FDA Policy/General
The FDA Bioresearch Monitoring (BIMO) FY 2023 metrics report shows there were 1,073 BIMO inspections across the five FDA Centers. CDER had by far the largest number of inspections at 779, with 141 through CDRH, 126 through CBER, and 26 through CVM. The report also gives common inspection observations
Biologics
FDA has approved a Johnson & Johnson BLA for Rybrevant (amivantamab-vmjw) plus Lazcluze (lazertinib) for the first-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations. The
Biologics
FDA has accepted for priority review an AstraZeneca supplemental BLA for Imfinzi (durvalumab) and its use in patients with limited-stage small cell lung cancer whose disease has not progressed following platinum-based concurrent chemoradiotherapy. The company says a review action target date is expected during the fourth quarter of
Biologics
FDA has approved a Vericel supplemental BLA for NexoBrid (anacaulase-bcdb) for eschar removal in pediatric patients with deep partial-thickness or full-thickness thermal burns. Previously the product was approved for the same indication but for use in adults only. The expanded approval was based on results from the
Biologics
FDA has approved a Galderma BLA for Nemluvio (nemolizumab), a pre-filled pen for subcutaneous injection for adults with prurigo nodularis. The underdiagnosed neuroimmune skin disease is characterized by several debilitating symptoms, including chronic itch, skin nodules covering large body areas, and poor sleep quality, the company says. Galderma says
Biologics
A 3/15-5/19/2023 FDA Bioresearch Monitoring Program inspection of a clinical study led by Konrad Rejdak in Lublin, Poland, found objectionable conditions. A just-released 5/29 Warning Letter says the agency has determined that Rejdak violated regulations governing the proper conduct of clinical studies involving investigational
Biologics
FDA has told Humacyte it needs more time to complete the review of the company's BLA for the acellular tissue-engineered vessel (ATEV), indicated for treating vascular trauma. “During the course of the BLA review, the FDA has conducted inspections of our manufacturing facilities and clinical sites and
Biologics
AdvaMed says it supports FDA’s long-standing bioresearch monitoring (BIMO) program and the provision of the FDA Omnibus Reform Act of 2022 that codified the program into law. In its comment on an FDA draft guidance on BIMO inspection processes and practices, AdvaMed says the agency should clarify that
Biologics
FDA has asked Actinium Pharmaceuticals for another Phase 3 study to support a BLA submission for Iomab-B in patients with active relapsed or refractory acute myeloid leukemia. “The FDA has now determined that demonstrating an overall survival benefit in a randomized head-to-head trial is required for a
Biologics
The American Society of Gene & Cell Therapy says it would like FDA to clarify aspects of its draft guidance on considerations for using human- and animal-derived materials in the manufacture of cellular and gene therapy and tissue-engineered medical products. Its comment letter asks for clarifications on these
Biologics
FDA has published a guidance, Recommendations for Investigational and Licensed Covid-19 Convalescent Plasma, with recommendations to blood establishments for submitting a BLA for the manufacture of Covid-19 convalescent plasma for transfusion intended to treat patients with immunosuppressive disease or receiving either outpatient or inpatient immunosuppressive treatment. The document
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The Massachusetts Institute of Technology Center for Biomedical Innovation (CBI) is recommending that FDA use a draft guidance on safety testing of human allogeneic cells expanded for use in cell-based medical products to recommend a transition to the high-throughput sequencing (HTS) molecular-based technology. In its comment letter
Biologics
FDA has created a docket to receive information and comments on how best to advance the development of new biosimilar biological products as part of the agency’s commitments under the Biosimilar User Fee Amendments of 2022. The agency is asking for industry input on whether biosimilar product development would
Biologics
FDA has accepted a Genentech supplemental BLA for Susvimo (ranibizumab injection) 100 mg/mL, indicated for treating diabetic macular edema (DME) and diabetic retinopathy (DR), the two leading causes of vision loss in adults with diabetes. Susvimo is the first refillable eye implant that provides continuous delivery of ranibizumab through
Biologics
FDA is planning to ask an advisory committee 7/25 whether a new trial should be conducted before approving an AstraZeneca supplemental BLA for Imfinzi (durvalumab) injection that is seeking its use in combination with platinum-containing chemotherapy as neoadjuvant treatment. The application proposes that this would then be followed
Biologics
FDA has approved a Samsung Bioepis BLA for Epysqli (eculizumab-aagh) as a biosimilar to Alexion’s Soliris (eculizumab), indicated for treating patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and atypical hemolytic uremic syndrome. The approval was based on a “totality of evidence” including analytical, non-clinical and
Biologics
FDA has published a guidance, Blood Pressure and Pulse Donor Eligibility Requirements—Compliance Policy, addressing the regulatory requirements for determining donor eligibility that apply to establishments that collect blood and blood components for transfusion or for further manufacturing use. The document says the agency has developed the guidance in response
Biologics
FDA has accepted for priority review an Atara Biotherapeutics BLA for gene therapy tabelecleucel (tab-cel) indicated as monotherapy for treating adult and pediatric patients two years of age and older with Epstein-Barr virus (EBV)-positive post-transplant lymphoproliferative disease who have received at least one prior therapy. The
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FDA has published a guidance, Application User Fees for Combination Products, explaining how fees are assigned for combination products reviewed in a single application or separate applications for each component. The document says the guidance replaces one issued in 4/2005 to provide consistency with current user fee programs and
Biologics
CBER says a Bioresearch Monitoring Program remote regulatory assessment of the Massachusetts Institute of Technology Committee on the Use of Humans as Experimental Subjects institutional review board (IRB) conducted between 5/30 and 6/9/2023 found “objectionable conditions” that were repeated observations from previous reviews. A just-released 6/
Biologics
FDA has granted Bayer-owned Asklepios BioPharmaceutical (AskBio) a fast track designation for AB-1005, a gene therapy candidate for treating moderate Parkinson’s disease. The investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy is designed for stable and continuous expression of
Biologics
FDA has issued Novo Nordisk a complete response letter (CRL) on its BLA for once-weekly basal insulin icodec, indicated for treating diabetes mellitus. In the letter, the agency requested more information about the manufacturing process and the Type 1 diabetes indication before the application’s review can be completed,