Vertex Pharmaceuticals’ (NASDAQ:VRTX) Orkambi (lumacaftor/ivacaftor) for cystic fibrosis (CF) has generated lacklustre physician reviews since its July 2015 US launch. They noted lung function (FEV1) improvements were mediocre and inconsistent in patients, and tolerability issues could spur more discontinuations than expected.
Access has been surprisingly smooth considering the hefty annual price of USD 259k per patient, physicians noted, but the situation is ripe for subsequent payer pushback if patients don’t show meaningful FEV1 improvements. However, the drug’s potential to reduce exacerbations and slow down disease progression could save its payer argument with confirmatory long-term data, they noted.
CF is a genetic condition where the lungs and digestive system become clogged with thick and sticky mucus, causing persistent cough, lung infections and poor weight gain. Orkambi – designed to correct the homozygous F508del CFTR mutation – was FDA approved in July 2015 for patients 12 years and older. By March 2016, around 65% or 5,500 of 8,500 eligible US patients had initiated Orkambi treatment, according to Vertex's 27 April earnings call.