Wider use of Kalydeco recommended in Europe

Vertex has received a positive opinion from Europe's CHMP committee recommending Kalydeco in a broader range of cystic fibrosis patients.

First approved in 2012, Kalydeco (ivacaftor) is the first ever drug for cystic fibrosis (CF) which treats the underlying disease, helping to counteract a faulty gene which means sufferers cannot clear mucus from their lungs.

The drug has been hailed as a breakthrough against the disease, which claims the lives of most sufferers in their 20s. But so far has only be licensed for use in Europe in patients who have at least one copy of the G551D gating gene mutation.

These represent a small proportion of all CF sufferers, and expanding the licence will mean more patients will benefit from the drug.

However the drug's very high cost is already a cause for concern in the US and Europe, and its expanded licence is certain to generate more debate about funding.

In the UK, the drug is officially priced at more than £182,500 a year per patient, although a discount, which remains confidential, has been agreed.

Expanded use

Europe's CHMP committee has now recommended the drug's use in CF patients aged six and older who have one of eight non-G551D gating mutations in the CFTR gene.

The eight additional gating mutations included in the recommendation are: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In Europe, approximately 250 people ages six and older have one of these non-G551D gating mutations.

"People with these additional gating mutations generally have severe cystic fibrosis, similar to patients with the most common gating mutation, G551D. There is an urgent need for new medicines that address the underlying cause of the disease for people with these mutations," said Jeffrey Chodakewitz, M.D., Senior Vice President and chief medical officer at Vertex.

"Today's recommendation for the use of ivacaftor in people with eight additional gating mutations represents an important step toward our goal of helping more people with CF."

The CHMP opinion is based on data from the first part of a Phase 3 study of 39 people with CF ages six and older who have a non-G551D gating mutation. The first part of the study showed statistically significant improvements in lung function (FEV1), sweat chloride, BMI and CFQ-R scores. Data from the second part of the study were presented at the European Cystic Fibrosis Society Conference in June 2014 and showed that these improvements were maintained through 24 weeks of treatment with ivacaftor. The safety profile was similar to prior Phase 3 studies of ivacaftor in people with the G551D mutation.

The CHMP opinion will now be reviewed for final approval by the European Commission. The Commission generally follows the recommendation of the CHMP and typically issues marketing approval within three to four months.

Combination treatment

Vertex has also just released promising phase 3 data for a combination of Kalydeco with another new drug, lumacaftor. This combination is the first regimen designed to treat the underlying cause of CF in people with two copies of the F508del mutation, the most common form of the disease.

This means that the lumacaftor-Kalydeco combination could have an even greater commercial potential than Kalydeco's current licensed use, although analysts doubt it can show the same level of efficacy in the F508del group as in the G551D patients.

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