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Secondary Endpoints Backed Kalydeco’s Efficacy, Despite Lack Of Correlation With Pulmonary Function

 
• By Sue Sutter

Although a decrease in sweat chloride levels was suggestive of benefit, this did not correlate with improvements in FEV1 function, FDA said. The agency also noted that the patient-reported outcomes instrument used in the pivotal clinical trials, though not fully validated, measured respiratory symptoms that are clinically relevant to patients.

Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco (ivacaftor) demonstrated generally positive effects on a slew of secondary endpoints in two pivotal trials, and yet it remains a question mark how, and whether, some of these measures translate into clinical benefit.

During the course of their review, FDA staff raised questions about the clinical meaningfulness of changes in sweat chloride and...

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More from United States

Podium Policy Returns To US FDA, Or Is It Podcast Policy?

 
• By Michael McCaughan

US FDA critics have long decried the practice of using informal communications to convey regulatory expectations as podium policy. In the Makary era, the phrase may need to be updated because the agency now seems to be specializing in policy by podcast.

US FDA’s Elsa AI Platform Is Not Guiding Regulatory Decisions, Official Says

 
• By Cathy Kelly

Tala Fakhouri said the agency’s use of the AI tool in the review process has generated lots of questions.

Branch Chief Vijay Kumar Named Acting Head Of US FDA’s Cell, Gene Therapy Office

 
• By Derrick Gingery

Kumar takes over as acting director of the Center for Biologics Evaluation and Research’s Office of Therapeutic Products days after director Nicole Verdun was ousted.

Recent And Upcoming FDA Advisory Committee Meetings

 
• By Sue Sutter

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

More from North America

BIO Notebook: Policy Uncertainty Constraining Dealmaking

 
• By Mandy Jackson and Joseph Haas

Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

Woodcock: Do Not Do The Wrong Study, Even If Against US FDA Advice

 
• By Sarah Karlin-Smith

The former CDER director said she tells sponsors not to conduct an FDA-recommended study design or randomized trial if it will not work.

AI Will Bolster, Not Squelch, Patient Voice In Clinical Research, US FDA Says

 
• By Sue Sutter

Artificial intelligence tools used for clinical research should be developed with input from patients and focus on their needs and abilities, FDA experts said.