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Novartis takes step towards ruxolitinib approval outside US

JAK inhibitor shows benefits in rare bone marrow disorder myelofibrosis

Novartis has published data for its investigational compound ruxolitinib that it says provides the basis for worldwide regulatory filings in a rare bone marrow disorder.

Two phase III studies published in the New England Journal of Medicine (NEJM) have demonstrated that treatment with the JAK inhibitor significantly reduced disease burden in patients with myelofibrosis, a condition that can lead to enlarged spleen and several other negative effects.

The COMFORT-II trial, conducted by Novartis, achieved its primary endpoint by demonstrating a spleen size reduction of 35 per cent or more in just over a quarter of patients treated with ruxolitinib after 48 weeks. This compared to 0 per cent of patients on alternative best available therapy.

Continuous treatment with the drug also showed improvement in other myelofibrosis symptoms, such as fatigue, pain and appetite loss.

Data from the COMFORT-I trial was also published in the NEJM, with 41.9 per cent of patients treated with ruxolitinib achieving at least a 35 per cent reduction in spleen volume at 24 weeks compared to 0.7 per cent of patients in a placebo group.

This study had previously helped support ruxolitinib's approval in the US, where the drug is marketed as Jakafi by Incyte Corp.

Novartis will now use the data to support its own regulatory filings, holding rights to the drug outside the US.

Ruxolitinib is the first JAK inhibitor to receive approval. The drugs work by blocking the enzymes JAK1 and JAK2, the functioning of which is disrupted in myelofibrosis and other autoimmune disorders.

Novartis is also investigating the drug's use in the treatment of polycythemia, and has expressed its optimism that the drug has potential for approval in a number of indications.

Other companies pinning their hopes on JAK inhibitors include Pfizer, with tofacitinib currently under review by the European Medicines Agency (EMA) for treatment in rheumatoid arthritis (RA).

Just yesterday, Abbott signed a $1.35bn deal with Belgium-based Galapagos to develop and commercialise its JAK inhibitor GLPG0634, which is currently in phase II development for RA.

1st March 2012

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