Vertex Continues to Pursue Broad Range of Therapies for Cystic Fibrosis Patients

Vertex Pharmaceuticals is continuing to develop treatments for all people with cystic fibrosis (CF), with clinical trials exploring combinations of its potentiator and corrector therapies. At the same time, efforts to expand the use of already-approved treatments are ongoing.

In addition, the company recently completed the purchase of Concert’s CTP-656, yet another CFTR potentiator in development for use as part of future combination regimens.

“During the first half of 2017, Vertex has made significant progress throughout the business and in particular, across our CF development programs,” Jeffrey Leiden, MD, PhD, chairman, president and CEO of Vertex, said in a press release.

Among the updates the company shared in its financial report for the second quarter of 2017, were efforts to get Orkambi (lumacaftor/ivacaftor) reimbursed in several countries in the EU. Orkambi is approved for patients aged 12 or older with two copies of the F508del mutation in the CFTR gene (the defective gene that causes CF).

Vertex has secured access to Orkambi for patients in Austria, Denmark, Germany, Ireland, Italy and Luxembourg. Meanwhile, they continue to negotiate in a row of other countries, including France, the Netherlands and the U.K., among others.

As reported earlier, the company also has worked with the FDA to get Kalydeco approved for people with more rare CFTR mutations. The number of mutations approved for treatment with Kalydeco is now 33, and Vertex continues working with the FDA to include additional rare mutations in the drug’s prescription label.

Meanwhile, Vertex has submitted a new drug application with the FDA for the combination treatment tezacaftor/ivacaftor. The treatment had been granted orphan drug designation, and is intended for patients 12 years old or older.

Vertex also is exploring the tezacaftor/ivacaftor combination in a Phase 3 trial in patients with one F508del mutation and a second mutation that causes a gating deficit in the CFTR protein. In the financial report, Vertex underscored that enrollment in the trial is complete.

Finally, the company is running trials exploring three triple combinations — tezacaftor/ivacaftor together with either VX-440, VX-152, or VX-659. They recently provided an update on the trials, demonstrating that all three were effective in patients with difficult-to-treat minimal function mutations.

“Our progress has been marked by the progression of multiple combination regimens that allow us to treat more people with CF today and to potentially treat up to 90% of patients with this disease in the future,” concluded Leiden.