Biologics
FDA has approved a bluebird bio BLA for its lentiviral vector gene therapy Zynteglo (betibeglogene autotemcel) (beti-cel) for beta-thalassemia. The agency says Zynteglo is the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. The inherited
Biologics
Brainstorm Cell Therapeutics says it will submit a BLA for it NurOwn stem cell therapy for treating amyotrophic lateral sclerosis (ALS and also known as Lou Gehrig’s Disease). Last year, FDA released an unprecedented notice (see story) saying that despite high hopes in the ALS community for NurOwn, Phase
Biologics
Gilead Sciences has filed a supplemental BLA for Trodelvy (sacituzumab govitecan-hziy) in patients with HR+/HER2-metastatic breast cancer who received prior endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy. The submission includes just-announced “statistically significant and clinically meaningful results from the second interim
Biologics
Novartis says two children died of acute liver failure weeks after being administered Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy indicated for treating patients under age 2 with spinal muscular atrophy. The therapy currently carries a Boxed Warning for acute liver failure. The deaths were first reported by Stat. “While
Biologics
FDA has granted Daiichi Sankyo accelerated approval for Enhertu (fam-trastuzumab deruxtecan-nxki) for treating adult patients with unresectable or metastatic non-small cell lung cancer whose tumors have activating human epidermal growth factor receptor 2 HER2 mutations (as detected by an FDA-approved test). The agency also approved Life
Biologics
An FDA Bioresearch Monitoring Program 8/26/21-10/8/21 inspection at Valley Biosystems, West Sacramento, CA, found “objectionable conditions” in the firm’s conduct of two nonclinical Good Laboratory Practice studies. An 8/3 Warning Letter says specific violations were: * failing to ensure that each individual engaged in
Biologics
FDA has issued Bavarian Nordic an emergency use authorization (EUA) for its smallpox/monkeypox vaccine Jynneos to allow healthcare providers to administer the vaccine by intradermal injection for individuals 18 years of age and older who are determined to be at high risk for monkeypox infection. The agency says allowing
Biologics
FDA has accepted for priority review a Gamida Cell BLA for omidubicel for treating patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. Omidubicel is described as a first-in-class, advanced NAM-enabled stem cell therapy candidate. The agency has set a user fee review
Biologics
FDA has accepted for review an ImmunityBio BLA for its antibody cytokine fusion protein as a treatment for patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer with or without Ta or T1 disease. The agency has set a 5/23/2023 user fee review action target
Biologics
FDA has lifted a clinical hold on Celyad Oncology’s CYAD-101-002 (KEYNOTE-B79) Phase 1b trial after the company modified the eligibility criteria for the trial. In March, the company said the agency issued the hold due to insufficient information to assess the risk to study subjects. At
Biologics
FDA has approved Bavarian Nordic’s fill and finish facility in Denmark to manufacture its Jynneos smallpox/monkeypox vaccine. “With the approvals of the final drug production – the process by which the vaccine is formulated and filled into vials – the company is now allowed to deliver drug product manufactured at
Biologics
Three people who work in the artificial cell industry say FDA has not yet begun to develop a regulatory framework for artificial cell technologies. “Such lack of preparation for what could be the next forefront of medicine could largely delay the development of these therapies,” the three write in a
Biologics
FDA has accepted for review a Sandoz BLA for what the company describes as the first biosimilar copy of Biogen’s Tysabri (natalizumab). The submission seeks approval for all indications of the reference product, which include relapsing forms of multiple sclerosis including clinically isolated syndrome, relapsing-remitting MS, active secondary
Biologics
Keynoting the International Society for Pharmaceutical Engineering 2022 Biotechnology Conference, CBER director Peter Marks said regulatory pathways and review were not the biggest rate-limiting factor in Covid-19 vaccine development. Rather, he said, the real bottleneck was process development and manufacturing scaleup, as reported by Bioprocess Online. Under Operation
Biologics
The U.S. CDC Advisory Committee on Immunization Practices 7/19 voted unanimously to recommend the use of the Novavax Covid-19 Vaccine, Adjuvanted as a two-dose primary series in individuals aged 18 and older. This follows last week’s FDA emergency use authorization (see earlier story). The company
Biologics
FDA has completed an inspection in Denmark of Bavarian Nordic’s monkeypox vaccine manufacturing plant, a step that will lead to the release of thousands of doses for shipping to the U.S., according to an AP report. A recent investigative article in New York Magazine criticized the agency’s
Biologics
FDA has granted an emergency use authorization for Novavax’s Covid-19 Vaccine, Adjuvanted for preventing disease caused by severe acute respiratory syndrome coronavirus 2 in individuals 18 years of age and older. This is the fourth Covid vaccine permitted for use in the U.S., joining vaccines from Pfizer/
Biologics
FDA has accepted for review a Byondis BLA for SYD985 ([Vic-] trastuzumab duocarmazine) for use in patients with HER2-positive unresectable locally advanced or metastatic breast cancer (MBC). The agency has set a user fee review action target date of 5/12/2023. The submission is based on data from
Biologics
An investigative article in New York Magazine says the lack of monkeypox vaccines in New York City, where cases have risen 370% in the last two weeks, is due in part to a slow FDA response to the need to inspect a vaccine manufacturing facility in Denmark. “As monkeypox cases
Biologics
Genmab says it will submit in the second half of this year a BLA for subcutaneous epcoritamab, an investigational bispecific antibody for treating patients with relapsed/refractory large B-cell lymphoma. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells and induces T
Biologics
FDA has told Covid-19 vaccine manufacturers seeking to update their vaccines that they should develop modified vaccines that add an omicron BA.4/5 spike protein component to the current vaccine composition to create a two-component (bivalent) booster vaccine. This follows advice from the agency’s Vaccines and
Biologics
FDA has extended by three months the review of a Provention Bio BLA for teplizumab, indicated for delaying progression to Stage 3 clinical Type 1 diabetes in at-risk individuals. The extended user fee review action goal date is 11/7. The agency recently told the company that its response