About 6 months after the FDA gave the nod to Vertex Pharmaceuticals' ($VRTX) pricey drug Kalydeco, the European Commission also approved the treatment for patients with cystic fibrosis that have a certain genetic mutation.
The approval helps offset damage caused in May the company was forced to revise the data on its closely watched cystic fibrosis combo therapy of Kalydeco and another treatment after learning that the initial interim data report had been bungled. It has since provided new data that shows the treatment is effective, but not as impressive as the first announcement.
Kalydeco, a "breakthrough" product, is the first to address the cause and not just the symptoms of cystic fibrosis. In the FDA's case, it was approved a couple of months ahead of schedule. The treatment, however, is very expensive at $294,000 a year. About 60% of the eligible patients have commercial insurance, while government programs will cover the remainder. Vertex's co-pay assistance program will cover up to $88,000 of patients' co-insurance or co-payments.
Even with a small pool of potential patients in the U.S., analysts forecast Kalydeco will deliver more than $500 million in sales, and now even more in Europe. Vertex is actually testing two different combinations, each pairing Kalydeco with a different experimental drug. If those combos prove successful, then use could expand to up to 90% of CF patients. And that would turn Kalydeco into a blockbuster with peak sales of up to $4 billion.
The company is still dealing with the fallout from the messed up clinical data. Less than two weeks after it corrected the interim results, Sen. Charles Grassley sent a letter to the SEC asking to investigate whether company officials' stock transactions that took place before the true data were released.
- here's the release