Vertex Pharmaceuticals ($VRTX) has hit an unexpected snag with its cystic fibrosis pill Kalydeco. The FDA is raising questions about a potential risk of cataracts in children using the drug, and asked the drugmaker to conduct a two-year study to assess that risk.
The FDA's worries arose after a study in juvenile rats. After receiving a dose of Kalydeco about one-tenth the maximum recommended for humans, the young rodents developed cataracts. Whether the rat results mean similar risks apply to humans is unclear, the agency said.
And that's why FDA asked Vertex to follow kids who are already taking Kalydeco, which targets cystic fibrosis in patients with a particular genetic mutation. The study would involve kids up to 11 years old, and would monitor participants for at least two years by providing eye exams every 6 months. In the meantime, the agency is adding information about the rat study to Kalydeco's label.
Vertex said that it hasn't received complaints about cataracts from any patients who used the drug during clinical trials or since its launch in January. The drug is approved for use in kids 6 years and older, but Vertex plans to expand that range to patients as young as 2 years.
- read the Reuters news
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