Biologics
FDA has approved a Regeneron Pharmaceuticals BLA for Evkeeza (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and pediatric patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza works by binding to and blocking the function of
Biologics
Freeline Therapeutics says it has modified its clinical development plan for its adeno-associated virus vector-mediated gene therapy FLT180a program for Hemophilia B to address FDA feedback related to the characterization and comparability of the investigational drug product used in its Phase 1/2 B-AMAZE trial that was
Biologics
Despite an all-hands-on-deck approach to address Covid-19 issues in 2020, CBER was also able to squeeze out its new CBER Advanced Technologies Team (CATT) to “address the need for a maximally efficient, agile, flexible pharmaceutical manufacturing sector that reliably produces high quality biologics,” according to Center
Biologics
FDA is limiting the emergency use authorization (EUA) for Covid-19 convalescent plasma after evaluating the most recent information available. “Based upon data from new clinical trials analyzed or reported since the original EUA was issued in August 2020, we have revised the EUA to limit the authorization to the
Biologics
Janssen Biotech has filed an emergency use authorization (EUA) application for its single-dose Covid-19 vaccine candidate. Last week, the company reported that its vaccine was 72% effective in the U.S. and 66% effective overall at preventing moderate to severe Covid-19. The vaccine’s Phase 3 data
Biologics
CBER says it plans to issue 14 guidances in three categories during calendar year 2021. The Center’s guidance agenda anticipates six guidances under Blood and Blood Components, seven guidances under Tissues and Advanced Therapies, and one guidance under Other.
Biologics
FDA has approved a Biogen supplemental BLA for an intramuscular (IM) injection route of administration for Plegridy (peginterferon beta-1a), indicated for treating relapsing forms of multiple sclerosis. A subcutaneous (SC) formulation of Plegridy was originally approved in 2014. Plegridy uses pegylated interferon beta-1a to extend its half-life
Biologics
Janssen and Novavax have both reported positive data from Phase 3 clinical trials involving each of their investigational Covid-19 vaccines. Janssen says its vaccine was 72% effective in the U.S. and 66% effective overall at preventing moderate to severe Covid-19. The vaccine’s Phase 3 data also
Biologics
FDA has extended by three months the review of a Biogen BLA for aducanumab, indicated for treating Alzheimer’s disease. The updated user fee review action target date is 6/7. The extension was triggered by a Biogen-submitted “information request” response that included FDA-requested analyses and clinical data,
Biologics
Cardiac surgeon Hooman Noorchashm, known to FDA for his opposition to power morcellation, has contacted the agency and vaccine makers Pfizer and Moderna about the immunological danger he says could be posed by the Covid-19 vaccine to the recently convalescent or asymptomatic carriers of SARS-CoV-2, and especially
Biologics
FDA has resumed conducting “prioritized” inspections by investigator staff in China and they will begin shortly in India too, according to CDER acting director Patrizia Cavazzoni. In a recent Twitter posting, Cavazzoni noted that inspections deemed “mission critical” are still being conducted. “We’re prioritizing inspections so the agency will
Biologics
CBER says its review of a Web site and Facebook page indicate that The Body Building, Clovis, CA, is marketing cellular products derived from human umbilical cord or human umbilical cord blood for many diseases and conditions. A 1/25 Notice of Violation says that FDA regulates such products as
Biologics
FDA is continuing to closely monitor the various identified variants of the virus that causes Covid-19 and also monitoring for emerging variants, according to a series of 1/25 Tweets by FDA acting commissioner Janet Woodcock. Woodcock says the agency “will continue to factor in newly emerging information in
Biologics
FDA says Edge Pharma, Colchester, VT, is marketing mixtures of allergenic extracts for patient-specific allergy treatment, including some intended for sublingual immunotherapy. A 1/22 Notice of Violation says the firm’s patient-specific extracts and custom mix allergy immunotherapy vials that are not patient-specific can only be
Biologics
FDA has published an immediately effective guidance, Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During Covid-19 Public Health Emergency, to give manufacturers of such products risk-based recommendations to minimize potential transmission of the SARS-CoV-2 novel coronavirus. The document is intended to supplement
Biologics
The Peggy Lillis Foundation for C. Diff Education and Advocacy is asking FDA to take several actions to improve the availability of microbiome treatments for C. difficile infections. The 1/12 petition asks FDA to: * engage patients and caregivers in developing agency guidelines for potential new therapies for C. difficile,
Biologics
FDA commissioner Stephen Hahn and senior advisor Colin Rom say the agency has signed a memorandum of understanding (MOU) with the National Institute of Standards and Technology (NIST) to increase U.S. medical supply chain resilience and advanced domestic manufacturing of drugs, biological products, and medical devices through the adoption
Biologics
U.S. Stem Cell, Inc., Sunrise, FL, says it argued 1/13 before the 11th Circuit Court of Appeals that it should be allowed to present evidence and testimony in support of its position and practices in a trial. The case involves a 5/9/18 FDA suit against the
Biologics
FDA has accepted for priority review an Omeros BLA for narsoplimab for treating hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). HSCT-TMA is a significant and often lethal complication of stem cell transplantation. “This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning
Biologics
Operation Warp Speed chief adviser Moncef Slaoui has resigned at the request of the Biden administration, CNBC reports. The cable business network says the controversial former GlaxoSmithKline executive will remain in his role for a month to assist in the transition. Sources tell CNBC that Slaoui’s role in leading
Biologics
FDA commissioner Stephen Hahn and CBER director Peter Marks say the available data “continue to support the use of two specified doses of each authorized [Covid-19] vaccine at specified intervals.” Hahn and Marks issued a joint statement to counter mass media articles and opinion pieces, such as this one
Biologics
FDA 12/18 issued an Emergency Use Authorization (EUA) for Moderna’s Covid-19 vaccine for use in individuals 18 years of age and older. The EUA follows a 20 to 0 advisory committee vote the day before recommending its issuance. Shipments of almost six million vaccine doses could begin