‘Kalydeco’ may sound like a type of Californian interior design style, but it’s actually the name of a new cystic fibrosis drug that Vertex Pharmaceutical announced today it has submitted for market approval in the US.
It’s been a good year for Cambridge, Massachusetts-based Vertex. In May, the US Food and Drug Administration approved the company’s protease inhibitor drug Incivek (telaprevir) — one of the first therapies designed to specifically target proteins of the hepatitis C virus. And now Vertex is hoping to be provide the first treatment that specifically hits the mutated protein responsible for cystic fibrosis, a fatal genetic disorder that afflicts around 80,000 people worldwide.
The small molecule drug formerly known as VX-770 is designed to treat the 4% of people with cystic fibrosis who have a rare mutation called G551D in their CFTR (cystic fibrosis transmembrane conductance regulator) gene. Kalydeco — which, according to Vertex’s press release, is actually pronounced more like the first part of ‘kaleidoscope’ — works by propping open the defective chloride channel protein encoded by CFTR to allow the transport of ions across the cell membrane. As we reported earlier this year, in a study of 161 people with at least one copy of the G551D mutation participants taking the Vertex drug saw their ability to blow air from their lungs improve by 10% and their sweat levels also returned to normal. (See ‘Mutation-specific cystic fibrosis treatments on verge of approval’ for more.)
In a statement, Robert Beall, president and chief executive of the Cystic Fibrosis Foundation, which has invested approximately $75 million in Vertex’s product pipeline, remarked that Kalydeco is “an important potential new medicine for the treatment of some people with CF.” But his words hint at the fact that the vast majority of the people living with the disease will not benefit from Kalydeco alone. Instead of the G551D mutation, 90% of cystic fibrosis sufferers carry an alternate coding error that prevents the CFTR protein from folding properly in addition to disturbing its chloride pumping activity.
In hopes of treating these people, Vertex is combining Kalydeco with another experimental agent called VX-809 in a 160-person clinical trial that is currently enrolling participants. Meanwhile, the company said it plans to turn in its EU application of Kalydeco for people with the G551D mutation by the end of the month.
Last year, the World Health Organization released updated procedures on how best to tackle the global scourge of tuberculosis. The fourth edition of the “Treatment of tuberculosis: Guidelines” recommended, among other changes, increasing the dosage of tuberculosis medication required to treat children. But, in a sense, the new guidance provided a destination without a map: it failed to address the larger problem of how to improve the accuracy of pediatric dosing.
At first sight, it seems like a rather perplexing experiment. In several hospitals across France, some 60 patients are taking part in a clinical trial to test a combination of three drugs. The first striking thing about this trial is that the drugs have already been approved for human use. The second is that each drug is being given at doses between 10 and 100 times weaker than those usually prescribed. Finally, and most surprising of all, none of the patients suffer from the conditions for which these drugs were originally developed. They all have Charcot-Marie-Tooth disease type 1A, a currently incurable neurological condition that causes muscle weakness and loss of touch sensation, and this trial is the result of a radically new approach to understanding and treating disease.
