Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.

FDA’s Center for Biologics Evaluation and Research saw IND clinical holds for gene and cell therapies dropped to 70 in calendar year 2022, a 52% decline from 2018. Center for Drug Evaluation and Research’s total clinical holds in fiscal year 2022 were 380, the highest number in the past 12 years.

Pink Sheet’s Drug Review Profile looks at the timeline behind the development and FDA review of CellTrans’ treatment for type 1 diabetes.

Although the CRL delayed the cell therapy’s approval by almost two years, additional CMC-related testing requested by the FDA ultimately came to be seen as a good thing by sponsor CellTrans because it provided assurance of product consistency.

A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.

The median approval time for new drugs in Switzerland lengthened in 2022, mainly because of applications filed during the peak of the COVID-19 pandemic, says the country’s regulator.

A newly-translated guidance document from Japan’s drug and device regulator outlines the points that clinical trial sponsors should consider when using electronic methods to collect informed consent from participants.

Rhythm is working with country-level authorities and centers of excellence in Europe to gain reimbursement and access for its obesity treatment Imcivree.

A move to cut senior FDA positions including CDER Director through the appropriations process is rare and almost certain to fail, but experts say the attempt is indicative of an environment that seems to be encouraging these personal amendments.

Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.

New tendering rules in Belgium aim to improve competition between biosimilars and originators, for example by setting contract durations for tendering contracts and limiting the criteria for such contracts.

The Health Research Authority has developed UK-wide Quality Standards and Design and Review Principles to improve the information given to people who are invited to take part in research. Their use will become mandatory in December.

Sponsor’s assertion of a ‘floor effect’ cannot explain the lack of difference versus placebo on clinical endpoints, and there is no evidence that assessed biomarkers are reasonably likely to predict clinical benefit, agency says. Although advisory committee will focus primarily on efficacy, FDA also cites concerns about ‘grossly deficient’ product quality information.

FDA’s Center for Biologics Evaluation and Research saw IND clinical holds for gene and cell therapies dropped to 70 in calendar year 2022, a 52% decline from 2018. Center for Drug Evaluation and Research’s total clinical holds in fiscal year 2022 were 380, the highest number in the past 12 years.

Japanese originator Takeda has agreed to settle two-year-old anticompetitive claims lodged by wholesalers in the US that it conspired with several generics manufacturers to maintain supracompetitive pricing for its Colcrys branded treatment for gout.

Democrats and Republicans in the US Congress are not getting along on most topics these days. But when it comes to criticizing the pharmacy benefit management sector, they see eye-to-eye.

EU marketing authorization applications have been submitted for a number of new drugs, including Eli Lilly’s donanemab, Italfarmaco's givinostat and Arcturus Therapeutics/CSL’s ARCT-154.

A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.

The World Health Organization has published a “step-by-step guide” for countries wishing to increase their capacity for monitoring pathogens with pandemic and epidemic potential. The EU and the UK have cleared an updated version of Moderna’s COVID-19 vaccine Spikevax targeting the XBB.1.5 variant, while the EU has followed the US in approving Gilead’s Veklury as the first treatment for COVID-19 patients with mild-to-severe liver damage.

Robust natural history data sets with well-chosen outcomes are fundamental in Aetion’s regulatory feasibility tool for real-world evidence, built through analysis of US FDA case studies and guidance.