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Amylyx Relyvrio: Pricing, Compounding, And The Shadow Of Makena

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.

Approvals Intellectual Property

Keeping Track: US FDA Clears Santen’s Omlonti, Fennec’s Pedmark; Lilly’s Retevmo Adds Tumor-Agnostic Claim

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

US FDA Performance Tracker Approvals

Biogen/Eisai’s Lecanemab Suggests A New Model For Accelerated Approval In The US

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?

Review Pathway Approvals

Recent And Upcoming FDA Advisory Committee Meetings

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

Advisory Committees Drug Review

Pink Sheet Podcast: Drug Fix: ALS Drug Cleared, Lecanemab’s Hopeful Alzheimer’s Data, CMS’ Price Negotiation Needs

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.

Approvals Clinical Trials

October Outlook For US FDA Approvals: Goal Dates Are Sparse, But Backlog Looms

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

US FDA Performance Tracker Drug Review

OIG Accelerated Approval Report Spotlights Financial Costs Ahead Of Congressional Debate

First OIG report on FDA’s accelerated approval program as part of Aduhelm-spurred review doesn’t break new ground but will likely bring new wave of focus on the costs incurred to the government when drug companies fail to complete confirmatory trials in a timely manner just as reform advocates are hoping to muster enough attention on the topic to get legislative change this year.

FDA Clinical Trials

Amylyx's Relyvrio: The Road To Regulatory Flexibility And US FDA Approval

A Pink Sheet timeline shows the regulatory highs and lows of AMX0035's path to US approval for treatment of amyotrophic lateral sclerosis.

Approvals Drug Review

Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’

The single positive Phase II CENTAUR study, along with confirmatory evidence of benefit on long-term survival in the open-label extension, demonstrate substantial evidence of effectiveness, the agency says, acknowledging a level of uncertainty that is acceptable in the disease context.

Approvals Drug Review

How Will Approval Of Amylyx’s Relyvrio Impact The Ongoing PHOENIX Trial?

Launched in part to help resurrect AMX0035’s regulatory prospects, the multiregional PHOENIX study is winding down in the US. There still may be some risk of potential dropouts, however.

Approvals Clinical Trials

Medicare Part B Premium Reduction In 2023 Has Reserves Built In If Lecanemab Launches

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

Reimbursement Medicare

Eager But Wary: Industry Seeks More Useful, More Detailed Remote Regulatory Assessment Processes

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.

Manufacturing Quality
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