Tag: kalydeco

FDA approves Orkambi

Orkambi, a New Cystic Fibrosis Drug, Wins F.D.A. Approval

By ANDREW POLLACK
JULY 2, 2015

nytimes.com
A new drug for people with the most common genetic type of cystic fibrosis won approval from the Food and Drug Administration on Thursday.

The drug, Orkambi, is the second cystic fibrosis treatment brought to market by Vertex Pharmaceuticals, a biotechnology company based in Boston. It is expected to make the company consistently profitable; it has lost several billion dollars since it was founded 26 years ago.

Both Vertex drugs are the first to try to counteract the underlying genetic defect that causes the disease, as opposed to treating symptoms.

Vertex’s first cystic fibrosis drug, Kalydeco, which was approved in 2012, is applicable to a small percentage of those with cystic fibrosis, those with particular genetic muations. Sales of Kalydeco reacched $464 million in 2014, accounting for the bulk of Vertex’s revenue.

Orkambi, by contrast, could be useful for nearly half of cystic fibrosis patients, those who have two copies of the most common mutation, known as F508del. The initial approval, however, is only for patients 12 and older.

Analysts expect annual sales of Orkambi to eventually exceed $3 billion. Orkambi is a combination of ivacaftor, the active ingredient in Kalydeco, and a second drug called lumacaftor.

Approval was expected because a committee of outside advisers to the F.D.A. endorsed the drug in May by a 12-1 vote. The agency’s own staff, however, had given the product a lukewarm review, saying its effectiveness in improving lung function was small.

Vertex did not immediately announce the price of Orkambi. Kalydeco costs more than $300,000 a year for each patient, a price that has drawn criticism from some doctors who treat cystic fibrosis.

Wall Street analysts have been expecting Vertex to charge less for Orkambi, perhaps $200,000 to $250,000 a year.

That is because Orkambi would be used by more patients, meaning Vertex would not have to charage as much per patient to recoup its investment. Also, Orkambi is less effective than Kalydeco in improving lung function, meaning it will have less value in the eyes of insurance companies and government agencies that pay for these drugs.

An update on Kalydeco

Vertex pharmaceuticals, the company who pioneered the revolutionary Kalydeco drug treatment currently available for people with the GFF1D mutation – around 4% of the population, will be presenting further updates on how the drug works for people who are homozygous for the F508del mutation (the more standard mutation) at this year’s North American Cystic Fibrosis conference currently being held in Atlanta.

Vertex Pharmaceuticals will be presenting 15 abstracts at the 28th Annual North American Cystic Fibrosis Conference (NACFC) held in Atlanta from October 9-11. Each abstract is from Vertex’s cystic fibrosis research and development program. Data will primarily be related to TRAFFIC and TRANSPORT clinical trials, which were phase 3 rollover studies evaluating lumacaftor and ivacaftor combination therapy.

According to a news release from Vertex, presented data will include the first interim data from patients who completed TRAFFIC and TRANSPORT, which were highlighted previously on Cystic Fibrosis News Today. These studies were for individuals homozygous for the F508del mutation. Data from this subtype of cystic fibrosis patients, who were on combination lumacaftor and ivacaftor therapy for 24 weeks, were previously presented and will be revisited at NACFC. Data concerning combination therapy will be related to drug distribution in the body, safety in children on the treatment, and the effects of using bronchodilators while on concomitant combination therapy.

Presented studies will also focus on children receiving ivacaftor treatment. These studies evaluated individuals with R117H residual functional mutations who benefit from the potenitation mechanism of ivacaftor. Topics include the effects of ivacaftor on forced expiratory volume in one second (FEV1), the distribution of ivacaftor throughout the body following administration, and the effects of ivacaftor on patient weight.