July 6, 2015
By Mark Terry, BioSpace.com Breaking News Staff
The U.S. Food and Drug Administration (FDA) announced approval of Boston-based Vertex Pharmaceuticals ’s Orkambi (lumacaftor/ivacaftor) for patients with cystic fibrosis (CF) on July 2. It is the first medication approved that treats the underlying cause of CF in patients with two copies of the F508del mutation.
“Today is a remarkable day for science, medicine and the CF community,” said Jeffrey Leiden, chairman, president and chief executive officer of Vertex in a statement. “More than 15 years ago, our scientists set out to discover and develop medicines to treat the underlying cause of cystic fibrosis. Today, the approval of Orkambi represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community.”
Not surprisingly, Vertex stock took a jump on the news. Shares traded for $122.03 on June 29 and rose to $131.26 on July 2. It is currently trading at $131.26. Stock trading was temporarily halted on July 2 ahead of the news.
A filing with the U.S. Securities & Exchange Commission (SEC) indicate that company director Joshua Boger sold 11,700 shares dated Wednesday, July 1 for an average prices of $125.60 for a total of $1,469,520. Goldman, Sachs & Co. analysts reiterated a “buy” rating and set a $142.00 price target in a research note Friday July 3. Zacks analysts downgraded from a “hold” to “sell” and analysts at RBC Capital raised the price target from $130.00 to $156 and listed the company as “outperform” on a June 23 research note.
It is estimated that Orkambi will cost $259,000 for a year’s treatment. Speaking at a July 2 web conference Stuart Arbuckle, Vertex’s chief commercial officer, said, “The price of Orkambi reflects a number of things. Certainly, one of them is the fundamental benefit that we see from treating the underlying cause of this serious disease. There are certainly cost offsets from the benefits Orkambi delivers. That isn’t the only input in to our thinking of the benefit it delivers. We also had to take into account some of the things like the 15-year journey we’ve been on and the time and cost it’s taken us to get to this point. We took into account the eligible patient population, and of course, we want to continue to invest in new medicines for the future.”
The company expects broad access for patients to the drug, based on their talks with payers. Meanwhile, studies continue for younger patients.
“We still have over about a thousand patients in our open label extension,” said Jeff Chodakewitz, Vertex chief medical officer in the web conference. “Some additional patients … were subsequently put on therapy. We’re not concerned about an impact on our 661 studies. Our 661 program is on track. Three of the four studies have been initiated and the fourth study will be initiated shortly. The screening for the 508 homozygous study is complete for the 508 homozygous in the U.S. and randomization is almost done. We’re confident about our ability to continue and maintain the 661 program.”
VX-661 is a Phase II study that looks at CF patients who have two copies of the F508del mutation.
The Orkambi approval was based on two Phase II studies, TRAFFIC and TRANSPORT that looked at more than 1,100 patients with CF ages 12 and older who had two copies of the F508del mutation. The patients treated had statistically significant improvement in lung function.
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