US

In overwhelmingly concluding that efficacy has not been demonstrated for the mesenchymal stromal cell therapy, panelists went beyond just the failed Phase III trial and cited BrainStorm’s ‘statistical magic’ and failure to adequately describe the mechanism of action, as well as safety and product quality concerns.

The other seven drugs on the initial list for negotiation are already heavily rebated and CMS could choose not to go much lower than current net prices, SSR Health report points out.

Chamber files petition to have FTC amend its disqualification rules to have commissioners explain their rejection of recusal requests. FTC is seeking public comments on the petition.

However, user fee carryover balances will not last forever and service cuts are inevitable if a shutdown lingers for several weeks.

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.

BIO objects to guidelines seeking to block mergers that would eliminate a potential entrant in a concentrated market and give greater scrutiny to transactions that are part of a series of company deals. APhA and NCPA call for FTC to consider more factors in PBM mergers.

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.

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.

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.

Ideally, the studies would be underway at the time of approval, as the US FDA’s new authority allows, but Office of Therapeutic Products Director Nicole Verdun sys there will be exceptions.

Takeda’s TAK-721 is back, ARS Pharma will be back soon to appeal neffy complete response letter, US FDA’s Project Renewal brings Temodar labeling in current day, and more highlights from the Pink Sheet’s US FDA Performance Tracker.

Abrysvo is recommended for use in pregnant people 32-36 weeks gestation if administered from September to January. For infants whose mothers were not vaccinated in this window, nirsevimab is recommended for prevention of respiratory syncytial virus infection.

US FDA advisory panel feels there could be a place for Intarcia’s exenatide drug-device combo – if the company is willing to put in the work.