Cystic Fibrosis Drug Misses Goal but Helps Some Adults

December 19, 2013

By Reuters Staff

(Reuters) - Vertex Pharmaceuticals Inc said on Thursday that its drug for cystic fibrosis, ivacaftor, failed to meet its targeted treatment goal in a late-stage study in patients aged 6 and older with the R117H gene mutation.

However, a subset of patients aged 18 and older showed statistically significant improvement in lung function and other secondary measures. The total study included 69 people and 50 of them were 18 or older.

Pre-specified analysis of those patients at least 18 years old showed statistically significant mean absolute treatment difference of 5.0 percentage points (p=0.01); mean relative treatment difference was 9.1% (p=0.008), the company said in a statement.

Vertex plans to meet with the U.S. FDA in early 2014 to discuss these data and the potential submission of a supplemental New Drug Application in people with CF who have the R117H mutation, which can result in varying degrees of cystic fibrosis depending on what other gene mutations are present.

Ivacaftor (Kalydeco) KALYDECO (150mg, q12h) was first approved by the U.S. Food and Drug Administration in January 2012, by the European Medicines Agency in July 2012, by Health Canada in November 2012, by the Therapeutic Goods Administration in Australia in July 2013 and by MedSafe in New Zealand in December 2013 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene

Vertex said it has submitted the drug for approval for use in people with some other mutations and has two other studies underway.

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