As amended, the Give Kids A Chance Act would renew the rare pediatric disease voucher program for five years and give the FDA new authority to bring enforcement action when pediatric studies are overdue. However, it would not remove the exemption from pediatric study requirements for non-cancer orphan drugs, which Democrats had sought. 

Studies should enroll a representative subgroup of US patients based on the incidence or prevalence of cancer in the US, and the comparator arm should use the US standard of care when possible, the FDA says in draft guidance.

Obeticholic acid has not confirmed clinical benefit and the benefit-risk profile is not favorable in primary biliary cholangitis, the FDA advisory committee said. The agency must now decide whether to keep Ocaliva on the market with new study requirements or seek withdrawal.

Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.

The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.