Expanded Indication for Cystic Fibrosis Treatment Rejected

Article

Vertex Pharmaceuticals has received a complete response letter from the FDA for its supplemental new drug application for ivacaftor.

Vertex Pharmaceuticals has received a complete response letter from the FDA for its supplemental new drug application for ivacaftor (Kalydeco).

The supplemental new drug application was for the use of Kalydeco as a treatment for cystic fibrosis in patients ages 2 or older who have 1 of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator gene.

In the complete response letter, the FDA stated that it cannot approve of the application in its current form. Vertex said it planned to meet with the FDA to determine the next steps.

"Our intention with this submission was to rapidly bring Kalydeco to additional people with cystic fibrosis who we believe may benefit," said Vertex Executive Vice President and Chief Medical Officer Jeffrey Chodakewitz in a press release. "We chose to pursue this approach given our strong belief in the science of cystic fibrosis and in the well-established safety of Kalydeco across many different groups of people with cystic fibrosis. We are disappointed by this decision and look forward to discussing with the FDA the next steps to bring Kalydeco to people with cystic fibrosis who have these residual function mutations."

The FDA approved Kalydeco last year as a treatment for patients with cystic fibrosis who have mutations in 1 of the following genes: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H.

Dizziness is one of the most common adverse effects associated with the drug, but it can also cause high liver enzymes in the blood of some patients.

Related Videos
Female Pharmacist Holding Tablet PC - Image credit: Tyler Olson | stock.adobe.com
African American male pharmacist using digital tablet during inventory in pharmacy - Image credit: sofiko14 | stock.adobe.com
Young woman using smart phone,Social media concept. - Image credit: Urupong | stock.adobe.com
selling mental health medication to man at pharmacy | Image Credit: Syda Productions - stock.adobe.com
Medicine tablets on counting tray with counting spatula at pharmacy | Image Credit: sutlafk - stock.adobe.com
Concept of health care, pharmaceutical business, drug prices, pharmacy, medicine and economics | Image Credit: Oleg - stock.adobe.com
Image credit: rawpixel.com | stock.adobe.com
Medical team -- Image credit: Flamingo Images | stock.adobe.com
© 2024 MJH Life Sciences

All rights reserved.