Biologics
The House Select Subcommittee on the Coronavirus Pandemic has issued its final 550-page report that generally praises actions taken under the Trump administration and criticizes actions taken by the Biden administration. A committee statement (including a link to the full document) says “After Action Review of the Covid-19
Biologics
Neurogene is reporting that a patient dosed in an ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome has died following complications from a rare and life-threatening hyperinflammatory syndrome associated with systemic exposure to high doses of adeno-associated virus (AAV), the company says. Neurogene says FDA
Biologics
FDA has granted Jazz Pharmaceuticals accelerated approval for a BLA for Ziihera (zanidatamab-hrii), a bispecific HER2-directed antibody for previously treated, unresectable or metastatic HER2-positive biliary tract cancer. Approval is based on data from the HERIZON-BTC-01 (NCT04466891) open-label, single-arm trial in 62 patients, according
Biologics
Sen. Ron Johnson (R-WI) has directed FDA, HHS, and CDC to “immediately preserve all records referring or relating to the development, safety, and efficacy of the Covid-19 vaccines.” In a statement (includes a link to the text of a letter to the agencies), Johnson says that under the
Biologics
FDA has posted a draft guidance entitled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products.” The document is intended to provide industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during cell/gene therapy development. The FAQs cover multiple disciplines, including regulatory review,
Biologics
FDA has accepted for review a Sanofi and Regeneron supplemental BLA resubmission for Dupixent (dupilumab) to treat adults and pediatric patients aged 12 years and older with chronic spontaneous urticaria (CSU) whose disease is not adequately controlled with H1 antihistamine treatment. The user fee review action target date is 4/
Biologics
Adaptimmune Therapeutics says it plans to begin a rolling BLA next year for cell therapy lete-cel in people with synovial sarcoma or myxoid/round cell liposarcoma (MRCLS) who received previous anthracycline-based therapy. The company’s decision to seek marketing approval is based on just-announced positive data from
Biologics
Six Squire Patton Boggs attorneys say that at the end of the Supreme Court’s 2023-2024 term, the court issued four decisions that “have potentially wide-ranging consequences on federal agency rulemaking, adjudication, and regulatory compliance.” Writing in an online post, the attorneys analyze the impact of the decisions
Biologics
FDA has accepted for review an Abeona Therapeutics BLA resubmission for prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows an April complete response letter that requested additional information to satisfy chemistry manufacturing and controls requirements (see earlier
Biologics
FDA has lifted a clinical hold on a Novavax IND for its Covid-19-influenza combination (CIC) and stand-alone influenza vaccine candidates. The agency said the company can begin enrolling patients in a planned Phase 3 trial following the determination that it satisfactorily addressed all clinical hold issues, according
Biologics
FDA has approved an Autolus Therapeutics BLA for Aucatzyl (obecabtagene autoleucel), a CAR-T therapy for treating adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The product was approved based on data from the company’s FELIX clinical trial. “In the efficacy evaluable patients (n=65)
Biologics
Johnson & Johnson has submitted a supplemental BLA for Darzalex Faspro (daratumumab and hyaluronidase-fihj) and its use as monotherapy for treating adult patients with high-risk smoldering multiple myeloma. The filing is based on data from the ongoing Phase 3 AQUILA study (NCT03301220). Smoldering multiple myeloma is described as
Biologics
FDA has extended by three months its priority review of a Merus BLA for Zeno (zenocutuzumab) for treating patients with neuregulin 1 fusion (NRG1+) non-small cell lung (NSCLC) and NRG1+ pancreatic (PDAC) cancer. The extension will give FDA additional time to review information recently submitted in response to a
Biologics
Four stakeholders — St. Jude Children’s Research Hospital, the National Organization for Rare Disorders (NORD), the Muscular Dystrophy Association (MDA), and the Biotechnology Innovation Organization (BIO) — are voicing support for an FDA proposal to create an FDA Rare Disease Innovation Hub (RDI Hub). The four also respond to FDA questions
Biologics
FDA has lifted a clinical hold on three CARsgen Therapeutics clinical trials evaluating CT053, CT041, and CT071. The holds were placed 12/2023 due to chemistry, manufacturing and controls questions related to findings from an inspection of the company’s manufacturing site located in Durham, NC, according to the company.
Biologics
FDA may be backpedaling on safety warnings it required on CAR-T cell therapies related to secondary cancers, less than a year after placing class-wide black box warnings on the products (see earlier story). CBER director Peter Marks revealed the agency’s shift in an interview with Fierce Pharma,
Biologics
Responding to an FDA request for comments on total lifecycle considerations for generative artificial intelligence-enabled medical devices, AstraZeneca says it is important that the agency use a risk-based approach to the regulation of artificial intelligence/machine learning (AI/ML). “Risk-based frameworks are an important regulatory tool used
Biologics
Abeona Therapeutics has resubmitted a BLA for prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows an April complete response letter that requested additional information to satisfy chemistry manufacturing and controls requirements (see earlier story). “We have incorporated
Biologics
Intellia Therapeutics says new data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE) suggest the CRISPR-based gene editing therapy has the potential to eliminate HAE attacks following a one-time infusion. Results were published online 10/24 in The New England
Biologics
An 8/15-8/29/2022 FDA inspection at Pinnacle Transplant Technologies in Phoenix, AZ, documented that the firm manufactures an amniotic membrane and an amniotic fluid-derived product. A 10/1 Warning Letter says the firm’s two PalinGen products are legally considered to be biologics and require a
Biologics
FDA has approved expanding the indication for Pfizer’s respiratory syncytial virus (RSV) Abrysvo vaccine to cover the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 18 through 59 years of age. Abrysvo had been approved for those aged 60 and older. A company statement says
Biologics
An 8/15-/29/2022 FDA inspection at Amnio Technology in Phoenix, AZ, documented that the firm manufactures an amniotic membrane and fluid product and an amniotic fluid-derived product that have been distributed to third-party distributors, hospitals, and medical clinics in the U.S. A 10/1 Warning