January 28, 2016
By Alex Keown, BioSpace.com Breaking News Staff
BOSTON – An issue of patient compliance in taking Vertex ’s new blockbuster cystic fibrosis drug Orkambi is at the root of why the company has not issued sales guidance yet, the Boston Business Journal reported this morning.
During the J.P. Morgan Healthcare Conference earlier this month, Vertex did not issue sales guidance reports, which caused company stock to tumble, a decline from which it has yet to turn around. Vertex stock is down almost 9 percent this morning following the release of its final quarterly report of 2015. In a conference call with reporters and investors, Ian Smith, chief financial officer of Vertex, said Orkambi has been prescribed to about 55 percent of the 8,500 people with conditions for which it has been approved, the Journal said. Smith added he anticipated the “vast majority” of the 8,500 patients would be taking Orkambi by the end of 2016. However, he noted the company could not provide sales guidance until it determines patient compliance with the drug, meaning are they taking the prescribed doses correctly, and persistence, meaning are patients staying with the treatment. Company officials are expecting a high rate for both compliance and persistence and noted there is an 85 percent compliance rate with its other cystic fibrosis drug, Kalydeco.
Orkambi, approved by the U.S. Food and Drug Administration in the summer of 2015, is a combination of Kalydeco and lumacaftor. Orkambi will be used to treat patients 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 and finally put Vertex in the black. One reason analysts are so high on the profitability of the drug is its potential to treat more than 20,500 patients in the United States and the European Union, about seven times the reach of Kalydeco, which the company predicts will generate net revenue of $670 to $690 billion in revenue in 2016. In its first sixth months since approval in the U.S., Orkambi has generated net revenue of $350.7 million, Vertex said. The Canadian government approved the use of Orkambi in people ages 12 and older with two copies of the F508del mutation, approximately 1,500 people, on Jan. 26.
Vertex recently completed a Phase III trial of Orkambi for children ages 6 to 11 with two copies of the F508del mutation. During its quarterly conference call on Wednesday, the company announced the study met its primary safety endpoint and data shows the drug was well tolerated in the 58 children enrolled. Two patients discontinued the treatment due to adverse drug effects, which included cough, headache, infective pulmonary exacerbation, nasal congestion, abdominal pain, increased sputum and elevated liver enzymes, Vertex said. Vertex plans to submit a supplemental New Drug Application (sNDA) to the FDA in the second quarter of 2016. There are approximately 2,400 children ages 6-11 who have two copies of the F508del mutation in the U.S., Vertex said.
In addition to Kalydeco and Orkambi, Vertex is continuing to develop treatment for cystic fibrosis, including VX-661.
