G-BA issues final Kalydeco assessment

Germany's Federal Joint Committee (G-BA) said in a final assessment that cystic fibrosis drug Kalydeco Ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) provides a "significant" degree of additional benefit for patients older than 12 and a "marginal" degree of benefit for patients ages 6-11 years. Kalydeco is approved to treat CF in patients six years or older who have at least one copy of the G551D mutation in the CF conductance regulatory ( CFTR) gene. Vertex will now negotiate a price for the Orphan drug, a small molecule potentiator of the CFTR gene, with Germany's Statutory Health Insurance Funds Association (GKV-Spitzenverband). Vertex was off $0.21 to $45.65 on Thursday.

Under drug pricing law AMNOG, the additional benefit of Orphan products is regarded as having been demonstrated by the marketing authorization. Orphan products with an annual cost of less than EUR 50 million to the German statutory health insurance funds (GKV) do not undergo a formal benefit assessment through the Institute for Quality and Efficiency in Health Care (IQWiG), though G-BA still determines the extent of the additional benefit based on a product's marketing authorization studies. ...