Biologics
bluebird bio has submitted for priority review a BLA for lovotibeglogene autotemcel (lovo-cel), a gene therapy for patients with sickle cell disease ages 12 years and older who have a history of vaso-occlusive events. The submission is based on data from 36 patients in the HGB-206 Group
Biologics
FDA has amended the emergency use authorizations for the Moderna and Pfizer/BioNTech Covid-19 bivalent mRNA vaccines to simplify the vaccination schedule for most individuals and permit a second booster for certain at-risk populations. An agency notice lists these key changes: * most individuals, depending on age, previously vaccinated
Biologics
Federal Register notice: FDA is making available a revised draft guidance entitled “Assessing the Irritation and Sensitization Potential of Transdermal and Topical Delivery Systems for ANDAs.” The document provides recommendations for the design and conduct of studies to evaluate the in vivo skin irritation and sensitization potential of a proposed
Biologics
Researchers from the University of Rochester and around the world who are members of the RENOIR Clinical Trial Group say a Phase 3 trial of Pfizer’s investigational bivalent RSV (respiratory syncytial virus) prefusion F protein-based (RSVpreF) vaccine prevented RSV-associated lower respiratory tract illness and RSV-associated acute
Biologics
CBER’s Office of Compliance and Biologics Quality has issued an untitled letter to Fidia Pharma USA after reviewing the company’s Web sites and determining that its cell/tissue products require a BLA or NDA. The product, NuDYN, is derived from amniotic membrane and marketed for treating various orthopedic
Biologics
CBER director Peter Marks says he hopes to align global regulations to boost commercialization for gene and cell therapies so more treatments are launched for patients with rare diseases. Marks spoke on 3/29 at the Reuters Pharma USA conference. Health regulators including FDA and the European Medicines Agency have
Biologics
Janssen Pharmaceutical says that due to business concerns it is scrapping development of an investigational respiratory syncytial virus adult vaccine and discontinuing the related Phase 3 EVERGREEN study. The decision, it says, was made after the company reviewed its “portfolio to prioritize the most transformational assets for ongoing investment, and
Biologics
Reps. Brett Guthrie (R-KY) and Anna Eshoo (D-CA), the chair and ranking member of the House Energy and Commerce Committee’s Health Subcommittee, have written to CBER director Peter Marks expressing concern about an increase in the number of clinical holds on BLAs for cell and gene therapies.
Biologics
U.S. Representatives Mariannette Miller-Meeks (R-IA), Greg Murphy (R-NC), Nanette Barragán (D-CA), and Annie Kuster (D-NH) have introduced the Biologics Competition Act, a bipartisan bill that would order HHS to evaluate the process by which interchangeable biological products are approved and designated for substitution purposes
Biologics
BrainStorm Cell Therapeutics says it has refiled a BLA under the agency’s “file-over-protest procedure” for NurOwn, a stem cell therapy for treating amyotrophic lateral sclerosis (ALS and also known as Lou Gehrig’s Disease). On 11/10/2022, the agency issued a refusal to file letter for
Biologics
Iovance Biotherapeutics has completed a rolling BLA submission for lifileucel, a tumor infiltrating lymphocyte (TIL) therapy for treating patients with advanced (unresectable or metastatic) melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy. The company says it is seeking an accelerated approval for the
Biologics
CBER’s Office of Therapeutic Products is hosting a 4/27 virtual public listening meeting to obtain input on methods and approaches (e.g., use of real-world evidence, registries) for capturing post-approval safety and efficacy data for cell and gene therapy products. “The rapid pace of innovation for
Biologics
The Government Accountability Office (GAO) has published its review of the regenerative medicine and advanced therapies workforce in the commercial and academic sectors. The report was mandated by the Timely ReAuthorization of Necessary Stem-cell Programs Lends Access to Needed Therapies (TRANSPLANT) Act of 2021. The report has information on
Biologics
CBER says there were 15,010 biological product deviation reports submitted in FY 2022, a 3% increase over the FY 2021 total of 14,556 reports. The Center’s FY 22 annual summary says the total number of reporting establishments increased from 2,152 in FY 21 to 2,276
Biologics
FDA has approved an expanded indication for Regeneron Pharmaceuticals’ Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). The therapy was originally approved 2/2021 as an adjunct to other lipid-lowering therapies in those
Biologics
FDA has approved a Sandoz supplemental BLA for citrate-free Hyrimoz (adalimumab-adaz) injection, a biosimilar version of Abbvie’s Humira, indicated for treating rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis and plaque psoriasis. The original Hyrimoz BLA was approved in 2018. With
Biologics
Speaking on 3/20 to the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, CBER director Peter Marks discussed three ways FDA is working to overcome barriers to developing gene therapies for rare diseases. In a video summary of his keynote address posted by Neurology Live, Marks said FDA is
Biologics
FDA has told Sarepta Therapeutics that it has decided to hold an advisory committee meeting to review its gene therapy BLA for SRP-9001 (delandistrogene moxeparvovec), indicated for treating Duchenne muscular dystrophy. Previously when the submission was accepted for review, the agency said that an advisory committee meeting was not
Biologics
An Eastern PA federal judge has approved a $25 million class action settlement involving Johnson & Johnson’s Remicade (infliximab). The lawsuit (In re Remicade Antitrust Litigation, No. 2:17-cv-04326-KSM) claimed that defendants Johnson & Johnson and Janssen Biotech violated federal and state antitrust and consumer-protection
Biologics
GlaxoSmithKline says its meningococcal ABCWY vaccine candidate met all 11 primary endpoints in a pivotal Phase 3 trial and was well tolerated with a safety profile consistent with the company’s Bexsero and Menveo meningococcal vaccines. A company statement says the primary endpoint data demonstrated non-inferiority compared to Bexsero
Biologics
FDA has published an International Pharmaceutical Regulators Program (IPRP) reflection paper, General Considerations for Raw Materials Used in the Manufacture of Human Cell and Gene Therapy Products, to provide the perspectives of the IPRP cell therapy working group and gene therapy working group on the quality of raw materials used
Biologics
FDA has revised the fact sheets on Janssen’s Covid-19 vaccine to include a warning about reported adverse events that suggest increased risks of myocarditis and pericarditis, particularly within the first seven days after administration. “An additional revision to the fact sheets was made to include that facial paralysis