Novartis' Jakavi Safe And Effective In Phase 3B Myelofibrosis Trial

Novartis reported that its blood cancer drug Jakavi (ruxolitinib) was safe and effective in treating patients with myelofibrosis in a Phase 3B trial.

Jakavi is an oral inhibitor of JAK 1 and JAK 2 tyrosine kinases. The drug, licensed from Incyte, has received orphan designations from both the European Commission and the U.S. Food and Drug Administration (FDA) as treatment for myelofibrosis. Jakavi is approved in the EU for the treatment of disease-related splenomegaly in adults.

The expanded-access Phase 3B JUMP trial assessed the safety and tolerability of Jakavi with additional analyses in patient spleen size changes and symptom scores. Results show that the drug’s overall safety and efficacy was consistent with those seen in previous late-stage studies. Patients taking Jakavi also experienced clinically meaningful improvement in symptoms. Significant reduction in spleen size, which was maintained over time, was experienced by 69 percent of patients under treatment with Jakavi.

“Results to date from the JUMP study reinforce the critical role that Jakavi plays in the treatment of myelofibrosis, a life-threatening and debilitating blood cancer with limited treatment options. These data provide insight into the real world experience of more than 1,000 patients living with myelofibrosis and further validate the safety and efficacy of Jakavi as an important treatment for myelofibrosis,” said Haifa Kathrin Al-Ali, University Hospital of Leipzig in Germany.

Myelofibrosis is a rare, deadly type of blood cancer which affects approximately 1 in every 133,000 people. The disease develops when uncontrolled signaling in the JAK pathway drives the body to produce abnormal blood cells. This results in scarring of the bone marrow, an enlarged spleen, and other severe and debilitating symptoms.

Alessandro Riva, Global Head of Novartis Oncology Development and Medical Affairs, said, “Data presented at ASH demonstrate our ongoing commitment to the myelofibrosis community and reinforce the role of Jakavi as the current standard of care for these patients. It's exciting to see the depth of research in a rare blood cancer like myelofibrosis. Results from these studies help us to better understand and address the needs of patients and physicians.”

Novartis presented the results at the 56th Annual Meeting of the American Society of Hematology (ASH) in San Francisco, California.

Earlier this year, the company reported similarly positive results for Jakavi in the Phase 3 RESPONSE trial involving patients with blood cancer polycythemia vera. The drug was able to meet its primary endpoint of maintaining hematocrit control without phlebotomy.