Novartis drug Jakavi reached its main goal in a late-stage trial for those suffering from a rare blood cancer, placing it in the running to become a potential blockbuster treatment.

Jakavi is already approved to treat a form of blood cancer called myelofibrosis. It was tested in a Phase III trial for patients with the incurable cancer polycythemia vera.

According to a statement released by Novartis Friday, when compared with the best available therapy, the drug was able to maintain red blood cell volume without any need for phlebotemy and lower spleen size in certain patients.

Data from the study will be presented at a forthcoming medical congress and submitted to regulatory authorities this year.

"We are encouraged by these pivotal Phase III trial results, which show the potential of ruxolitinib to help patients with polycythemia vera," said Alessandro Riva, president, Novartis Oncology ad interim and global head, oncology development and medical affairs. "We plan to submit these data to worldwide regulatory agencies this year, as we seek to bring ruxolitinib to patients with polycythemia vera who are no longer responding to or are intolerant of prior therapy."

Polycythemia vera is a chronic, incurable blood cancer linked to an overproduction of blood cells, which leads to a thickening of the blood and increased risk of blood clots. These clots can lead to serious cardiovascular complications including heart attack and stroke, causing increased morbidity and mortality. Those with polycythemia vera often have enlarged spleen and other debilitating symptoms. Many of those treated with commonly available therapies become resistant or intolerant, which is linked to an increased risk of progression.

For the study, researchers randomized 222 patients with polycythemia vera resistant to or intolerant of hydroxyurea. Patients were randomized to receive either ruxolitinib or best available therapy, defined as investigator selected monotherapy or observation only. The dosage was adjusted as needed during the study.

Jakavi drew sales of $163 million last year, an increase from $30 million in 2012. Novartis licensed Jakavi from Incyte Corp and markets the medicine outside of the U.S.

"With the additional indication of polycythemia vera now likely to get approved next year, Jakavi definitely emerges as another new drug with blockbuster potential for Novartis," J. Safra Sarasin analyst David Kaegi said.

The data for Jakavi comes following French competitor Sanofi being forced to stop all clinical trials and cancel plans to seek regulatory approval of fedratinib, a drug for treating myelofibrosis, due to safety concerns.