Biologics
FDA has published a guidance, Blood Pressure and Pulse Donor Eligibility Requirements—Compliance Policy, addressing the regulatory requirements for determining donor eligibility that apply to establishments that collect blood and blood components for transfusion or for further manufacturing use. The document says the agency has developed the guidance in response
Biologics
FDA has accepted for priority review an Atara Biotherapeutics BLA for gene therapy tabelecleucel (tab-cel) indicated as monotherapy for treating adult and pediatric patients two years of age and older with Epstein-Barr virus (EBV)-positive post-transplant lymphoproliferative disease who have received at least one prior therapy. The
Biologics
FDA has published a guidance, Application User Fees for Combination Products, explaining how fees are assigned for combination products reviewed in a single application or separate applications for each component. The document says the guidance replaces one issued in 4/2005 to provide consistency with current user fee programs and
Biologics
CBER says a Bioresearch Monitoring Program remote regulatory assessment of the Massachusetts Institute of Technology Committee on the Use of Humans as Experimental Subjects institutional review board (IRB) conducted between 5/30 and 6/9/2023 found “objectionable conditions” that were repeated observations from previous reviews. A just-released 6/
Biologics
FDA has granted Bayer-owned Asklepios BioPharmaceutical (AskBio) a fast track designation for AB-1005, a gene therapy candidate for treating moderate Parkinson’s disease. The investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy is designed for stable and continuous expression of
Biologics
FDA has issued Novo Nordisk a complete response letter (CRL) on its BLA for once-weekly basal insulin icodec, indicated for treating diabetes mellitus. In the letter, the agency requested more information about the manufacturing process and the Type 1 diabetes indication before the application’s review can be completed,
Biologics
UniQure says it will meet soon with FDA to present updated interim data, including up to 24 months of follow-up data from 29 treated patients, from its ongoing U.S. and European Phase 1-2 clinical trials of gene therapy AMT-130 for treating Huntington’s disease. The company
Biologics
CBER has published an updated guidance agenda for 2024. The revised document lists the 20 guidance topics the Center is planning to publish in 2024 in three categories: Blood and Blood Components (6 guidances); Therapeutic Products (13 guidances); and Other (1 guidance).
Biologics
Several law firms have posted analyses of the Supreme Court Loper Bright decision that overturned the 40-year precedent of deference to agency interpretations of ambiguous laws first announced in Chevron. A Sidley Food, Drug, and Medical Device Update says the Supreme Court decision to overturn Chevron deference to agencies
Biologics
FDA has issued Rocket Pharmaceuticals a complete response letter on its BLA for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector-based gene therapy for treating severe leukocyte adhesion deficiency-I (LAD-I). According to the company, the agency letter requested “limited additional” chemistry manufacturing and controls (CMC) information to
Biologics
A 1/22-2/1 FDA inspection at the Indiana Lions Eye Bank, doing business as VisionFirst Indiana Lions Eye Bank in Carmel, IN, found significant deviations from the regulations for human cells, tissues, and cellular and tissue-based products. A 6/10 Warning Letter says the “items of concern”
Biologics
A House Judiciary subcommittee interim staff report issued 6/25 claims to detail “how the Biden administration pressured FDA to go beyond its regulatory authority to change its procedures, cut corners, and lower agency standards to approve the Pfizer Covid-19 vaccine and authorize boosters. This approval enabled the Biden
Biologics
CBER director Peter Marks has again overridden his Center’s review teams in expanding the limited approval of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy Elevidys to full approval. On 6/20, Marks signed off on the supplemental BLA and dismissed reviewers’ objections to broaden Elevidys’ use to about
Biologics
FDA is moving away from recommending switching studies to support an interchangeability determination for biosimilar products. In a just-published draft guidance, the agency is seeking comments on a revised approach where such studies will generally not be needed. FDA’s recommendation for switching studies first appeared in its interchangeability
Biologics
FDA has granted Merck accelerated approval for Capvaxive (Pneumococcal 21-valent conjugate vaccine) for: * Preventing invasive disease caused by Streptococcus pneumoniae serotypes 3, 6A, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15B, 15C, 16F, 17F, 19A, 20A, 22F, 23A, 23B, 24F, 31, 33F and 35B in individuals 18 years of
Biologics
Novavax has submitted an amendment to its Emergency Use Authorization for an updated JN.1 Covid-19 vaccine (NVX-CoV2705) for individuals aged 12 and older. “The submission is in line with guidance from the U.S. FDA, European Medicines Agency (EMA) and the World Health Organization (WHO) to target
Biologics
FDA is updating its advice on this fall’s Covid-19 vaccine composition based on a recent review of circulating Covid cases in the U.S. “Based on the most current available data, along with the recent rise in cases of Covid-19 in areas of the country, the agency
Biologics
Pfizer says that its Phase 3 CIFFREO placebo-controlled study evaluating an investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy did not meet its primary endpoint of improvement in motor function. The primary endpoint was assessed by the change in the North Star Ambulatory
Biologics
FDA has recommended that Toronto, Canada’s Revive Therapeutics submit an IND for agency evaluation of the company’s bucillamine, described as an oral thiol-based drug with anti-inflammatory and antiviral properties, as a potential treatment for Long Covid. In a Type C meeting with company executives, FDA officials
Biologics
FDA has approved an expanded use for GSK’s Arexvy (respiratory syncytial virus (RSV) vaccine, adjuvanted) for preventing RSV lower respiratory tract disease (LRTD) in adults aged 50 through 59 years and who are at increased risk. The vaccine was previously approved for use in adults aged 60 and older.
Biologics
Replimune Group is planning a BLA for its RP1 therapy in combination with nivolumab for treating melanoma patients who’ve failed an anti-PD1 therapy based on just-released data from its IGNYTE clinical trial. The results showed that one-third of patients receiving RP1 plus nivolumab responded to treatment,
Biologics
FDA has published a draft guidance, Standardized Format for Electronic Submission for Marketing Applications Content for the Planning of Bioresearch Monitoring (BIMO) Inspections for Center of Biologics Evaluation and Research Submissions, that applies to electronic submissions of data and information from pivotal studies used to support safety and efficacy claims