October 11, 2016
(Last Updated: October 17, 2016 @ 8:00am PT)
By Alex Keown, BioSpace.com Breaking News Staff
BOSTON – Juliana Keeping, the mother of a small boy with cystic fibrosis is on a quest to get Vertex Pharmaceuticals to reduce the cost of its cystic fibrosis treatment Orkambi. With a price tag of about $259,000 per year, Keeping said the drug is out of reach of many people who do not have adequate resources to pay for the life-saving treatment.
Keeping traveled to Boston to meet with David Altshuler, the chief scientific officer, and Jeffrey Chodakewitz, the chief medical officer of Vertex about the high cost of drugs that were pain in part, through funds raised through charitable efforts. Earlier BioSpace incorrectly reported she met with company CEO Jeffrey Leiden.
She brought her son and a petition with nearly 125,000 signatures to present to Vertex officials. The petition, which was posted at Change.org, points out that Vertex has benefitted from a $75 million infusion of cash from the Cystic Fibrosis Foundation for R&D efforts. On the petition Keeping said if people cannot afford the life-saving treatment, then it may as well not exist. Keeping and her young son, Eli, delivered the petition with the phrase “Stop price gouging our charity-funded drugs” emblazoned across the front, Forbes reported.
“We thought it was a partnership. We thought we were working toward the same goal -- to find a cure for a deadly disease. But I, like others who had been praying for these types of drugs, now feel betrayed,” Keeping wrote on the petition. “Vertex would not have discovered these drugs if not for the help of the CFF and others who cared about finding a cure. But now they have turned around and decided to charge more than the cost of a house for just one year’s prescription of this potentially life-saving medication.”
In a response on BioSpace’s Facebook page, Keeping said the two Vertex executives were friendly and the meeting was pleasant, but, she added they “did not explain the cost of our drugs.”
Eli Hamilton delivered our petition! We got a few shots before agreeing to shut down our cameras and take notes old-school! #fightgreed pic.twitter.com/d1VnFlYv1h— thisiseli (@hithisiseli) October 10, 2016
Orkambi, approved by the U.S. Food and Drug Administration (FDA) in the summer of 2015, is a combination of Vertex’s other CF drug, Kalydeco and lumacaftor. Orkambi will be used to treat patients with the with the F508del mutation of cystic fibrosis, a mutation the lead drug cannot treat on its own. The F508del mutation is the most common genetic mutation in cystic fibrosis. Analysts have predicted the drug will earn $5 billion by 2018. It was recently approved for use in children age six to 11 by the U.S. FDA. Orkambi has the potential to reach about seven times the patients that Kalydeco has. The drug is expected to bring in nearly $1 billion this year, Forbes said.
Cystic fibrosis is a life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. The median predicted age of survival for a person with cystic fibrosis is between 34 and 47 years, but the median age of death remains in the mid-20s. The disease is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. VX-661 is cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
The high price of drugs has been a hot topic lately, particularly with Martin Shkreli, the former CEO of Turing Pharmaceuticals increasing the cost of a 65-year-old toxoplasmosis treatment by 5,000 percent, as well as the high cost of Mylan ’s EpiPen Auto-Injector. Other companies, such as Valeant Pharmaceuticals, has also been under fire for price increases of its drugs. Valeant representative and Shkreli appeared before a Congressional hearing earlier this year to talk about drug prices.
