According to Vertex Pharmaceuticals, approximately 300 additional people with CF are now eligible to receive treatment.

Orphan Medicines, led by the gene therapy Zolgensma and the Cystic Fibrosis treatment Trikafta were the most successful launches according to IQVIA list price sales data in Europe in 2020 (figure 2).

Vertex’s significant investment in R&D in 2019 paid off with the earlier-than-expected approval of three-drug cystic fibrosis combination Trikafta (ivacaftor, tezacaftor and elexacaftor ...