Approvals

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.

Novo Nordisk’s application seeking EU marketing approval to use its GLP-1 receptor agonist to treat cirrhotic metabolic dysfunction-associated steatohepatitis is one of 15 new drug filings the European Medicines Agency has started reviewing. Meanwhile, Arrowhead’s filing for its familial chylomicronemia syndrome, plozasiran, is being fast tracked.

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Tivdak, Pfizer/Genmab’s treatment for previously treated recurrent or metastatic cervical cancer.

Eisai is working on securing reimbursement across the EU for Leqembi now that the Alzheimer’s disease treatment has secured marketing approval from the European Commission.

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.

Israel has introduced a new framework to expedite the marketing approval of medicines, make the country a more attractive destination for drug registration and help reduce drug prices through market competition.

Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.

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.

Japan recommends eight new drugs for approval, including two for cardiomyopathy, and will also raise prices for multiple products, including Enhertu, on 1 April.

Unofficial January results showed the US FDA continued to issue full and tentative ANDA approvals in line with previous months, but recent layoffs may not help the agency maintain that pace.

Congress continues to offer broad support for encouraging alternatives to opioid pain medicine, including proposals to speed coverage of new agents like Vertex’ Journavx.

March features nearly 20 user fee goal dates, including a crowd of novel candidates for crowded areas like hemophilia, heart disease and urinary tract infections, as well as the potentially first therapies for a rare eye disease and hyperphagia of Prader-Willi syndrome.

The European Medicines Agency is set to issue opinions this week on whether pan-EU marketing approval should be granted to a number of new products.