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BrainStorm’s NurOwn Gets Many, Many Thumbs Down From US FDA Panel

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.

Advisory Committees Drug Review

Imbruvica, Enbrel, Stelara Offer Best Chance Of Savings From Medicare Negotiation In First Round

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.

Reimbursement Pricing Debate

FTC Commissioner Recusal Decisions Should Be Public, US Chamber Of Commerce Says

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.

Enforcement M & A

Preparing For A Shutdown: US FDA To Retain 81% Of Workforce, Thanks Mostly To User Fees

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

FDA Legislation

Republican Lawmaker Takes Unusual Step Of Attacking FDA Salaries Over Abortion Pill Decisions

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.

Politics Legislation

FTC Merger Guidelines Cast ‘Perilous Shadow’ Over Biopharma

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.

M & A Enforcement

NurOwn Adcomm Is 20 Members Strong Despite US FDA’s ‘Struggle’ To Find Unconflicted Neurology Experts

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.

Advisory Committees Regenerative Medicine

BrainStorm’s NurOwn: Cell Therapy Lacks Substantial Evidence Of Efficacy In ALS, US FDA Says

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.

Advisory Committees Regenerative Medicine

Gene And Cell Therapy Clinical Holds Decline While Those For Drugs Hit 12-Year High

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.

Gene Therapy Drug Safety

Takeda Pens Mid-Trial Settlement To Dismiss US Colcrys Antitrust Suit

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.

Legal Issues Deals

Yet Another Bad Day For PBMs On Capitol Hill

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.

Pricing Debate Reimbursement

Finding The Sweet Spot For Real-World Evidence: Aetion Stresses Early Feasibility Assessment

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.

Real-World Evidence Drug Approval Standards

FDA Gene Therapy Office Chief Prefers Flexibility With Accelerated Approval Confirmatory Trials

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.

Gene Therapy Clinical Trials

Keeping Track: Return & Renewal At US FDA

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.

US FDA Performance Tracker Drug Review

CDC Panel Backs Pfizer’s Maternal RSV Vaccine But Leaves Room For AstraZeneca/Sanofi’s Beyfortus

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.

Advisory Committees Vaccines

Intarcia’s Implantable GLP-1 Gets Unanimous No Vote But Lots Of New Trial Design Advice

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.

Advisory Committees Drug Safety
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