Biologics
FDA has issued Replimune a complete response letter (CRL), declining to approve its BLA for vusolimogene oderparepvec in combination with nivolumab for advanced melanoma. In a 4/10 letter, the agency said a reanalysis of clinical data and review of additional evidence failed to demonstrate “substantial evidence of effectiveness” for
Biologics
Organogenesis says it has reached a key regulatory milestone for its knee osteoarthritis treatment ReNu, following a Type B meeting with FDA that has cleared the way for a rolling BLA submission later this year. ReNu, a cryopreserved amniotic suspension allograft, is being developed to treat symptomatic knee osteoarthritis, a
Biologics
Amgen says it has achieved positive topline results from a Phase 3 trial evaluating a subcutaneous version of its thyroid eye disease drug Tepezza (teprotumumab-trbw), potentially paving the way for a more convenient alternative to the currently approved intravenous formulation. The study met its primary endpoint, with patients receiving
Biologics
FDA has identified deficiencies in quality system procedures at a manufacturing facility operated by CSL Behring GmbH in Marburg, Germany, following an inspection conducted in April 2024. According to a just-posted FDA Form 483 issued at the close of the inspection, investigators cited the company for failing to adequately
Biologics
CBER has issued a Standard Operating Policy and Procedure (SOPP) for filing an application when the applicant protests a refusal-to-file (RTF) action. The SOPP applies to NMEs, NDAs, original BLA submissions, and associated efficacy or manufacturing supplements, including those subject to PDUFA, BsUFA, and non-PDUFA products. When
Biologics
Ultragenyx Pharmaceutical has resubmitted a BLA seeking accelerated approval for UX111, an AAV9 gene therapy for the treatment of Sanfilippo syndrome Type A, a rare and fatal pediatric neurodegenerative disorder. Sanfilippo syndrome type A is described as a rare inherited lysosomal storage disorder caused by mutations in the SGSH gene,
Biologics
FDA has granted full approval for Kite’s CAR-T cell therapy Tecartus (brexucabtagene autoleucel) for adult patients with relapsed or refractory mantle cell lymphoma (MCL), converting its prior accelerated approval into a traditional approval based on confirmatory clinical data. The decision is based on updated results from the ZUMA-
Biologics
Xbrane Biopharma said it expects to resubmit a BLA before June for ranibizumab, a biosimilar copy of Genentech’s Lucentis, which is indicated for treating neovascular age-related macular degeneration, macular edema following retinal vein occlusion, and myopic choroidal neovascularization. Ranibizumab is an anti-vascular endothelial growth factor therapy that
Biologics
A novel “off-the-shelf” CAR-T cell therapy developed by researchers at Washington University School of Medicine in St. Louis has received an FDA breakthrough therapy designation. The therapy, known as WU-CART-007 (soficabtagene geleucel), is designed to treat T-cell acute lymphoblastic leukemia and T-cell lymphoblastic
Biologics
CBER has issued four untitled letters in a single coordinated enforcement action, targeting promotional materials for leading T-cell therapies and signaling heightened scrutiny of how cutting-edge biologics are marketed. The letters fit into a wider FDA enforcement trend. Since late 2025, the agency has significantly increased scrutiny of
Biologics
FDA has granted Rocket Pharmaceuticals accelerated approval for its gene therapy Kresladi (marnetegragene autotemcel) for children with severe Leukocyte Adhesion Deficiency-I (LAD-1), marking the first approved treatment targeting the underlying genetic cause of the disease. The therapy’s approval covers pediatric patients with biallelic mutations in the ITGB2
Biologics
A Heartland Institute online newsletter quotes several experts with differing opinions on why CBER director Vinay Prasad is leaving FDA for a second time. The post says that during his tenure, Prasad, in his dual role as CBER director and agency chief medical and scientific officer, “raised concerns about drugs
Biologics
Ocugen is announcing encouraging 12-month results from a mid-stage clinical trial of its experimental gene therapy OCU410, aimed at treating geographic atrophy, an advanced form of dry age-related macular degeneration that leads to irreversible vision loss. According to the company, patients receiving the optimal dose of OCU410
Biologics
Pfizer and Valneva say they will submit a BLA for their investigational Lyme disease vaccine candidate based on data demonstrating more than 70% efficacy in a late-stage clinical trial, marking a significant step toward what could become the first approved human vaccine for the disease. The Phase 3 VALOR
Biologics
CBER director Vinay Prasad’s departure at the end of next month is raising questions across the biotechnology sector about how the agency’s approach to vaccines, gene therapies and rare-disease treatments could change in the months ahead. Prasad has had a tumultuous tenure that lasted less than a
Biologics
FDA has expanded the approved use of GSK’s Arexvy, a respiratory syncytial virus (RSV) vaccine that now includes the in adults aged 18 to 49 who are at increased risk of severe disease. The vaccine was previously authorized for preventing RSV-related lower respiratory tract disease (LRTD) in adults
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee has voted to recommend that seasonal influenza vaccines for the 2026–2027 U.S. season use a trivalent formulation, targeting two influenza A strains and one influenza B strain. Panel members voted 7–0 in favor of recommended strains for both
Biologics
FDA has cited a U.K.-based clinical research center for failing to promptly report adverse events and for potential falsification of study records during a late-stage clinical trial of a respiratory syncytial virus (RSV) vaccine. In a just-posted untitled letter dated 8/5/2025, FDA said an
Biologics
FDA has released an updated draft guidance outlining new and revised recommendations for companies developing biosimilar products, including clarifications on the use of foreign comparator products in clinical studies and requirements for retaining study samples. The document revises and replaces a 2021 draft guidance titled New and Revised Draft Q&
Biologics
FDA has issued Incyte a complete response letter (CRL) for its supplemental BLA for Zynyz (retifanlimab-dlwr) injection (375mg) for an additional indication for treating adult patients with metastatic non-small cell lung cancer in combination with platinum-based chemotherapy. The sBLA was based on data from the Phase 3
Biologics
CBER director Vinay Prasad will step down at the end of April, according to HHS. His departure comes on the heels of several recent controversial decisions at the Center and news of an internal investigation into complaints from staff. HHS did not provide additional details about the reason for his
Biologics
A senior FDA official sharply criticized UniQure’s investigational gene therapy for Huntington's disease, calling it a “failed product” during a 3/5 conference call with reporters and intensifying doubts about the treatment’s regulatory prospects, according to Reuters. The remarks came days after the company said FDA