Vertex Pharmaceuticals has bagged its first regulatory approval for Orkambi, a combination therapy for the genetic disease cystic fibrosis.
Orkambi combines Vertex’s already approved CF treatment Kalydeco (ivacaftor) with a new drug called lumacaftor that targets a different genetic mutation and – according to analysts – could become a $5bn-a-year product thanks to a list price of $259,000 a year.
Kalydeco is suitable for CF patients with the Gly551Asp mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – representing just 4%-5% of the total patient population – but adding lumacaftor means that it can be prescribed for around 45% of patients.
The second ingredient targets those carrying two copies of the much more common F508del mutation. According to Vertex, there are 8,500 people with CF who are aged 12 and older who have two copies of this mutation in the US and around 12,000 in Europe.
“More than 15 years ago, our scientists set out to discover and develop medicines to treat the underlying cause of CF,” said Vertex’ chief executive Jeffrey Leiden in a statement.
“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,” he added, whilst pledging to bring to market drugs to treat patients with other genetic defects as soon as possible.
Two pivotal trials that form the basis of Vertex’ marketing application for Orkambi in the US showed that the drug was able to improve lung function by 2.6%-4.0% over 23 weeks, and cut the rate of pulmonary infections, hospitalisations and antibiotic use. It would be expected that CF patients would see some lung function decline over that time period.
Given that the average survival age for CF patients is around 37, Orkambi was given breakthrough status by the FDA in 2012. An FDA advisory committee voted 12 to 1 in May that the drug should be approved and had shown safety and efficacy in CF.
Orkambi has also been submitted for approval in Europe, and a decision by the EMA is expected before the end of the year.
Kalydeco has a list price of more than $300,000 a year – although this is generally discounted to insurers – and brought in sales of $464m last year.
Medical charity the CF Foundation (CFF) in the US is jubilant about the approval and applauded the FDA for its swift regulatory review of the drug.
“It is our hope that everyone who is prescribed this drug will have immediate access to it so they can begin to live longer, healthier lives,” commented chief executive Robert Beall.