FDA warns on possible cataract risk with Vertex' Kalydeco

The US Food and Drug Administration this week notified health care professionals, parents/caregivers and patients in the cystic fibrosis (CF) community about a potential safety concern for cataract development in children with CF taking US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) Kalydeco (ivacaftor).

The agency approved Kalydeco, the first drug designed to treat the underlying cause of cystic fibrosis, early this year (The Pharma Letter February 7). The drug has also been approved in Europe (TPL July 30)

This potential safety concern is based on recent results from an animal study in juvenile rats that was conducted to support the treatment of young children less than two years of age with ivacaftor, the FDA noted. However, says Brian Orelli writing on The Motlely Fool: “My guess is that this is much ado about nothing, but we won't know for a while. Unfortunately having a safety issue hanging over the biotech could negatively affect the shares until the issue is worked out,” pointing out that juvenile rats are not the main consumers of Kalydeco.