Review Pathways

The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.

Companies that are admitted to the UK’s revised innovative licensing and access scheme will be mandated to work with the regulator on developing a target development profile for their product.

The inspector general said the council should be more involved in specific approval decisions when there is internal disagreement, but FDA said that would preclude some individuals from adjudicating a subsequent withdrawal proceeding.

Two recent accelerated approval guidances describe the FDA’s policy expectations for trial timeliness, but recognize the need for case-specific determinations. Regulatory actions in 2024 show the FDA is willing to delay application filing or approval over confirmatory trial status.

The European Medicines Agency’s chief medical officer, Steffen Thirstrup, said the surge in positive opinions adopted by the agency in 2024 for new marketing approvals signals a post-COVID recovery.

The MHRA’s new scientific dialog program will offer drug developers “closed-door meetings” that will offer “confidential, commercially sensitive discussion” with its staff to help them “refine” their real-world evidence generation strategies.

Vanda’s fight against a complete response letter for its gastroparesis drug results in a notice of opportunity for hearing on a formal FDA proposal to refuse to approve the NDA.

Regenerative medicines using expedited review pathways dominate novel approvals at the Center for Biologics Evaluation and Research, while the Center for Drug Evaluation’s higher volume comes with lower first-cycle approval rates and more standard reviews.

2024 saw important regulatory changes in India including in areas such as GMP, clinical trials and efforts to rein in unethical marketing practices. Further action is expected to play out in the new year as well.

The policies for China’s biopharma industry in 2024 centered around innovative small molecules, biologics and cell and gene therapies. Regulation changes for the industry in 2025 could be a continuation of that.

The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.

The US FDA drugs center cleared 50 novel agents and the biologics center contributed 11 novel biologic approvals. The agency also acted on 77 novel applications, including 16 complete response letters.

A Catalyst fix, a pediatric rare disease voucher reauthorization that should prevent future workload imbalances for FDA, and a boost for out-of-state coverage in Medicaid are among Congress’s year end commitments to pediatric and rare disease patients that now seem in doubt.

The European Medicines Agency has recommended several new products for pan-EU approval, including CSL Behring’s drug for hereditary angioedema and BridgeBio’s treatment for transthyretin amyloidosis in cardiomyopathy patients.

The FDA's view that current monitoring recommendations may not be sufficient to mitigate harm increases the likelihood it could seek withdrawal.

Clene’s ALS drug CNM-Au8 and UniQure’s gene therapy for Huntington’s disease emerged from US FDA discussions with accelerated approval plans using real-world evidence from compassionate use and natural history data sources.

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.

Sponsors of 13 new products, including BridgeBio’s acoramidis, could soon learn whether or not the European Medicines Agency recommends their drugs for pan-EU marketing approval.

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.