The CHMP has approved Novartis’ Jakavi (INC424, ruxolitinib) for the rare conditions of adults with myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.
The recommendation was based on findings from the COMFORT clinical trials which demonstrated Jakavi to improve overall survival rates and reduce spleen volume compared to placebo.
Hervé Hoppenot, President, Novartis Oncology, said the oral inhibitor “may address unmet patient needs”.
The CHMP’s opinion has now been passed on to the European Commission (EC) to deliver a final decision. The EC has previously granted Jakavi orphan drug designation for myelofibrosis.
Currently, there are only a handful of treatments available to treat the life-threatening blood cancer disease. Myelofibrosis is characterised by multiple severe complications including bone marrow failure, an enlarged spleen and shortened survival.
Myelofibrosis affects around 0.75 out of 100,000 people each year in Europe. Studies have shown that patients with the disease have a decreased life expectancy with a median survival of 5.7 years.
In the COMFORT-I trial, almost half (41.9%) of patients treated with Jakavi achieved at least a 35% reduction in spleen volume at 24 weeks compared to just 0.7% of patients in the placebo group.
In the COMFORT-II trial, the treatment produced a reduction in spleen volume of greater than 35% in 28.5% of patients compared with 0% of patients in the best available therapy – or not therapy at all – group at 48 weeks.
Results also showed how continuous use of the therapy provided a marked and durable improvement in overall quality of life at 48 weeks.
“The Committee’s positive opinion today validates the strong data in support of Jakavi and the very high unmet need in treating myelofibrosis,” said Hervé Hoppenot.
“The recommendation for European Commission approval of Jakavi also marks a significant step forward in bringing the first and only JAK 1 and JAK 2 inhibitor to patients with very limited options.”
A decision is now expected from the EC within the next three months.