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.

CSL Behring said it remains confident that a market access agreement for its hemophilia B gene therapy Hemgenix can be reached after England’s HTA body NICE rejected the product because of uncertainties over its long-term benefit and cost-effectiveness.

The drug-pricing watchdog gave the gene therapy for metachromatic leukodystrophy an “A” grade for use in presymptomatic patients receiving the therapy, which already is approved in Europe.

Biopharma fears that the Joint Clinical Assessment, a first step to harmonizing health technology assessments in the EU, is not fit for purpose for cell and gene therapies and will increase rather than eliminate market access delays.