Biologics
A series of recent FDA regulatory decisions is sharpening focus on the evidentiary standards underpinning cell and gene therapy development, as sponsors face increasing difficulty translating early promise into approvable data packages — an issue highlighted in a recent opinion piece by Jenna DiRito, co-founder and chief operating officer of
Biologics
FDA is planning a 6/4 town hall meeting aimed at guiding drug developers through the process of preparing BLAs for cell and gene therapy products. The CBER virtual session will feature regulatory experts addressing common questions about BLA readiness and submission strategies. Officials from the Office of Review Management
Biologics
FDA has cited significant manufacturing and quality control concerns at a Celgene cell therapy facility in New Jersey, including deficiencies in sterile processing, environmental monitoring, and facility maintenance. The observations were detailed in a just-released FDA Form 483 issued following an inspection conducted last year at the company’s
Biologics
FDA is still weighing candidates to lead CBER as controversial director Vinay Prasad prepares to leave the agency at the end of the month. FDA commissioner Marty Makary says a decision is expected “in the coming weeks,” according to reporting from Reuters. Makary had previously indicated a successor would be
Biologics
Intellia Therapeutics has begun a rolling BLA submission for its experimental gene-editing therapy lonvoguran ziclumeran, or lonvo-z, as a one-time treatment for hereditary angioedema (HAE). The company expects to complete submission in the second half of the year and, if approved, launch the therapy in the first
Biologics
FDA has issued AbbVie a complete response letter for its BLA for trenibotulinumtoxinE, an investigational, fast-acting botulinum toxin intended for aesthetic use. The agency’s letter focused on manufacturing-related issues and did not cite concerns related to safety or efficacy, according to the company. Importantly, FDA did not
Biologics
FDA cited multiple manufacturing and quality control concerns at a Swiss drug substance facility operated by Lonza Group, following an inspection in late 2025, according to a just-posted agency inspection report. The findings, detailed in a highly redacted Form 483 issued to the company’s Visp site, identify deficiencies
Biologics
FDA has accepted a Roche supplemental BLA that seeks to expand use of Gazyva to treat Systemic Lupus Erythematosus (SLE). The agency is expected to make a decision by December. If approved, Gazyva, which is already approved for lupus nephritis, could become the first anti-CD20 therapy specifically indicated for
Biologics
Prominent ophthalmologist and biotech executive Houman Hemmati is reportedly being considered as CBER’s next director to oversee vaccines and gene/cell therapies. The board-certified eye surgeon and frequent commentator on Fox News, if selected, would replace Vinay Prasad, whose controversial tenure comes to an end later this month
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