Biologics
CBER director Peter Marks is predicting that 2024 will be a “breakout year” for gene therapy approvals as several initiatives work to advance the development of such products. Speaking 2/26 at the BioPharma Congress, Marks said, “What we are trying to accomplish this year is taking a number of
Biologics
FDA has accepted for review a BeiGene BLA for Tevimbra (tislelizumab) for combination use with fluoropyrimidine- and platinum-containing chemotherapy in treating locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma. The agency has set a December user fee review action target date. The submission is based on data
Biologics
After two complete response letters over inspection concerns, FDA has now granted approval to Alvotech and Teva Pharmaceuticals for Simlandi (adalimumab-ryvk) injection, an interchangeable biosimilar to AbbVie’s Humira. Earlier this year, an FDA reinspection of Alvotech’s Iceland manufacturing facility resulted in a Form FDA-483 with one
Biologics
FDA has approved a Johnson & Johnson supplemental BLA for Tecvayli (teclistamab-cqyv) for a reduced dosing frequency of 1.5 mg/kg every two weeks in patients with relapsed or refractory multiple myeloma (RRMM) who have achieved and maintained a complete response (CR). The therapy was originally approved 10/
Biologics
FDA has granted accelerated approval to Iovance Biotherapeutics for Amtagvi, a cellular therapy indicated for treating adult patients melanoma that is unable to be removed with surgery (unresectable) or has spread to other parts of the body (metastatic) that previously has been treated with other therapies (a PD-1 blocking
Biologics
FDA has accepted for priority review a Sarepta Therapeutics supplemental BLA for Elevidys (delandistrogene moxeparvovec-rokl) to convert the earlier accelerated approval to a traditional approval, and to expand the labeled indication for treating Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene. The agency has
Biologics
FDA has approved a Novartis supplemental BLA for Xolair (omalizumab) for reducing allergic reactions, including anaphylaxis, that may occur with accidental exposure to one or more foods in adult and pediatric patients aged one year and older with Immunoglobulin E (IgE)-mediated food allergy. Approval is based on data from
Biologics
Speaking on 2/14 at the National Press Club, FDA commissioner Robert Califf challenged journalists to “continue striving for approaches that benefit public health in the best possible manner.” Saying he wanted to bring some Valentine’s Day “tough love” to the audience, Califf spoke on his pressing theme of
Biologics
Citius Pharmaceuticals has resubmitted a BLA for Lymphir (denileukin diftitox), an interleukin-2-based immunotherapy for treating patients with relapsed or refractory cutaneous T-cell lymphoma after at least one prior systemic therapy. The resubmission addresses issues raised in a 7/28/2023 complete response letter. “Citius believes it has
Biologics
FDA has lifted a clinical hold against Hemogenyx Pharmaceuticals’ IND for the company’s chimeric antigen receptor T-cells (CAR-T) therapy for treating acute myeloid leukemia. The hold was issued 7/2023 and was related to a “splicing that occurs during the manufacturing process of the lentivirus that is
Biologics
FDA has accepted for priority review an Amgen supplemental BLA for Blincyto (blinatumomab) for treating early-stage, CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL). Currently, the drug is approved for the B-ALL patient population after the first or second complete remission with minimal residual disease. The
Biologics
FDA has accepted for priority review a GSK supplemental BLA to extend the indication of its Arexvy adjuvanted respiratory syncytial virus (RSV) vaccine to adults aged 50-59 who are at increased risk for RSV disease. The vaccine is currently approved for adults aged 60 and over for the prevention
Biologics
Valneva SE, a French specialty vaccine company, says it sold a Priority Review Voucher (PRV) it received from FDA for $103 million. The company says it was awarded the tropical disease PRV in 11/2023 following agency approval of its Ixchiq single-dose, live attenuated vaccine indicated to prevent disease
Biologics
FDA has accepted for priority review an Adaptimmune Therapeutics BLA for afami-cel, an investigational engineered T-cell therapy for advanced synovial sarcoma. The agency has assigned an 8/4 user fee review action target date. The submission is based on data from cohort 1 of the company’s pivotal
Biologics
Biogen says it is discontinuing the further development and commercialization of controversial Alzheimer’s drug Aduhelm (aducanumab-avwa) 100 mg/mL injection for intravenous use as part of a move to reprioritize its resources in the disease space. Aduhelm’s 2021 approval was met with public concern and outrage, much
Biologics
CBER has posted its “guidance agenda” for 2024 that includes a listing of topics upcoming guidances will address. The agenda includes topics that currently have no guidance associated with them, topics where updated guidance may be helpful, and topics for which CBER has already issued earlier draft guidances that may
Biologics
FDA has accepted for priority review an AstraZeneca and Daiichi Sankyo supplemental BLA for Enhertu (trastuzumab deruxtecan) for treating adult patients with unresectable or metastatic HER2-positive solid tumors who have received prior treatment or who have no satisfactory alternative treatment options. The submission is based on data from the
Biologics
FDA has posted a guidance entitled “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products.” The document provides recommendations regarding chemistry, manufacturing, and control, pharmacology and toxicology, and clinical study designs for oncology indications (including hematologic malignancies and solid tumors). It also provides recommendations for analytical comparability
Biologics
Merck says data from its Phase 3 AMBASSADOR (A031501)/KEYNOTE-123 trial evaluating Keytruda (pembrolizumab) are favorable for the therapy as a potential add-on treatment in high-risk patients with localized muscle-invasive urothelial carcinoma and locally advanced resectable urothelial carcinoma. “At the trial’s first pre-specified interim
Biologics
The CBER Office of Compliance and Biologics Quality says its review of a Web site maintained by Vitacell Biologics in Lewisburg, TN, found the company is illegally selling cellular products derived from human umbilical cords and an exosome product. A 1/16 untitled letter lists Web site testimonials for the
Biologics
FDA has issued Theratechnologies a complete response letter on its supplemental BLA for a F8 formulation of Egrifta SV (tesamorelin), indicated for reducing excess abdominal fat in adults with HIV and lipodystrophy. The company says the letter cited issues related to chemistry, manufacturing and controls concerning the microbiology, assays, impurities
Biologics
FDA has accepted for priority review a Genentech supplemental BLA for Xolair (omalizumab) for reducing allergic reactions, including anaphylaxis, that may occur with an accidental exposure to one or more foods in adult and pediatric patients with a food allergy. The submission is based on the “positive” data from the