Dive Brief:
- The Food and Drug Administration has approved Vertex Pharmaceuticals' Orkambi as the first drug to treat the cause of cystic fibrosis in children aged two to five years who have two copies of the F508del mutation, the company announced Tuesday. Orkambi is already approved in older children and adults.
- The approval is based on a Phase 3 study that confirmed equivalent safety and tolerability to children aged six years or more over 24 weeks, along with improvements in sweat chloride and in key growth parameters. Formulated as oral granules, Orkambi should be available within two to four weeks.
- Vertex has submitted a marketing authorization application in Europe, and expects a decision in the first half of 2019.
Dive Insight:
At one time, children with CF were barely expected to live beyond school age, whereas babies born with the disease between 2012 and 2016 have a life expectancy of 43 years, with over half living to 47 or older, according to the Cystic Fibrosis Foundation. As with many chronic disorders, early diagnosis and early treatment in CF may have an impact in reducing or even preventing lung damage.
The approval of Orkambi (ivacaftor/lumacaftor) in children aged two to five years who have two copies of the F508del-CFTR mutation has brought the age of treatment down; the previous approval was for ages six and over.
"This approval is a significant development that enables physicians to begin treating the underlying cause of the disease in this population earlier than ever before," said John McNamara, medical director of the cystic fibrosis program at Children’s Minnesota hospital and lead study researcher, in a statement.
Vertex's other CF drugs also have potential in younger populations. Kalydeco (ivacaftor) is pending approval for use in children as young as one year of age, and Symdeko (tezacaftor/ivacaftor and ivacaftor) is being assessed in children aged six to 11 years.
The U.S. launch of Symdeko helped drive growth in Vertex's total CF product revenues up to $750 million last quarter, a 46% increase from the second quarter of 2017. Symdeko itself had $186 million in sales, after being approved in the U.S. in February.
Symdeko is Vertex's third marketed CF drug and is treating both new patients and patients switching from Orkambi, either directly or after a break. Notably, Orkambi's U.S. revenues have fallen from $282 million in the first quarter to $236 million in the second quarter.
"We've actually seen strong uptake [for Symdeko] across the three different patient populations that we were anticipating that we'd see demand in. That's those who have tried Orkambi previously and discontinued, those who have never been exposed to a CFTR modulator, either Orkambi or Kalydeco, and we saw a fair amount of transitions from Orkambi to Symdeko," said Stuart Arbuckle, Vertex's chief commercial officer.
The CF franchise grew 33% in the first quarter of 2018, and this looks set to continue.
Vertex recently revised its annual guidance for CF product revenues to $2.9 billion to $3 billion from a prior range of $2.65 billion to $2.8 billion.