Biologics
FDA is adding a Boxed Warning to Amgen’s osteoporosis drug Prolia (denosumab) about increased risk of severe hypocalcemia in patients with advanced chronic kidney disease (CKD), especially those on dialysis. “Severe hypocalcemia appears to be more common in patients with CKD who also have a condition known as mineral
Biologics
FDA has approved a Vertex Pharmaceuticals BLA for Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy for treating transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. This is the second approval granted for Casgevy; the first approval was granted 12/9/2023 for
Biologics
The DC federal court has approved an FDA request to stay its response to a Children’s Health Defense Freedom of Information Act (FOIA) request for records connected with the safety monitoring of Covid-19 vaccines through the VAERS (Vaccine Adverse Event Reporting System) database. But it cut the requested
Biologics
Anti-vaxxers and those opting to shun vaccines over safety concerns or religious beliefs are threatening U.S. public health by risking “population immunity” against some vaccine-preventable infectious diseases. Writing in a 1/5 JAMA Viewpoint online article, FDA commissioner Robert Califf and CBER director Peter Marks sound the
Biologics
FDA has issued a “ Draft Report and Plan on Best Practices for Guidance.” In the document, the agency identifies the best practices for the efficient prioritization, development, review, clearance, issuance, and use of guidance documents. The agency says it is also considering opportunities to further improve its guidance development and
Biologics
Invivyd says it is seeking FDA emergency use authorization for VYD222, which is described as a “broadly neutralizing, half-life extended monoclonal antibody candidate for the pre-exposure prevention of Covid-19 in immunocompromised adults and adolescents.” The submission is based on “positive initial results” from the CANOPY Phase 3
Biologics
FDA has published a draft guidance, Potency Assurance for Cellular and Gene Therapy Products, with recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy product. The document says a potency assurance strategy is “a multifaceted approach that
Biologics
FDA investigators cited GMP issues during a September inspection at Moderna’s primary facility in Norwood, MA, according to a Reuters report based on a related Form FDA-483 the news service obtained under a Freedom of Information Act request. The 9/11-21 inspection of the facility that makes
Biologics
FDA has accepted for priority review an Amgen BLA for tarlatamab for treating adult patients with advanced small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. The agency has set a review action target date of 6/12/2024. The BLA is based on data
Biologics
FDA has accepted for review a Pfizer BLA for its anti-tissue factor pathway inhibitor (anti-TFPI) candidate marstacimab for treating hemophilia A or hemophilia B without inhibitors to Factor VIII or Factor IX. The agency has set a user fee review action target date in the fourth quarter of
Biologics
FDA has approved the first two cell-based gene therapies for treating sickle cell disease in patients 12 years and older — Vertex Pharmaceuticals’ Casgevy and Bluebird Bio’s Lyfgenia. Casgevy is the first FDA-approved treatment to use the novel genome editing technology CRISPR/Cas9, “signaling an innovative advancement in
Biologics
CBER director Peter Marks says his Center has high hopes to speed gene therapy development through the harmonization of manufacturing protocols. This effort is mainly being led by the Bespoke Gene Therapy Consortium, a partnership between NIH, FDA and various public and private organizations, and it includes pilot programs that
Biologics
FDA has accepted for priority review a Bristol Myers Squibb (BMS) supplemental BLA for Opdivo (nivolumab) for combination use with cisplatin-based chemotherapy for first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (bladder cancer). The agency has set a user fee review action target date of
Biologics
A panel presentation at the 2023 AFDO/RAPS Healthcare Products Collaborative stressed the positive role FDA Project Jurisdiction Officers (PJO) play in coordinating the consideration of combination products. “The PJO is your best contact if you know, or think you know, your product’s Center or if you already have
Biologics
FDA has accepted for priority review an ImmunoGen supplemental BLA to convert the accelerated approval of Elahere (mirvetuximab soravtansine-gynx) to full approval for treating patients with folate receptor alpha-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been given one to three prior systemic
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 products when such standards are appropriate. The document says CBER encourages the use of
Biologics
FDA has accepted for priority review an Abeona Therapeutics BLA for pz-cel (prademagene zamikeracel) autologous COL7A1 gene-corrected epidermal sheets for treating patients with recessive dystrophic epidermolysis bullosa (RDEB). The agency has set a 5/25/2024 review action target date, according to the company, adding that an advisory
Biologics
CBER director Peter Marks and CDC National Center for Emerging and Zoonotic Infectious Diseases director Daniel Jernigan say the Vaccine Adverse Event Reporting System (VAERS) their agencies co-manage “works and has a track record that proves it.” The two wrote an online FDA Voices post that “explains how VAERS
Biologics
FDA has published a final guidance, Covid-19: Developing Drugs and Biological Products for Treatment or Prevention, to assist sponsors in developing such products. The document describes the agency’s current recommendations for Phase 2 and 3 trials, with a focus on trial population, trial design, efficacy endpoints, safety considerations,
Biologics
Pharmaceutical Research and Manufacturers of America says it backs the overall aim of an FDA draft guidance to support a “lifecycle approach” to management and reporting of manufacturing changes for human cellular and gene therapy (CGT) products. The trade association’s comment letter gives its perspective on CGT lifecycle management
Biologics
A BMJ investigation has raised concerns that the federal government’s vaccine adverse event system is not operating as planned and safety signals are likely being missed. The national Vaccine Adverse Event Reporting System (VAERS) is co-managed by FDA and the Centers for Disease Control and Prevention (CDC). Investigations
Biologics
FDA has approved a Valneva Austria BLA for Ixchiq, a vaccine for preventing chikungunya in individuals aged 18 years and above who are at heightened risk of exposure. Chikungunya, a mosquito-borne virus, has emerged as a global health threat, with over five million reported cases in the last 15