Commissioner Robert Califf urged a change in clinical trial diversity discussions, emphasizing the need to consider the realities of US leadership in a global drug development ecosystem.

US FDA review divisions will determine whether the meeting is appropriate because there are no one-size-fits-all requirements.

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

Sun’s deuruxolitinib and Novo Nordisk’s insulin icodec stand out as new molecular entities among the small cluster of US FDA user fee goal dates in July.

The June meeting of the European Medicines Agency's human medicines committee brought mixed fortunes for companies. 10 new products were recommended for pan-EU approval, two drugs were rejected, while the conditional approval of one drug is set to be revoked. PTC Therapeutics again faced disappointment regarding its Duchenne muscular dystrophy drug Translarna, but has vowed to fight back.

The US Medicare agency is not moving forward with a proposal to cut payments for drugs with accelerated approval status pending further discussions with the FDA about whether new regulatory authorities have addressed concerns about timely completion of confirmatory trials.

Regeneron, whose blood cancer treatment odronextamab was recently turned down by the US regulator but not for reasons relating to efficacy or safety, could soon learn whether its product will be among the drugs to get the thumbs up from the CHMP, the European Medicines Agency’s human medicines committee.

Failure of the EMBARK confirmatory trial convinced US FDA reviewers the Duchenne muscular dystrophy gene therapy did not show clinical benefit, but biologics center director Peter Marks invoked totality of evidence to grant full approval.

Sepiapterin, PTC Therapeutics' treatment for hyperphenylalaninemia in adult and pediatric patients with phenylketonuria, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

A Pfizer exec says that the old prescription drug user fee formulas that usually increase funding and staffing for many FDA programs no longer deliver the same return on investment.

Vyluma’s eyedrop for treating myopia in children and Vertex’s next-generation triple combination treatment for cystic fibrosis are among the latest drugs that the European Medicines Agency has begun reviewing for potential EU marketing approval.

With likely more fee-paying applications arriving at the agency, sponsors may be wondering whether user fees should have been increased as much as they were at the start of FY 2024.

With the much-anticipated guidance still pending, OCE’s Tamy Kim reminds industry of the requirements and timing around avoiding the soon-to-be requirements on study enrollments.

CBER's Lola Fashoyin-Aje offers some thoughts on when and how the FDA might consider the accessibility of a product in its approval decisions at BIO 2024.

The notice of opportunity for hearing on the US FDA’s proposal not to approve an insomnia sNDA for Vanda’s Hetlioz opens another front in the company’s aggressive campaign against complete response letters and generic competition to the sleep disorder drug.

Regulators from the US, Canada, European Union, Switzerland and Japan talk about the benefits of global collaboration, and reasons why they might reach different decisions on the same drug, at the American Society of Clinical Oncology annual meeting.

‘Short-term effects of midomafetamine were not captured’ in the clinical program for post-traumatic stress disorder, FDA notes. Proposed REMS for the psychedelic therapy to be discussed at 4 June would limit dispensing setting, restrict post-treatment driving.

June goal dates include RSV and pneumococcal vaccines, two COPD therapies, Rocket and Sarepta gene therapies, and lots of oncology.

Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.

Advances in cell and gene therapy, together with the forthcoming EU joint clinical assessments, have raised questions about the ethics of having to use randomized controlled trials to test new advanced therapies.

Stakeholders have until 30 June to comment on a set of common quality standards for mRNA vaccines that are said to be needed in light of rapid developments in this field and the emergence of novel vaccines coding for a range of specific antigens.

FDA oncology review staff discuss cases studies involving Amgen’s Lumakras and Novartis’ Pluvicto, where there was a perceived loss of equipoise, and how mitigation strategies helped save the latter’s pivotal trial.

As sponsors explore development in autoimmune and infectious diseases, plus a range of hematologic and solid tumors, manufacturing processes can be centralized or decentralized, but Marks says the FDA is ‘neutral’ on that decision.

Seeking to clear up a ‘misconception,’ Oncology Center of Excellence officials say earlier endpoints, such as progression-free survival and overall response, continue to be useful in getting therapies to market quicker, but sponsors still should plan to systematically collect OS data to ensure there is no detriment to survival.