One of the (many) things that makes cystic fibrosis so difficult to treat is that there are many mutations of the disease, some affecting very, very tiny subsets of the CF population.
That means not a lot of research dollars are being spent for these subsets. So anytime there’s an announcement for a new drug approval—or an expanded approval of an existing drug—it’s a pretty big deal. And it’s an even bigger deal when the approval covers very young children, who are too often left behind in clinical trials.
So step forward and take a bow, Vertex Pharmaceuticals. The European Commission—whose responsibilities include drug approval—just delivered some early Christmas presents for people living with CF, or have children with CF.
In November, the Commission approved expanded indications for Vertex’s CF drug KALYDECO® (ivacaftor). These expanded indications include children with CF between two and five years old who have one of nine different mutations, including G551D. The expansion also covers people with CF over age 18, with an R117H mutation—this is the first time a medication has been made available to treat the underlying cause of this mutation.
The approvals impact about 125 children and 350 adults across Europe…which admittedly, doesn’t sound like a lot. So why does this matter? Precisely because the numbers are smaller and because many of the patients are so young.
As one of the lead Principal Investigators on the study pointed out, damage caused by CF can begin at birth. This approval means doctors can begin treating the underlying cause of the disease much sooner. That gives these children a much better shot at a good long-term prognosis.
The Commission also approved a new drug, ORKAMBI® (lumacaftor/ivacaftor). ORKAMBI is the very first medicine for people over 12 years of age with two copies of the F508del mutation—that’s about 12,000 people in Europe. In a clinical trial, the drug (which is actually a combination of two drugs) showed “significant improvements in lung function,” along with other benefits.
These approvals represent another triumph in the long-standing partnership between Vertex and the Cystic Fibrosis Foundation Therapeutics, Inc., a nonprofit affiliation of the CFF focused on discovering and developing new drugs.
Vertex and the CFFT have been working together since 1998, and both KALYDECO and ORKAMBI were developed as part of the collaboration.
While the studies used to obtain these approvals was conducted in Europe, if the results hold up in the long haul it could provide good ammunition for Vertex to push for a similar expansion in America.
Fingers crossed, and we’ll keep you posted.
Want to learn more? Check out the full list of mutations covered by the expansion here and read all about the ORKAMBI approval here. To learn more about Vertex and KALYDECO go here. Don’t forget to check out this latest research initiative in conjunction with CysticLife. And share this post!