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.

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

Givinostat, Italfarmaco’s investigational treatment for Duchenne muscular dystrophy, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

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.

Securing a place on the European Medicines Agency’s priority medicines scheme is not easy, but some companies have managed to make the grade more than once.

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.

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.

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.

However, FDA officials appear skeptical that imaging-based and circulating biomarkers are ready for prime time when it comes to supporting accelerated approval of drugs for noncirrhotic nonalcoholic steatohepatitis.

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

The European Medicines Agency has recommended new uses for 11 approved medicines, including an antibody drug conjugate for treating advanced non-small cell lung cancer.

The European Medicines Agency has OKd nine new medicines for EU-wide approval, but recommended against renewing the conditional marketing authorization of two drugs. 

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.

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.

However, FDA officials appear skeptical that imaging-based and circulating biomarkers are ready for prime time when it comes to supporting accelerated approval of drugs for noncirrhotic nonalcoholic steatohepatitis.

Draft guidance describes seven types of confirmatory evidence that can be used with one adequate and well-controlled clinical study to demonstrate substantial evidence of effectiveness, including evidence from expanded access, real-world data, and studies of other drugs in the same class.

CDC’s universal recommendation for XBB COVID vaccines means that Novavax’s protein-based vaccine can enter the market immediately upon FDA authorization, but some final questions from the agency are keeping the review going.

A restrictive label could be the result of the lukewarm endorsement from the majority of the agency’s Cardiovascular and Renal Drugs Advisory Committee for use of Onpattro for the treatment of ATTR-associated cardiomyopathy.

Twelve applications with September goal dates include six novel agents but only one breakthrough designation.

The US FDA is free from the need to reconsider Accelerated Approvals in a formal hearing in front of an advisory committee. But now the agency is now trying that approach to hear an appeal of a rejected application.

Sterile drug manufacturers get one year to comply with most revisions in updated EU, PIC/S and WHO GMP Annex 1 guideline. PDA survey hints at scale of challenge facing firms that still use traditional open filling lines, a practice expected to end under the Annex 1 revisions.

Paralleling the challenges of the wet AMD market it hopes to enter, the CRL for Lytenava cites manufacturing issues, which Outlook Therapeutics feels it can easily address, as well as the need for confirmatory clinical evidence, which has left the firm scrambling for a meeting with FDA.

Pink Sheet analysis finds big jump in the share of novel approvals that received PDUFA extensions, largely driven by clinical and safety data submissions.

Shared REMS, diverse confirmatory trials and manufacturing delays are among the highlights in the latest US FDA drug approval news from the Pink Sheet’s US FDA Performance Tracker.