Biologics
FDA has published a draft guidance, Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments, the third in a series of four methodological patient-focused drug development guidances that describe how stakeholders such as patients, caregivers, researchers, medical product developers, and others, can collect and
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee 6/28 voted 19 to 2 to recommend that the SARS-CoV-2 strain composition of Covid-19 vaccine boosters should be modified to include an Omicron variant component for use this fall and winter. This was a follow-up meeting
Biologics
A 7/19-7/28/21 FDA inspection at Re-Gen Active Lab, an Irving, TX, manufacturer of cellular products for allogeneic use, found multiple violations, including marketing the products illegally without required agency approvals. A just-released 5/27 Warning Letter says the inspection also found evidence of significant
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee is meeting 6/28 to discuss whether and how the SARS-CoV-2 strain composition of Covid-19 vaccine boosters should be modified for use this fall and winter. This is a follow-up meeting to a 4/6 panel deliberation
Biologics
Krystal Biotech has submitted a BLA for B-Vec (beremagene geperpavec) and its use in treating patients with dystrophic epidermolysis bullosa (DEB). The topical gene therapy is designed to treat DEB at the molecular level by providing the patient’s skin cells with two copies of the COL7A1 gene to
Biologics
CBER has issued an updated agenda of guidances it plans to publish this year. The document lists eight guidances under Blood and Blood Components, eight under Tissues and Advanced Therapies, and two under Vaccines.
Biologics
FDA has denied a 5/20 petition submitted by the Informed Consent Action Network relating to emergency use authorizations for and development of vaccines to prevent Covid-19. The petition asked that FDA: * revoke its 5/10/21 emergency use authorization for the use of Pfizer/BioNTech’s Covid-19
Biologics
FDA has amended the emergency use authorizations for Moderna’s Covid-19 vaccine and Pfizer-BioNTech’s Covid-19 vaccine to allow their use in children as young as six months of age. The Moderna vaccine is administered as a primary series of two doses, one month apart, to individuals
Biologics
Lonza Biologics says it appreciates FDA’s efforts to make available the agency’s current thinking on a range of topics applicable to the development of cell and gene therapy products. “Lonza particularly appreciates information specific to chemistry, manufacturing, and controls (CMC) expectations,” the letter says. The company includes detailed
Biologics
FDA has published a draft guidance, Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies, describing a CBER program designed to identify and recognize voluntary consensus standards to facilitate the development and assessment of regenerative medicine therapy products regulated by the Center. The document says CBER encourages the use of
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee unanimously voted 6/15 to recommend the agency expand the Covid-19 vaccine emergency use authorizations (EUA) for Pfizer-BioNTech and Moderna to allow their use in children as young as six months of age. Panel members concluded that the Pfizer-
Biologics
An FDA advisory committee 6/14 voted unanimously (22 to 0) to recommend the agency grant Moderna an emergency use authorization for its Covid-19 vaccine in children aged six months to 17 years. An FDA briefing document showed that vaccine efficacy was 93.3% for participants 12-17 years
Biologics
FDA briefing documents for its advisory committee review on 6/14-15 of the Pfizer-BioNTech and Moderna Covid-19 vaccines show that they are both safe and effective in children aged six years and younger. In the Pfizer vaccine, vaccine efficacy was observed at 75.6% in trial participants
Biologics
Some 18 members of Congress led by Sen. Ted Cruz (R-TX) have written to FDA commissioner Robert Califf and the agency’s Vaccines and Related Biological Products Advisory Committee with questions about emergency use authorizations (EUA) for Covid-19 vaccines in children under age 5. A statement (contains a
Biologics
Fresh off a positive FDA advisory committee vote (see earlier story) to recommend an emergency use authorization of its Covid-19 vaccine, Novavax now finds its vaccine stalled at the agency because of last minute manufacturing data the company submitted, which have to be reviewed before the agency renders a
Biologics
Moderna says its Omicron-containing bivalent Covid vaccine booster candidate “met all pre-specified endpoints including superior neutralizing antibody response against the Omicron variant one month after administration” when compared to the original Moderna vaccine. A company statement says the Omicron booster was generally well-tolerated, with side effects comparable
Biologics
FDA says it is challenged to determine the overall benefit-risk profile of Bluebird bio’s eli-cel (elivaldogene autotemcel) gene therapy, indicated for treating patients less than 18 years of age with early cerebral adrenoleukodystrophy who do not have an available and willing human leukocyte antigen-matched sibling hematopoietic
Biologics
FDA’s Vaccines and Related Biological Products Advisory Committee 6/7 voted unanimously (21 to 0) to recommend that the agency grant Novavax an emergency use authorization for its Covid-19 vaccine and its use in individuals 18 years of age and older. FDA’s briefing document said vaccine efficacy
Biologics
FDA has approved a GSK BLA for Priorix (measles, mumps and rubella vaccine) for use in individuals 12 months of age and older. The company notes that recently measles outbreaks have been rising in the U.S. and globally, “with more than 400,000 cases confirmed in 2019, reversing decades
Biologics
Bristol Myers Squibb has withdrawn a supplemental BLA for Reblozyl (luspatercept-aamt) for treating anemia in adults with non-transfusion dependent (NTD) beta thalassemia. The company said it could not appropriately address FDA’s questions about the benefit-risk profile of Reblozyl in the patient population based on the current
Biologics
An FDA advisory committee briefing document says that Novavax’s Covid-19 vaccine’s two-dose primary series demonstrated significant efficacy, but it also showed similar risk signals seen with other Covid vaccines, such as myocarditis/pericarditis. The agency’s Vaccines and Related Biological Products Advisory Committee is being convened
Biologics
Gamida Cell has completed a rolling BLA submission for omidubicel, indicated for patients with blood cancers in need of stem cell transplant. The BLA is based on data from a Phase 3 study showing that omidubicel achieved a statistically significant reduction in time to neutrophil engraftment, reduced hospitalization time, decreased