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FDA Liked What It Saw In Kalydeco’s Development Program

 
• By Sue Sutter

Agency reviewers looked favorably upon the pivotal trials’ primary efficacy endpoint and low drop-out rate, as well as the overall quality of the NDA submission.

While robust efficacy and a relatively clean safety profile were the basis for FDA approval of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco (ivacaftor), a solid development program unencumbered by problems certainly worked to the company’s advantage, particularly when it came to speeding the compound through the NDA review process.

Agency review documents suggest FDA saw a high-quality development program that generally followed the review division’s advice and produced robust data.

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