Biologics
FDA has issued Aeglea BioTherapeutics a refusal-to-file letter for its BLA for pegzilarginase, indicated for treating arginase 1 deficiency (ARG1-D). The letter requested additional data to support effectiveness, “such as evidence showing that plasma arginine and metabolite reduction predicts clinical benefit in patients with ARG1-D or
Biologics
A 12/6/21-2/4/22 FDA inspection at OsteoLife Biomedical, Miami, FL, documented significant deviations from the regulations for human cells, tissues, and cellular and tissue-based products (HCT/Ps). A just-released 3/31 Warning Letter says specific items of concern were: * failing to process HCT/Ps
Biologics
FDA has denied a 3/23/21 petition for reconsideration submitted by the Informed Consent Action Network (ICAN) concerning the agency’s denial of ICAN’s 10/16/20 original petition asking it to amend the study design for a Phase 3 trial of Johnson & Johnson’s Covid-19
Biologics
FDA has accepted for priority review a CSL Behring BLA for etranacogene dezaparvovec, an investigational gene therapy for treating adults with hemophilia B. The therapy is designed to “make near-normal blood-clotting ability possible by addressing the underlying cause of hemophilia B: a faulty gene that causes a deficiency
Biologics
The Informed Consent Action Network (ICAN) is petitioning FDA to revoke the 5/10/21 reissuance of an emergency use authorization (EUA) for Pfizer/BioNTech’s Covid-19 vaccine in children ages 12 through 15. ICAN also asks FDA not to authorize Moderna’s request for EUA of its Covid-
Biologics
ImmunityBio has submitted a BLA for N-803, which it describes as a first-in-class IL-15 superagonist, plus Bacillus Calmette-Guérin (BCG) for treating BCG-unresponsive non-muscle invasive bladder cancer carcinoma in situ. The submission is based on data from the company’s pivotal QUILT 3032 study
Biologics
Pfizer and BioNTech say three doses of their Covid-19 vaccine had a strong immune response, high efficacy, and favorable safety in children 6 months to under age 5. “Our Covid-19 vaccine has been studied in thousands of children and adolescents and we are pleased that our formulation for
Biologics
FDA has amended the emergency use authorization granted for the Pfizer/BioNTech Covid-19 vaccine to authorize the use of a single booster dose in children 5-11 years old. The booster shot is to be given at least five months after completion of a primary series with the vaccine,
Biologics
Dark Horse Consulting Group has submitted to FDA a proposed draft guidance, Testing of Adeno-Associated Viral (AAV) Vector-Based Human Gene Therapy Products for Empty Capsids During Product Manufacture. The company says the development of the draft guidance was led by former CBER CMC reviewer Donald Fink, who has
Biologics
CBER director Peter Marks wants to continue the “Operation Warp Speed” attention and quick approval process given to Covid-19 vaccines after the public health emergency has ended and apply the processes to certain priority products. Speaking at an Alliance for a Stronger FDA briefing 5/10, Marks said a
Biologics
FDA has approved an ADMA Biologics request to extend the expiration date from 24 to 36 months for Bivigam (immune globulin intravenous), indicated for treating primary humoral immunodeficiency. An FDA notice says the new expiration date is valid for 120 Bivigam lots that were manufactured and distributed in 2020-2022.
Biologics
CBER has updated its calendar year 2022 guidance agenda to list 17 guidances the Center is considering for development this year. The revised agenda lists eight topics under Blood and Blood Components, seven topics under Tissues and Advanced Therapies, and two topics under Vaccines. The previous agenda projected 13 guidances,
Biologics
A staff report prepared for the House Committee on Oversight and Reform and the House Select Committee on the Coronavirus Crisis details manufacturing issues at the Emergent Biosolutions Bayview vaccine manufacturing facility that the report says wasted hundreds of millions of taxpayer dollars and affected the country’s ability to
Biologics
CBER director Peter Marks briefed the House Select Subcommittee on the Coronavirus Crisis 5/6 and denied news reports the agency was planning to delay authorizing Moderna’s Covid-19 vaccine for children under five years old so that it can be considered at the same time as Pfizer’s
Biologics
FDA 5/5 limited the emergency use authorization (EUA) of Janssen’s (Johnson & Johnson) Covid-19 vaccine to individuals 18 years of age and older for whom other Covid-19 vaccines are not accessible or clinically inappropriate, citing its risk of thrombosis with thrombocytopenia syndrome. It is also indicated
Biologics
As FDA begins to move into the next phase of the Covid-19 public health emergency, it is positioning the agency and public to accept that Covid infections will be with us for the foreseeable future and will likely become an annual concern like seasonal influenza. Writing in a 5/
Biologics
June is shaping up to be one of the busiest months in recent FDA history for agency vaccine reviews and associated meetings by its Vaccines and Related Biological Products Advisory Committee. The agency has set tentative advisory committee dates in anticipation of receiving complete emergency use authorization (EUA) submissions. The
Biologics
Moderna is asking FDA for an emergency use authorization for its mRNA-1273 Covid-19 vaccine in children 6 months to under 2 years and 2 years to 6 years old. A company statement says results from the Phase 2/3 KidCOVE study showed “a robust neutralizing antibody response in
Biologics
The House Select Subcommittee on the Coronavirus Crisis is asking FDA for a briefing on the status of coronavirus vaccine candidates for young children. A 4/25 letter says “millions of young children still remain unprotected because no vaccine has yet been authorized for children under five.” The select subcommittee
Biologics
A 4/21 letter signed by 69 experts working in academic centers, companies, and non-profit organizations is urging FDA to include a recommendation for T-cell assessment in its guidance to vaccine developers for Covid-19 vaccine trials. The letter says that “measuring T-cell responses in addition to
Biologics
NBC News says two people familiar with the plans have indicated that FDA could authorize a Covid-19 vaccination for children under age 5 in June. The report says the agency has asked members of its Vaccines and Related Biological Products Advisory Committee to set aside several days in June
Biologics
The CBER Office of Compliance and Biologics Quality says its review of a Web site and social media sites maintained by the Regenerative Stem Cell Institute, Elgin, IL, determined that the firm markets cellular products derived from adipose tissue to treat diseases or conditions such as cerebral palsy, asthma, emphysema,