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Defining REMS Success An Unanswered Question For Daiichi's Pexidartinib

 
• By Derrick Gingery

US FDA still deliberating on how best to measure success of the liver safety program for Daiichi's TGCT treatment, but REMS will probably assess both process and outcomes. 

Liver cancer, CT scan upper abdomen.
FDA wants to collect long-term data on pexidartinib's liver toxicity risks as part of a Risk Evaluation and Mitigation Strategy. • Source: Shutterstock

The US Food and Drug Administration has yet to determine what would constitute successful risk management for Daiichi Sankyo Co. Ltd.'s proposed rare cancer treatment pexidartinib.

Pexidartinib is proposed for adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional

The product likely will need a Risk Evaluation and Mitigation Strategy with Elements to Assure Safe Use to ensure patients and prescriber understand the hepatotoxicity risks associated with it. However, FDA officials still are not certain how to measure success

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Opioid Safety: US FDA Hears Mixed Messages On Adding Posmarketing Studies To Labeling

 
• By Sue Sutter

Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.

US FDA Asks Adcomm If Opioid Postmarketing Studies Are Relevant, Warrant Action

 
• By Sue Sutter

Two US FDA advisory committees will discuss results from epidemiological studies that attempted to quantify the prevalence, incidence and risk factors for misuse, abuse, overdose and death with opioid analgesics.

US FDA Commissioner Makary Wants To Combine Adverse Event Reporting Systems

 
• By Sarah Karlin-Smith

In discussing FDA’s adverse event monitoring, Makary also seemed to falsely imply the agency did not fully investigate the myocarditis signal with COVID-19 vaccines.

Industry Urged To Respond To EU Expectations On Using Animal Testing Alternatives

 
• By Eliza Slawther

The chair of a European Medicines Agency working group on reducing animal testing said that companies can provide “thoughts and suggestions” in relation to a review of its scientific guidelines on regulatory testing requirements and new approach methodologies.

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Brazil To Upgrade Pricing Framework To Keep Up With Market Developments

 
• By Francesca Bruce

Brazil plans to clarify its medicine pricing framework, for example by outlining the procedures for setting medicine prices and bring it up to date with recent developments in the sector.

England: HTA Sandbox Helping To Assess ‘Challenging’ Drugs And Indications

 
• By Eliza Slawther

England’s health technology assessment institute explains how its sandbox environment is helping to test new evaluation methods for drugs or indications with which it does not have experience, such as metabolic dysfunction-associated steatohepatitis.

Unlocking Opportunities: How To Engage With The EMA On Animal Testing Alternatives

 
• By Neena Brizmohun

The European Medicines Agency, like its counterpart in the US, is increasingly focusing on the use of alternatives to animal testing.