Novartis snags new indication for Jakavi in Europe

Novartis' blood cancer drug Jakavi has been cleared in the EU to treat polycythaemia vera (PV), a new indication that could catapult the drug to blockbuster status.

Jakavi is already approved in Europe to treat myelofibrosis and adding PV to its label expands the number of patients eligible for treatment with the drug. PV is a rare and incurable blood cancer associated with an overproduction of blood cells in the bone marrow that affects roughly 100,000 people in Europe.

The drug has been approved by the European Medicines Agency (EMA) to treat adults with PV who are resistant to, or intolerant of, therapy with hydroxyurea, the most commonly-used drug for the cancer. Around 25 per cent of PV cannot be treated effectively with hydroxyurea, according to Novartis.

Jakavi's approval is based on data from the phase III RESPONSE trial, which showed that a significantly greater proportion of patients on the drug achieved a blood response and a reduction in spleen size compared to best available therapy. PV patients show elevations in haematocrit, the volume percentage of red blood cells in whole blood, which can lead to a thickening of the blood and an increased risk of blood clots.

Overall, 21 per cent of patients treated with Jakavi achieved the efficacy objectives in the trial, compared to 1 per cent of the control group. Moreover, a 50 per cent or greater improvement in PV-related symptoms was seen in 49 per cent of Jakavi-treated patients and just 5 per cent of those on best available therapy.

Novartis reported sales of Jakavi reached $279 million last year, while development partner Incyte – which sells the drug in the US as Jakafi – booked $358 million from the drug. Analysts have suggested that adding PV to its label claims could drive sales above the $1 billion threshold, and even higher if it secures approval in additional indications such as pancreatic cancer. Incyte secured US FDA approval for Jakafi in PV last December.

"The European Commission's approval of Jakavi is encouraging news for patients," said Dr Claire Harrison, study investigator and consultant haematologist at Guy's and St Thomas' NHS Foundation Trust in London, UK.

"Jakavi will fill an unmet need as the first treatment shown to significantly improve haematocrit, as well as symptom control and reduce spleen size in patients with PV resistant to, or intolerant of, hydroxyurea."