Novartis presents strong results in rare blood disorder trials

pharmafile | June 10, 2016 | News story | Medical Communications, Research and Development Jakavi, Novartis, Revolade, blood disorder 

Novartis has released data from two late-stage trials for rare blood disorders, which showed that Javi (ruxolitinib) is superior to best available therapy for patients with polycythemia vera and Revolade’s (eltrombopag) long-term safety and efficacy profile in chronic immune thrombocytopenia.

Polycythemia vera is a rare and incurable blood cancer with an overproduction of blood cells that can cause serious cardiovascular complications, such as blood clots, stroke and heart attack. In the RESPONSE-2 Phase III trial, Jakavi achieved superior haematocrit control to best available therapy at 28 weeks in patients with less advanced forms of the disease.

In addition to achieving this primary endpoint of haematocrit control, the trial data shows that nearly five times more patients with polycythemia vera achieved haematologic response with Jakavi compared to best available therapy at 28 weeks.

Alessandro Riva, global head of oncology at Novartis, says: “Given the limited research and treatment options for polycythemia vera, this trial was initiated to gain a better understanding of Jakavi in patients whose disease is not adequately controlled with hydroxyurea. The results demonstrate the potential benefit of Jakavi to help manage the disease in patients who have few other options.”

Additionally, Phase III data from the COMFORT-1 study suggests an overall survival advantage in patients with intermediate-2 or high-risk myelofibrosis randomised to Jakavi compared to patients randomised to placebo. The five-year survival showed a 31% reduced risk of death in the Jakavi arm.

In the case of Revolade, the results of the EXTEND study provided long-term safety data for the drug consistent with the findings from the pivotal Phase III RAISE study. This latest study includes data up to six years in patients with rare blood disorder, immune thrombocytopenia, which is characterised by bruising, bleeding and in some cases, serious haemorrhaging.

The efficacy results of EXTEND showed that median platelet counts were increased and then maintained for more than four years. Bleeding rates declined, also.

Riva adds: “Patients living with chronic diseases will likely remain on therapy for many years, so data about the long-term use of treatments, particularly around safety, are critical. EXTEND is the largest study of its kind and reinforces Revolade as a trusted option that adults with chronic immune thrombocytopenia can use for the long-term.”

Sean Murray

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