Vertex (VRTX) Gets Positive CHMP Opinion for Orkambi in Kids

Vertex Pharmaceuticals Inc. VRTX announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval for expanded use of Orkambi. The opinion is given for Orkambi (lumacaftor/ivacaftor) to treat children with cystic fibrosis (CF) aged two to five years who have two copies of the F508del mutation.

Approximately 1,500 people in Europe in this age group suffer from this most common form of CF disease, thus making Orkambi eligible to treat expanded patient populations.

Orkambi is already approved in the U.S. and Europe for the treatment of CF in patients aged six or older who have two copies of the F508del mutation. It was approved in the United States for the two to five years age group in August.

The CHMP opinion was based on data from a phase III open-label safety study in 60 patients that showed treatment with Orkambi to be generally well tolerated for 24 weeks, with a safety profile similar to that in patients aged 6 years or older.

The stock has gained 10.2% so far this year, against the 18% decline registered by the industry.

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Sales of Orkambi were down 16.1% year over year in the third quarter of 2018 to $282 million due to a switch in patient base to Vertex’s third and newest CF medicine, Symdeko from Orkambi, which targets the same population. Orkambi revenues are expected to be hurt by this switch during the third quarter of 2018.

Other than Orkambi and Symdeko, Vertex also has Kalydeco (ivacaftor) in its CF product portfolio. We remind investors that, Symdeko is a combination of tezacaftor and ivacaftor, which was launched in the United States in February 2018 for treating CF patients. The drug has shown strong uptake so far.

 The company is developing many new combination regimens with CFTR modulators. The CF correctors could bring in multi-billion dollar sales for Vertex.

Vertex is evaluating two next-generation CFTR correctors (VX-659 and VX-445) in phase III studies as part of a triple combination with tezacaftor and ivacaftor. Vertex is evaluating both the combinations in two separate studies for F508del/Min and F508del homozygous patients.

Both AbbVie ABBV and Galapagos NV’s GLPG were jointly developing CF product candidates, which included a triple regimen combination. However, last month, AbbVie announced that it is taking full commercial and development responsibility of all clinical/pre-clinical programs in cystic fibrosis products, which were originally discovered and developed in partnership with Galapagos.

AbbVie will now continue the development of the triple combination therapy for CF. Corbus Pharmaceuticals Holdings, Inc. CRBP is developing a CF candidate, lenabasum, in a mid-stage study.

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