Biologics
CBER has released a 6/6 letter to sponsors, applicants, and regulated entities with updated information to assist the recipients to transition back to normal operations in this calendar year following the expiration of the Covid-19 public health emergency. The letter covers information about CBER meetings; FDA guidance; the
Biologics
Legend Biotech has submitted a supplemental BLA seeking to expand the label for Carvykti (ciltacabtagene autoleucel) to include the treatment of adult patients with relapsed and lenalidomide-refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent. The submission
Biologics
FDA has accepted for review a GSK supplemental BLA for Jemperli (dostarlimab) in combination with chemotherapy for treating adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high primary advanced or recurrent endometrial cancer. “If approved in this patient population, dostarlimab plus chemotherapy could represent the first meaningful frontline treatment
Biologics
FDA has granted Kyverna Therapeutics a fast track designation for KYV-101, an anti-CD19 chimeric antigen receptor T-cell therapy for treating patients with refractory lupus nephritis. KYV-101 is designed to deplete B cells, including autoreactive B cells, in autoimmune disease patients, the company says, adding that a
Biologics
FDA has approved Pfizer’s Abrysvo (respiratory syncytial virus vaccine), a bivalent RSV (respiratory syncytial virus) prefusion F vaccine, to prevent lower respiratory tract diseases caused by RSV in people age 60 and older. A company statement says Abrysvo is unadjuvanted and is composed of two preF proteins selected to
Biologics
FDA has completed a previously delayed pre-licensing inspection of China-based Shanghai Junshi Biosciences’ toripalimab manufacturing site. The product is being developed by Coherus BioSciences and Junshi for treating recurrent or metastatic nasopharyngeal carcinoma (NPC). Coherus says the inspection concluded with a three-item FDA Form-483. It says
Biologics
FDA has accepted for priority review an Iovance Biotherapeutics BLA submission for lifileucel, a tumor infiltrating lymphocyte therapy for treating patients with advanced (unresectable or metastatic) melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy. The company is seeking accelerated approval from FDA, which
Biologics
The National Organization for Rare Disorders (NORD) says it applauds FDA efforts to seek extensive comments and community engagement on post-market surveillance for cell and gene therapy products, and greatly appreciated the April public listening session on the topic. The NORD comment letter discusses these topics with recommendations: * post-
Biologics
The CBER Office of Compliance and Biologics Quality says its review of a Web site and YouTube channel maintained by AT Venture Center for Global Techtrepreneurship, doing business as Regenerelle in Fairport, NY, determined that the firm is marketing a human mesenchymal stem cell product intended to treat various diseases
Biologics
FDA has approved a Celltrion BLA for Yuflyma (adalimumab-aaty), biosimilar copy of AbbVie’s Humira. The high-concentration (100mg/mL) and citrate-free formulation is approved for treating eight conditions — rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, plaque psoriasis, and hidradenitis suppurativa.
Biologics
FDA has extended by three weeks its review of a Sarepta Therapeutics BLA for SRP-9001 (delandistrogene moxeparvovec), an adeno-associated virus vector-based gene therapy product for treating ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. The submission is currently under review
Biologics
FDA has approved a Krystal Biotech BLA for Vyjuvek, a herpes-simplex virus Type 1 vector-based gene therapy for treating wounds in patients six months of age and older with dystrophic epidermolysis bullosa (DEB). The topical gene therapy is designed to treat DEB at the molecular level by providing
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee 5/18 voted 14-0 that a Pfizer BLA demonstrated effectiveness for its Abrysvo (RSVpreF) vaccine for preventing respiratory syncytial virus (RSV) in infants from birth through six months by active immunization of pregnant individuals. A separate vote of 10-4
Biologics
FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee 5/12 voted 8 to 6 to recommend approval of a Sarepta BLA seeking accelerated approval for its SRP-9001 (delandistrogene moxeparvovec), an adeno-associated virus vector-based gene therapy product, to treat ambulatory patients with Duchenne muscular dystrophy (DMD) with
Biologics
FDA has issued Byondis a complete response letter on its BLA for SYD985 ([Vic-] trastuzumab duocarmazine) for use in patients with HER2-positive unresectable locally advanced or metastatic breast cancer (MBC). The letter requests additional information that “requires time and resources that extend beyond the current evaluation period,” the company
Biologics
FDA has published a guidance, Recommendations for Evaluating Donor Eligibility Using Individual Risk-Based Questions to Reduce the Risk of Human Immunodeficiency Virus Transmission by Blood and Blood Products, revising the agency’s recommendations for evaluating blood donor eligibility due to human immunodeficiency virus (HIV) using individual risk-based questions.
Biologics
FDA has approved a Chiesi Global Rare Diseases and Protalix BioTherapeutics BLA for Elfabrio (pegunigalsidase alfa-iwxj) for treating adult patients with Fabry disease. The PEGylated enzyme replacement therapy (ERT) contains the recombinant human α–Galactosidase-A enzyme, which is expressed in a plant-cell culture that is designed to
Biologics
FDA has approved a GlaxoSmithKline BLA for Arexvy (respiratory syncytial virus vaccine, adjuvanted), for preventing lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. “Older adults, in particular those with underlying health conditions, such as heart or lung disease or
Biologics
Reps. Lori Trahan (D-MA) and Dan Crenshaw (R-TX) say they are concerned about FDA’s pace for authorizing and approving updated Covid-19 therapeutics. In a 5/3 letter to FDA commissioner Robert Califf, the two say that while FDA has modernized its process in the past for
Biologics
The Biotechnology Innovation Organization (BIO) says the scope of an FDA draft guidance on considerations for the design and conduct of externally controlled trials for drugs and biologics “is rather narrow, excluding aggregate-level data as well as hybrid designs using external controls to augment a randomized trial.” The BIO
Biologics
FDA has published a draft guidance, Decentralized Clinical Trials for Drugs, Biological Products, and Devices, with recommendations for sponsors, investigators, and other stakeholders on the implementation of decentralized clinical trials (DCTs) for drugs, biological products, and medical devices. The document defines a decentralized clinical trial as one in which some
Biologics
FDA has released an International Council for Harmonization final guidance entitled “S12 Nonclinical Biodistribution (BD) Considerations for Gene Therapy Products.” It provides harmonized recommendations for the overall design of nonclinical BD assessments. Considerations for interpretation and application of the BD data to support a nonclinical development program and the design