Biologics
FDA has accepted for priority review a Bristol Myers Squibb supplemental BLA for Breyanzi (lisocabtagene maraleucel) to expand its current indication to include treating adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma who received a prior Bruton tyrosine kinase inhibitor and B-cell lymphoma 2
Biologics
FDA has approved Takeda’s Adzynma, a recombinant protein product designed for prophylactic or on‑demand enzyme replacement therapy (ERT) in both adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) — a rare life-threatening blood clotting disorder. cTTP is linked to a disease-causing mutation in the ADAMTS13
Biologics
CBER director Peter Marks and researchers from three academic medical centers say that registries used in the U.S. and several other countries to track the use of Covid-19 convalescent plasma (CP) showed that CP was effective when used early and with high titer. Writing in an mBio editorial,
Biologics
The International Society for Cell & Gene Therapy (ISCT) says an FDA draft guidance on manufacturing changes and comparability for human cellular and gene therapy products “could benefit from more detailed information as it covers a very broad range of therapeutic products.” The ISCT comment letter says the analytical approach
Biologics
A BMJ investigation by senior editor Peter Doshi explores the “revolving door” between FDA and its regulated industries as shown in the case of two senior CBER Office of Vaccine Research and Review officials who now work for Moderna. One of the officials, Doran Fink, had an active public government
Biologics
FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee supported Vertex Pharmaceuticals and CRISPR Therapeutics safety assessments on their sickle cell disease (SCD) gene therapy exagamglogene autotemcel (exa-cel). This moves the therapy one step closer to FDA approval which would make it the first CRISPR gene-edited therapy for
Biologics
FDA has approved a Tenaya Therapeutics IND for the company to begin a Phase 1b trial of TN-401, an adeno-associated virus serotype 9 (AAV9)-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the plakophilin-2 (PKP2) gene.
Biologics
FDA has approved an Amgen BLA for Wezlana (ustekinumab-auub), an interchangeable biosimilar to Janssen’s Stelara for multiple inflammatory diseases. Both products are indicated for adults and pediatric patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy, and active psoriatic arthritis. They are
Biologics
Pfizer and BioNTech have announced “positive topline results” from their Phase 1/2 study (NCT05596734) that is evaluating the safety, tolerability and immunogenicity of mRNA-based combination vaccine candidates for influenza and Covid-19 among healthy adults 18 to 64 years of age. “The data from the trial showed that
Biologics
GSK says it will seek FDA approval for an expanded use of Arexvy (respiratory syncytial virus vaccine, adjuvanted) in adults aged 50 to 59, including those at increased risk of respiratory syncytial virus (RSV) lower respiratory tract disease (LRTD) due to certain underlying medical conditions. The submission will be based
Biologics
FDA has approved a Celltrion BLA for Zymfentra (infliximab-dyyb) for maintenance therapy in adults with moderately to severely active ulcerative colitis (UC) and Crohn’s disease (CD) following treatment with an infliximab product administered intravenously. Zymfentra is a subcutaneous version of Celltrion’s infliximab (Janssen’s Remicade) biosimilar, which
Biologics
ImmunityBio has resubmitted its BLA for Anktiva (N-803) in combination with Bacillus Calmette-Guérin (BCG) for treating patients with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (CIS) with or without Ta or T1 disease. The original BLA was issued a complete response letter in May
Biologics
FDA has issued Sanofi and Regeneron a complete response letter on their supplemental BLA for Dupixent (dupilumab) and its use in chronic spontaneous urticaria, described by the companies as an inflammatory skin condition, which causes sudden, debilitating hives and swelling on the skin. The letter indicates that “additional efficacy data
Biologics
FDA has published a revised guidance entitled “Development and Licensure of Vaccines to Prevent Covid-19.” It updates the guidance under the same name that was released 8/2020. The document is intended to assist the agency and sponsors in the clinical development and licensure of vaccines for preventing Covid.
Biologics
FDA has published a guidance, Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies, describing a CBER program to identify and recognize voluntary consensus standards (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products when such standards are appropriate. The document says CBER encourages the use
Biologics
BrainStorm Cell Therapeutics says it is withdrawing its BLA for NurOwn to treat amyotrophic lateral sclerosis (ALS), a move it says was coordinated with FDA. A company statement says the agency invited BrainStorm to request an expedited face-to-face meeting to discuss a path forward for NurOwn. BrainStorm says
Biologics
FDA has approved a UCB BLA for Bimzelx (bimekizumab-bkzx) for treating moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. The company says the therapy is designed to selectively inhibit two key cytokines driving inflammatory processes – interleukin 17A and interleukin 17F. The approval
Biologics
FDA has granted Diakonos Oncology a fast-track designation for the company’s dendritic cell vaccine, DOC1021, for use in patients with glioblastoma multiforme (GBM). The designation was based on “positive preliminary safety and efficacy data” from a Phase 1 clinical trial in GBM patients, the company says. “Because Phase
Biologics
FDA has issued Alvotech a complete response letter for its BLA for AVT04, a biosimilar of Janssen’s Stelara (ustekinumab). The letter cited “certain deficiencies” that were documented during an FDA March inspection of the company’s Reykjavik facility in Iceland, an Alvotech release says. “No other deficiencies in the
Biologics
Eli Lilly says it will seek FDA approval in 2024 for mirikizumab after reporting the drug met the co-primary and all major secondary endpoints compared to placebo in VIVID-1, a Phase 3 study in adults with moderately to severely active Crohn’s disease. In the study, a higher
Biologics
FDA has granted MimiVax a fast track designation for its SurVaxM vaccine that is being studied for treating newly diagnosed glioblastoma (nGBM). The designation was based on data from the company’s Phase 2a Study of SurVaxM for nGBM that found 51% of patients receiving SurVaxM survived at least two
Biologics
A Tufts Medical Center for Biomedical System Design analysis finds that durable cell and gene therapy (CGT) programs for rare orphan conditions and CAR-T/TCR therapies for blood cancers have higher success once they enter the clinic compared to the combined pipeline of all therapies. A Center newsletter says