Novartis AG drug Jakavi gets tagged as a ‘potential blockbuster’ after positive late-stage study

Positive Phase 3 study points to significant clinical benefit for patients with debilitating and life-threatening blood-cancer myelofibrosis

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DORVAL, Quebec—Study results presented at the 55th American Society of Hematology (ASH) annual meeting showed that patients with myelofibrosis who received treatment with Novartis Pharmaceutical Canada’s Jakavi® (ruxolitinib) sustained reductions in spleen volume and sustained improvements in quality of life after three years of treatment. These results were demonstrated in analyses from the Phase 3 COMFORT-I (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Therapy) study. These long-term follow-up results build on previously established data and reinforce the sustained efficacy and long-term safety profile of Jakavi® in patients with myelofibrosis.
Of the patients treated with Jakavi, 59 percent experienced at least a 35 percent reduction in spleen size at any time during the three year study. In an exploratory analysis, patients who continued on therapy experienced sustained improvements in the Global Health Status/Quality of Life and the functional domains of the EORTC Core Quality of Life questionnaire (EORTC QLQ-C30). Median follow-up was 149 weeks at the time of the updated analysis.
This scientific data was presented at ASH as new clinical information regarding Jakavi and is not contained in the current Canadian approved product monograph. Jakavi is indicated for the treatment of splenomegaly and/or its associated symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), postpolycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.
"The long-term results of the COMFORT-I study provided data supporting the safety and efficacy of ruxolitinib in myelofibrosis, and the benefit on quality of life is significant," said Dr. Vikas Gupta a myeloproliferative neoplasms specialist at the Princess Margaret Cancer Centre in Toronto. "Several novel agents are on the horizon in the treatment of myelofibrosis, and we are entering an era of renewed hope for myelofibrosis patients."
The COMFORT-I study is part of the largest clinical trial program in myelofibrosis to date. Further analyses of Phase 3 data were presented at the 2013 ASH annual meeting early in December. Jakavi continues to reduce risk of deaths for patients with myelofibrosis as reported in the following abstracts, the company reported in a press release.
The COMFORT (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Therapy) studies are randomized, Phase 3 studies comparing the safety and efficacy of Jakavi with placebo or best available treatment (BAT) in patients with intermediate-2- or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis. COMFORT-I is a double-blind study whereas COMFORT-II is open label. Patients initially received Jakavi 15 or 20 mg twice daily based on their platelet counts at baseline (100-200 and > 200 x 109/L, respectively) and were individually titrated to maximize safety and efficacy. Patients were allowed to cross-over from the control arms of each study upon protocol-defined progression events (primarily progressive splenomegaly, defined as a = 25 percent increase in spleen volume from baseline or on-study nadir in COMFORT-I and -II, respectively). At the time of the analysis reported at the 2013 American Society of Hematology annual meeting, all ongoing control patients had crossed over to Jakavi. Overall survival was a secondary endpoint in both studies.
Myelofibrosis is a life-threatening blood cancer with a poor prognosis and limited treatment options. It develops when uncontrolled signaling in the JAK pathway—which regulates blood cell production—causes the body to make blood cells that do not work properly, which scars the bone marrow and results in an enlarged spleen and other severe complications. Although the exact prevalence is uncertain, a recent analysis done in the United States estimates myelofibrosis prevalence ranged from 3.6-5.7 per 100,000 patients. Myelofibrosis is typically diagnosed in people between 50 and 80 years old, although the disease can arise at any age. Both men and women are affected.
Studies showed that patients with myelofibrosis have a decreased life expectancy, with a median overall survival of 5.7 years. Although allogeneic stem cell transplantation may cure myelofibrosis, the procedure is associated with significant morbidity and transplant-related mortality, and is available to fewer than 5 percent of patients who are young and fit enough to undergo the procedure.
Jakavi (ruxolitinib) is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases approved by Health Canada in July 2012 for the treatment of splenomegaly (enlarged spleen) and/or its associated symptoms in adult patients with myelofibrosis.
In 2012, Novartis Pharmaceuticals Canada Inc. invested close to $100 million in research and development and employs more than 600 people in Canada. The company is a subsidiary of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis Group of Companies offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas, the company claims. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 136,000 full-time-equivalent associates and operate in more than 140 countries around the world.
SOURCE Novartis Pharmaceuticals Canada Inc.

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