Review Pathways
US FDA critics have long decried the practice of using informal communications to convey regulatory expectations as podium policy. In the Makary era, the phrase may need to be updated because the agency now seems to be specializing in policy by podcast.
US FDA Commissioner Martin Makary will establish "national priorities" used to select sponsors for the pilot of “tumor board-style” drug reviews that would start before Phase III is completed.
Despite following the path of the popular Project Orbis, the cell and gene therapy international collaborative review pilot is being reconsidered by the FDA's new management.
Key expert panel go-ahead with a trial waiver put’s Eisai's Alzheimer's therapy on track for a debut in India where tailored pricing will be pivotal. Lilly’s Kisunla is also under regulatory review.
Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.
The House Appropriations Committee wants an analysis of how issues that led to CRLs could have been resolved within the first review cycle and seeks an agency crackdown on counterfeit GLP-1 agonists.
The UK government has acknowledged that sponsors of clinical trials for rare diseases face challenges around patient recruitment and trial design, which will be addressed in its clinical trials reform, a lawyer says.
The developers of the ChAdOx1 NipahB vaccine will be able to benefit from the early and enhanced support that the European Medicines Agency offers through its priority medicines scheme.
Julian Beach, interim executive director of healthcare quality and access at the UK drug regulator, the MHRA, tells the Pink Sheet how the agency is responding to increased efforts to develop bacteriophage therapies.
The reductions across medical product programs in the agency’s budget request include hundreds of positions funded by user fees, which may foreshadow its upcoming user fee restructuring strategy.
A European Medicines Agency official provided an overview and update of the support the EMA offers developers of innovative products and discussed, among other things, its Innovation Task Force, Quality Innovation Group and a scientific advice pilot for clinical trials.
The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.
The agency said it will consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, but Stealth CEO Reenie McCarthy said announcing it via complete response letter is confusing and inefficient.
The European Medicines Agency says that a regulatory reliance pilot that it is supporting to speed up assessments of post-approval changes appears to be accelerating evaluation timelines in non-EU countries.
Sponsors can expect faster evaluation processes for key medicines and greater support in mitigating medicines supply shortages this year as part of the European Medicines Agency’s digital transformation overhaul.
Patients from Asia saw a differential treatment effect versus non-Asian regions in the Phase III multiregional study, with an adverse survival trend seen in the latter group, the FDA said about the lymphoma drug.
Pink Sheet sister publication In Vivo spoke to the former FDA commissioner about the recent layoffs and their impact on vaccine and other product development.
Brensocatib, Insmed's investigational treatment for non-cystic fibrosis bronchiectasis, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
Companies that use the European Medicines Agency as a reference regulator for the UK’s International Recognition Procedure can expect a faster approval than those that use other national regulators, such as those in the US and Canada, an MHRA spokesperson has said.
Silicon Valley investor Jim O’Neill’s Senate confirmation hearings showcased the unique background of someone with government and tech experience and avoided discussions of his past statements about lowering the bar for regulatory approval.