In pivotal trials for Alnylam’s Onpattro and Ionis’ Tegsedi, statistically persuasive results on a patient-reported, quality-of-life measure were needed to show the drugs had a meaningful benefit on how hATTR patients with polyneuropathy feel and function, US FDA said.

Chronicle of the development and US FDA review of Ionis Pharmaceuticals’ inotersen for treatment of polyneuropathy of hATTR amyloidosis.

Chronicle of the development and US FDA review of Alnylam’s patisiran for polyneuropathy of hATTR amyloidosis.

The Pink Sheet takes a closer look at recent clinical announcements showing how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.

Janssen, Krystal Biotech and Takeda are the latest companies to win a place on the European Medicines Agency’s popular priority medicines scheme for getting drugs for unmet medical needs through the review process faster.

In sharp disagreement, FDA says postmarketing trial shows risk of taking alcohol with the female sexual dysfunction drug; Sprout will appeal agency's decision.

Bernstein Research analysts said CMS could charge pharmaceutical manufactures a fee to offset increases in insurance premiums anticipated from the elimination of rebates in a "win-win" move.

Faced with a proposed regulation that aims to eliminate rebates and an upcoming hearing on Capitol Hill, US pharmacy benefit managers including OptumRx are working to validate the concept of rebates when redirected to patients at the point-of-sale. 

FDA asks about regulating foreign firms more and destroying drugs at the border, and questions firms about particles and aseptic practices.

FDA announces the rollout of a pilot program with industry to exchange data on quality specifications for the Common Technical Document’s Module 3. The agency plans to exchange data on other portions of the CTD’s quality section later this year. The data exchange would largely replace the “hunting and pecking” for information in PDFs that reviewers have to do to locate quality information in NDAs and ANDAs.

Major pharmaceutical industry groups in the US, like their counterparts in the EU, are concerned that the draft ICH Q12 guideline does not align with the established legal framework in certain ICH regions, including the EU, in terms of implementing key concepts such as established conditions and product lifecycle management.