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Kalydeco Passed FDA’s Approval Test With The Help Of A Failed Study

 
• By Sue Sutter

FDA’s speedy approval of Vertex’s disease-modifying, genetically targeted cystic fibrosis treatment ivacaftor appears to have been made easier by the company’s failure to show efficacy in a population with a different genetic mutation. The failed study not only had important implications for preventing off-label use, it added considerably to the safety database in an orphan indication.

FDA’s review of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco (ivacaftor) reflected an unusual situation: a failed efficacy study actually bolstered the case for approval.

Ivacaftor, the first-disease modifying therapy approved for cystic fibrosis patients, targets the G551D “gating” mutation that only a small percentage of U.S. CF patients carry. Two pivotal trials in that genetically targeted population showed significant improvements in pulmonary function and other endpoints

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More from United States

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• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

US FDA Expands Surprise Foreign Inspections But Loses Associate Commissioner Michael Rogers

 
• By Sue Sutter

Commissioner Martin Makary’s repeated characterization of foreign facilities as being subject to lower standards than domestic counterparts may have contributed to Rogers’ decision to retire as head of the recently formed Office of Inspections and Investigations.

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• By Kate Rawson

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• By Sarah Karlin-Smith

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More from North America

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.

Viatris Agrees To $335m Opioid Settlement Ending US State, Local Government Cases

 
• By Dean Rudge

Viatris joined Hikma and Amneal, in signing agreements for cash settlements that resolve allegations its practices fueled the US’ deadly opioid epidemic.

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• By Jessica Merrill

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