Biologics
A 3/1-3/22 FDA inspection at Invitrx Therapeutics, Lake Forest, CA, found multiple violations in the firm’s manufacturing of cellular and acellular products for allogeneic use. An 11/9 Warning Letter says the products are intended to be administered by injection, ophthalmically, or by nebulization and purport
Biologics
FDA has extended by three months the review of a Gamida Cell BLA for omidubicel for treating patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. Omidubicel is described as a first-in-class, advanced NAM-enabled stem cell therapy candidate. The review extension is related
Biologics
Iovance Biotherapeutics has delayed until the first quarter next year its completion of a rolling BLA submission for lifileucel, an investigational tumor infiltrating lymphocyte therapy for patients with advanced (unresectable or metastatic) melanoma who progressed on or after prior anti-PD-1/L1 therapy, and targeted BRAF/MEK inhibitor therapy.
Biologics
Pfizer and BioNTech have begun a Phase 1 study of their next-generation Covid-19 vaccine that they say is designed to “enhance SARS-CoV-2 T cell responses and potentially broaden protection against Covid-19.” The study is part of the companies’ strategy to generate more robust, longer-lasting,
Biologics
FDA has denied in full citizen petitions filed on 3/24/21 and 8/12/22 by the Informed Consent Action Network relating to certain provisions of the emergency use authorizations (EUAs) for Covid-19 vaccines. The agency response lists the lengthy and detailed requests made in the petitions. The
Biologics
Protalix BioTherapeutics and Chiesi Global Rare Diseases have resubmitted a BLA for PRX–102 (pegunigalsidase alfa) for treating adult patients with Fabry disease. The companies describe pegunigalsidase alfa as a purposefully-designed, long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate. Last year, the agency issued a
Biologics
FDA has granted ImmunoGen an accelerated approval for Elahere (mirvetuximab soravtansine-gynx) for treating adult patients with folate receptor alpha (FRa)-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Approval was based on objective response rate (ORR)
Biologics
As expected, BioMarin Pharmaceutical says FDA has requested results from an upcoming three-year data analysis from the ongoing Phase 3 GENEr8-1 study on its resubmitted valoctocogene roxaparvovec, a gene therapy for adults with severe Hemophilia A that is currently under BLA review. The agency also told the company
Biologics
FDA has released a final guidance entitled “Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial.” The document provides recommendations to sponsors interested in studying (umbrella trials) multiple versions of a cellular or gene therapy product in an early-phase clinical trial for
Biologics
FDA has issued Gilead a complete response letter on its BLA for bulevirtide, an investigational entry-inhibitor for treating adults with hepatitis delta virus (HDV) infection and compensated liver disease. FDA’s letter cited concerns about the “manufacture and delivery of bulevirtide,” according to the company, adding that no new
Biologics
FDA’s Oncologic Drugs Advisory Committee 10/28 voted 16 to 0 that Y-mAbs Therapeutics’ BLA for I-omburtamab, seeking an indication for treating pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma, did not provide sufficient evidence to conclude that omburtamab improves overall survival. FDA medical reviewers told panel
Biologics
FDA has released a final guidance entitled “Human Gene Therapy for Neurodegenerative Diseases.” The document focuses on considerations for product development, preclinical testing, and clinical trial design. The guidance says that general chemistry, manufacturing and controls for product manufacturing, testing and release of gene therapy products should address challenges associated
Biologics
FDA has announced five research grants made under the Biosimilar User Fee Act (BsUFA) research grant program in the areas of (1) improving the efficiency of biosimilar product development and (2) advancing the development of interchangeable products. The five grants are: * assessment of the performance of multi-attribute method versus
Biologics
FDA has granted Novavax an emergency use authorization for its Novavax Covid‑19 Vaccine, Adjuvanted as a first booster dose (0.5 mL) for certain individuals at least six months after completing their primary vaccination with an authorized or approved Covid-19 vaccine. According to the agency, the booster is
Biologics
FDA has accepted for review a BioMarin BLA resubmission for gene therapy valoctocogene roxaparvovec for adults with severe hemophilia A. The agency has set a user fee review action target date of 3/31, and currently it has not communicated any plans to hold an advisory committee meeting, according to
Biologics
GlaxoSmithKline says its older adult respiratory syncytial virus (RSV) vaccine candidate showed a 94.1% reduction in severe RSV disease and overall vaccine efficacy of 82.6% in a Phase 3 pivotal trial. A company statement says consistent high vaccine efficacy was observed against RSV-lower respiratory tract disease in
Biologics
FDA has amended the emergency use authorizations for the Moderna and Pfizer/BioNTech bivalent Covid-19 vaccines to authorize their use as a single booster dose in younger age groups. An agency statement says the Moderna bivalent is authorized for administration at least two months following completion of primary or
Biologics
Merck has exercised its option to join with Moderna to develop and commercialize a personalized cancer vaccine currently being evaluated in combination with Merck’s Keytruda (pembrolizumab), an anti-PD-1 therapy, as adjuvant treatment for patients with high-risk melanoma in a Moderna Phase 2 trial. A Merck statement
Biologics
FDA has approved GlaxoSimthKline Biologicals’ Boostrix (tetanus toxoid, reduced diphtheria toxoid and acellular pertussis vaccine, adsorbed [Tdap]) for immunization during the third trimester of pregnancy to prevent pertussis (whooping cough) in infants younger than two months of age. Most serious pertussis cases, hospitalizations and deaths occur in infants younger than
Biologics
CBER has published four Standard Operating Policies and Procedures (SOPPs) relating to the administrative processing of applications. SOPP 8401: Administrative Processing of Original BLAs and NDAs includes those subject to the Biosimilar User Fee Act (BsUFA) and Prescription Drug User Fee Act (PDUFA). It does not apply to BLAs subject
Biologics
FDA has awarded the Biologics & Biosimilars Collective Intelligence Consortium (BBCIC) a $1.3 million grant to support biosimilars research and a study focused on increasing the efficiency of biosimilar drug development and review. The study is entitled “Improving the Efficiency of Regulatory Decisions for Biosimilars and Interchangeable Biosimilars by
Biologics
Sarepta Therapeutics has submitted a BLA seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients with Duchenne muscular dystrophy. SRP-9001 is a gene therapy being developed in partnership with Roche. The submission relies on the expression of SRP-9001 dystrophin protein, “an internally shortened and functional