Vertex’s Kaftrio Plus Kalydeco Offers Considerable Added Benefit, Finds G-BA

BERLIN, Germany – Germany’s Federal Joint Committee (G-BA) has concluded that Vertex’s triple-combination therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor) plus Kalydeco (ivacaftor) offers significant benefit versus other therapeutic options for patients with cystic fibrosis (CF).

Kaftrio in combination with Kalydeco was found to offer considerable added benefit in CF patients aged 12 years and up who are homozygous for the F508del mutation in the CFTR gene compared to Symkevi (tezacaftor/ivacaftor) plus Kalydeco, as well as in patients who are heterozygous for the F508del mutation in the CFTR gene and have a minimal function (MF) mutation compared to best supportive care (BSC).

Professor Josef Hecken, impartial chairperson of G-BA, commented, “The combination therapy with ivacaftor that we have tested can achieve very good results with the patients treated: In the studies, pulmonary exacerbations, i.e. acute worsening of lung symptoms, which often lead to hospital admissions and have an unfavorable effect on the course of the disease, occur significantly less often. It is also highly relevant for the patients that we saw advantages in many other patient-reported endpoints and in all measurable aspects of quality of life. For example, in terms of physical well-being, vitality and emotional state.”

The rating for Kaftrio with Kalydeco is Germany’s highest, the agency noted. Hecken also added, “It is not often that we can certify a significant additional benefit to a drug: This has only been the case three times so far. The special feature of the assessed combination therapy for cystic fibrosis – an orphan drug – is that the result is already based on detailed comparative study data and we have carried out a regular benefit assessment compared to the appropriate comparator therapy.”

Vertex submitted a complete dossier to G-BA, despite Kaftrio plus Kalydeco being considered an orphan drug. Within four months of being on the market, Kaftrio exceed the EUR 50 million threshold for sales, meaning its status as an orphan drug was repealed and the drug was subject to standard assessment. Since a full dossier was submitted, G-BA could get right to work.

G-BA’s rating will serve as the basis of upcoming pricing negotiations between Germany’s umbrella payer GKV-Spitzenverband (GKV-SV) and Vertex, with pricing set to apply starting from September 1, 2021. Of the 8,000 patients with CF in Germany, 2,400 are older than 12 and are expected to benefit from today’s decision.