The latest drug approval news and highlights from the Pink Sheet’s US FDA Performance Tracker

Why not wait for the confirmatory trial to read out before approval, at least in the context of an especially uncertain or controversial surrogate marker?

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

Pink Sheet reporters and editors discuss the newly approved ALS drug Relyvrio, the promising data on the Alzheimer’s drug lecanemab, and CMS’s efforts to build the drug price negotiation program.

Investigational products from Minoryx and a host of other companies targeting a range of diseases are now under review by the European Medicines Agency for potential EU approval.

Upcoming user fee goal dates include AstraZeneca’s tremelimumab, Amicus’ AT-GAA, and drug delivery technology-driven NDAs from Supernus/US Worldmeds and scPharmaceuticals

With the fourth quarter just around the corner, China's closely-watched annual drug reimbursement and prices negotiations are entering a key final stage. But will applicants need to be more strategic than expedient?

But premiums in future years made need to keep pace with accelerating Medicare spending on Alzheimer’s drugs.

Brazil’s health technology assessment institute is to incorporate cost-effectiveness thresholds into its decision making.

Pink Sheet reporters and editor discuss the impact of the long wait for a user fee deal to be negotiated, how the FDA will interpret President Biden’s statement that the COVID-19 pandemic is over, and concerns with the clinical development of the monkeypox treatment Tpoxx.

Investigational products from Ultragenyx and MinervaX secured a place on the European Medicines Agency’s priority medicines scheme this month.

Increases in approved ANDAs that were affected by GDUFA research shows the importance of the US FDA’s regulatory science program.

The new ALS product is a fixed-dose combo of two ingredients – sodium phenylbutyrate and taurursodiol – that some patients currently are compounding. Amylyx says it will explore ‘all options’ in addressing that, noting its strong IP for Relyvrio, but also committing to access.

Excellence, innovation, deal-making and individual achievement across the Japanese biopharma sector were celebrated at the inaugural Pharma Intelligence Japan Awards 2022 in Tokyo on 1 September.

Moderna seeks damages for sales of Comirnaty in US and other wealthy countries from 8 March onward, a timeframe driven by Moderna's accessibility pledge. Suit claims Pfizer and BioNTech copied two key features of its mRNA technology for their rival COVID vaccine.

The new ALS product is a fixed-dose combo of two ingredients – sodium phenylbutyrate and taurursodiol – that some patients currently are compounding. Amylyx says it will explore ‘all options’ in addressing that, noting its strong IP for Relyvrio, but also committing to access.

A senior Parexel executive and ex-FDA pharmaceutical quality assessor emphasizes at a conclave why “first impressions matter now more than ever” as the agency  deploys complex alternate inspections tools which the pandemic provided, and that companies need to ensure that they get things right the first time.

Responses to US FDA draft guidance suggest more promptness while also more elaborate processes for agency decision making based on remote methods. Questions include what’s voluntary, what’s mandatory, what can be decided remotely, and how to remotely move on from warning letters.