Pink Sheet - News And Expert Analysis On Pharma Policy And Regulation since 1939

The agency wants sponsors to design clinical trials more carefully where overall survival is not the primary endpoint.

Convening an advisory committee before a formal proposal to withdraw has been the standard practice to date, but under FDORA it allows the agency to streamline the withdrawal process. The new accelerated approval draft guidance also codifies the practice of tapping other product center leaders to serve as decision-makers in withdrawal disputes.

Strategies championed by the Oncology Center of Excellence, including early planning and use of a single randomized study to support accelerated approval and later verify clinical benefit, are reflected in a new draft guidance broadly applicable to drugs and biologics.

New draft guidance discusses the data necessary for deciding whether an effect on a surrogate or intermediate clinical endpoint is reasonably like to predict clinical benefit and can support accelerated approval.

As efforts to improve diversity in clinical trials gain momentum globally, the Pink Sheet asked regulators in the UK, the EU, Canada, Australia and Japan about their efforts to support representative enrolment. 

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.

In this third article of a series on new drug reimbursement recommendations by the HTA body NICE, the Pink Sheet finds that fewer innovative medicines are reimbursed in England than in eight other European nations.

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

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.

Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.

The European Commission’s proposed pharma legislation overhaul could expand the definition of a gene therapy medicinal product, posing challenges both from a regulatory and public perception perspective.

The European Parliament proposed introducing joint procurement for orphan medicinal products in the EU pharma legislation overhaul – a move that the new CEO of EURORDIS says could lead to “faster and more equitable” access to drugs for rare diseases.

The head of the Korea Regulatory Science Center talks exclusively to the Pink Sheet about why regulatory science is crucial, the country's efforts to improve in the area and the center's strategic bridging role.

Greater transparency around EU health technology assessment processes “can only be a good thing” for innovative rare disease therapies, Virginie Bros-Facer, the new CEO of the EU network of rare disease patient organizations, EURORDIS, tells the Pink Sheet.