Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) reported Tuesday morning that two Phase 3 studies of the company’s lumacaftor cystic fibrosis drug in combination with its Kalydeco ivacaftor drug showed statistically significant improvement in lung function in people ages 12 and older with a specific type of cystic fibrosis, a life-threatening genetic lung disease that affects some 75,000 people in North America, Europe and Australia.
Based on the results of the trials, Vertex plans to submit regulatory applications for approval in multiple countries, including the United States and in Europe, in the fourth quarter of this year.
About 4% (around 3,000) of cystic fibrosis sufferers have the specific mutation that is treated by the Kalydeco drug. But the treatment costs $300,000 annually and as many as half of all cystic fibrosis patients carry the specific combination of genetic mutations that may be addressed by the Kalydeco-lumacaftor combination.
An analyst at RBC Capital Markets told The Wall Street Journal that the combination treatment could cut the annual treatment price to a range $150,000 to $175,000. But the number of patients for whom the treatment provides a benefit rises by more than an order of magnitude.
Shares of Vertex traded up about 41% Tuesday to $93.77, after posting a new 52-week high of $98.80 earlier. Over the past 12 months Vertex’s shares have fallen more than 13%, even including Tuesday’s sharp rise. We noted the potential for this price spike two weeks ago.