating patients with relapsed or refractory multiple myeloma. The bispecific T-cell engager antibody is designed to target both GPRC5D, a novel drug target that is on some normal cells but overexpressed on myeloma cells, and CD3 on T cells, according
Biologics
FDA has amended the emergency use authorizations of the updated (bivalent) Moderna and Pfizer-BioNTech Covid-19 vaccines for use in children as young as six months. An FDA release says that the Moderna bivalent vaccine is now authorized for use in children aged six months through five years as
Biologics
A 2/9-3/8 FDA inspection at Cryos International in Orlando, FL, identified significant deviations from the regulations for human cells, tissues, and cellular and tissue-based products. A just-released 10/24 Warning Letter says specific violations were: * failing to test a specimen from an anonymous semen donor
Biologics
FDA is defending criticism (see earlier story) from three former FDA Team Biologics inspectors who left the agency over working conditions and a lack of training, which they claim leaves the team with inspectors ill-equipped to identify manufacturing quality control issues. The inspectors were granted anonymity for a lengthy
Biologics
Three former FDA Team Biologics inspectors who left the agency in recent years due to frustration over working conditions, say needed training for team members has decreased dramatically in recent years, leading to concerns that inspectors are not equipped to identify manufacturing quality control issues. The inspectors were granted anonymity
Biologics
Pfizer and BioNTech have submitted an application seeking an emergency use authorization for their Omicron BA.4/BA.5-adapted bivalent Covid-19 vaccine as a third dose in the primary series for protecting children aged six months through four years of age. If authorized, eligible children would receive a
Biologics
FDA has issued Y-mAbs Therapeutics a complete response letter on its BLA for I-omburtamab and its use in treating CNS/leptomeningeal metastasis from neuroblastoma, a serious condition in which cancer cells spread from the original tumor to the meninges (the thin layers of tissue that cover and protect
Biologics
FDA has granted in part and denied in part a 7/2/20 petition submitted by the Microbiome Therapeutic Innovation Group (MTIG) asking the agency to take certain actions regarding fecal microbiota for transplantation (FMT) to treat Clostridium difficile infection. The agency’s 11/29 response says MTIG sought these
Biologics
Janssen has filed a patent infringement suit in Delaware federal court against Amgen and its plans to seek FDA approval and market a biosimilar copy of Janssen’s blockbuster Stelara (ustekinumab), indicated for treating plaque psoriasis, psoriatic arthritis, Crohn’s disease, and ulcerative colitis. Earlier this month, Amgen provided Janssen
Biologics
FDA has accepted for review a Sarepta Therapeutics BLA that is seeking accelerated approval for SRP-9001 (delandistrogene moxeparvovec) for treating ambulant individuals with Duchenne muscular dystrophy (DMD). The priority review submission has been given a user fee review action date of 5/29/2023. SRP-9001 is an investigational
Biologics
Questions are being raised about Eisai and Biogen’s lecanemab (BAN2401), an investigational anti-amyloid beta protofibril antibody for treating Alzheimer’s disease after a second death was reported in a patient taking the therapy. The journal Science is reporting that a 65-year-old woman who was receiving the
Biologics
FDA has told BioMarin Pharmaceutical that it no longer plans to hold an advisory committee meeting to review the company’s resubmitted BLA for Roctavian (valoctocogene roxaparvovec AAV gene therapy), indicated for adults with severe hemophilia A. The agency’s decision comes on the heels of its request earlier this