Review Pathways
India is pressing ahead with “big shifts” in its regulatory approach for cell and gene therapies. Reduced “layers of review”, tighter internal timelines, upcoming guidelines to facilitate CGT development and reforms in the constitution of expert committees are some of the key changes.
The African Medicines Agency is to appoint a director general and become operational at the end of this month.
Antabio’s next-generation, broad-spectrum antibacterial, MEM-ANT3310, has made it onto the European Medicines Agency’s priority medicines scheme. The company says its product is designed to make a significant impact on the growing problem of antimicrobial resistance.
The new telework policy returns to the pre-COVID-19 pandemic standard, but still requires reviewers to be at White Oak for sponsor meetings and divisions to have an in-office presence every day.
The former principal deputy commissioner said the “decapitation” of senior leadership will make resolving internal disputes more difficult, which will slow application reviews. Woodcock also called the FDA layoffs a slow-moving catastrophe.
The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.
Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.
The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm clinical benefit.
Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.
Levodopa/carbidopa (ND0612), Mitsubishi Tanabe Pharma’s investigational drug-device combination therapy for the treatment of motor fluctuations in people with Parkinson’s disease, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
An enhanced role for patients in the European Medicines Agency was a key proposal within the EU pharmaceutical legislation overhaul – but patient groups warn this provision could be scrapped or weakened due to ongoing negotiations.
The UK drug regulator has made a number of improvements to its innovative licensing and access pathway, including a more “core” role for National Health Service partners, which is expected to facilitate flexible commercial discussions around drug reimbursement.
The European Medicines Agency is considering whether apitegromab, brensocatib and diazoxide choline, from Scholar, Insmed and Soleno respectively, are drugs that are of potential major public health interest, particularly from the point of view of therapeutic innovation.
The agency sent the neuroscience-focused company a refuse-to-file letter for Wakix as a treatment for Idiopathic Hypersomnia, a disease with only one approved treatment and few others in development.
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.