Review Pathways
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among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.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.
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.
A total of 141 products have made it onto the European Medicines Agency’s PRIME (priority medicines) scheme since 2016 and over 370 applications have been denied entry.
India continues to reform biopharma regulatory processes and widen digitization efforts. The regulator will curb "re-deliberations" at subject expert committee meetings unless applicants furnish new data.
The company said its financial viability is in jeopardy, along with patient access to Lyfgenia, if US FDA Commissioner Robert Califf does not reverse the denial of a rare pediatric disease priority review voucher.
Advanz Pharma, the company that markets Ocaliva for primary biliary cholangitis in the EU, will continue to supply the drug on a compassionate use or named patient basis.