However, the US FDA’s approval of donanemab comes with little guidance on when to stop treatment or when the anti-amyloid agent may need to be restarted in Alzheimer’s patients.

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.

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.

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.

Advisory Committee on Immunization Practices expected to vote 26-28 June on use of Merck’s 21-valent pneumococcal vaccine, Moderna’s mRNA vaccine for RSV, and an expanded age range for GlaxoSmithKline’s Arexvy RSV vaccine.

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.

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.

Feedback is being sought on revised guidelines that tell companies how they should make post-authorization changes to their drug approvals once new rules take effect in January 2025.

As of today, drug developers can apply to take part in two pilot programs that will involve regulators across the EU strengthening their coordination when it comes to providing companies with advice on their applications for clinical trials and marketing authorization.

Ninety-two approvals, seven revocations and myriad standard-of-care treatments are all discussed in a first-of-its kind study examining the data and nuances of oncology submissions processed under Health Canada’s 26-year-old notice of compliance with conditions (NOC/c) pathway.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add three new products, including Qalsody, Biogen's treatment for amyotrophic lateral sclerosis associated with a mutation in the superoxide dismutase 1 gene.

However, the US FDA’s approval of donanemab comes with little guidance on when to stop treatment or when the anti-amyloid agent may need to be restarted in Alzheimer’s patients.

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.

Rocket Pharmaceuticals’ gene therapy Kresladi, Daiichi Sankyo/Merck’s patritumab deruxtecan, and AbbVie’s foscarbidopa/foslevodopa (ABBV-951) received CRLs because of manufacturing concerns.

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 Center for Drug Evaluation and Research’s medical policy chief says artificial intelligence can aid patient recruitment and increase trial diversity, but warned of “unique” potential pitfalls for sponsors.

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.

The FDA commissioner said that supply chains will remain interconnected no matter how much nationalism dominates the political landscape, meaning international cooperation will still be necessary.

Agency should be more proactive in issuing communications after advisory committee meetings, stakeholders advised; public interest group and industry representatives said the voting process is important for transparency and accountability.

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.

The CDC's Advisory Committee on Immunization Practices voted for a universal COVID-19 vaccine recommendation for 2024-2025, but said the decision would have been easier with a lower-cost product.

The unpopular shared clinical decision-making policy for RSV vaccines in adults age 60 and older should be replaced by risk-based criteria in people age 60-74 and a universal age-based recommendation at 75 and older, the CDC’s Advisory Committee on Immunization Practices agrees.

Advisory Committee on Immunization Practices expected to vote 26-28 June on use of Merck’s 21-valent pneumococcal vaccine, Moderna’s mRNA vaccine for RSV, and an expanded age range for GlaxoSmithKline’s Arexvy RSV vaccine.

Agency should be more proactive in issuing communications after advisory committee meetings, stakeholders advised; public interest group and industry representatives said the voting process is important for transparency and accountability.

Many positions are vacant or filled by individuals with ties to academia, industry or patient groups, representatives from public interest and consumer groups say during an FDA listening session on advisory committee reforms.

The US FDA is considering labeling that calls greater attention to the symptoms of amyloid-related imaging abnormalities to ensure proper treatment and avoid misclassification as ischemic stroke.

The advisory committee also hails the treatment-to-amyloid clearance approach used in the Phase III trial as innovative, but questions how it should apply in clinical practice.

Safety concerns related to MDMA patient suggestibility and impairment troubled the panel reviewing psychedelic therapy for PTSD, although the FDA advised that therapist misconduct is outside its control.

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

Given the frequent occurrence of ARIA and an imbalance of deaths in the pivotal TRAILBLAZER-ALZ 2 study, the agency also will ask the Peripheral and Central Nervous System Drugs Advisory Committee whether there are patient subgroups where the benefit-risk is more or less favorable.

Advisory committee members said having Novavax’s protein-based vaccine available in the fall was important, but would not have been possible if a different variant strain were used. They also worried separate recommendations by vaccine type would cause confusion.

Functional unblinding and vague psychotherapy guidance lead to two negative votes for Lykos’ pioneering psychotherapy-assisted psychedelic therapy at the US FDA’s Psychopharmacologic Drugs Advisory Committee.