Novartis drug Jakavi® first medication to receive European Commission approval to treat patients with myelofibrosis

Novartis International AG / Novartis drug Jakavi first medication to receive European Commission approval to treat patients with myelofibrosis . Processed and transmitted by Thomson Reuters ONE. The issuer is solely responsible for the content of this announcement.

* Jakavi() (INC424, ruxolitinib) approval based on results from the most extensive myelofibrosis clinical trial program to date * Myelofibrosis is a life-threatening blood cancer associated with progressive, debilitating symptoms that can severely impact quality of life and shorten survival * In Phase III studies, Jakavi reduced spleen size and debilitating manifestations of myelofibrosis by targeting the underlying mechanism of disease

Basel, August 28, 2012 - Novartis received approval today from the European Commission for Jakavi() (INC424, ruxolitinib), a JAK 1 and JAK 2 inhibitor for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post- polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

The European Commissions decision was based on positive findings from the COMFORT (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Therapy) clinical trial program.

"The approval of Jakavi by the European Commission brings an urgently needed new treatment option with the potential to make a real difference in patients lives, " said Dr. Claire Harrison, MD, Guys and St. Thomas NHS Foundation Trust, Guys Hospital, London. "By targeting the dysregulated JAK pathway, Jakavi delivers a rapid and durable benefit that has the potential to become a new standard of care."

Myelofibrosis is an uncommon, life-threatening blood cancer characterized by bone marrow failure, enlarged spleen (splenomegaly), debilitating symptoms, such as extreme fatigue, night sweats and intractable pruritus (itching), poor quality of life and weight loss, as well as shortened survival[1]. In the EU, the disease affects about 0.75 out of every 100,000 people annually[2], [3]. Myelofibrosis develops when uncontrolled signaling in the JAK pathway - which regulates blood cell production - causes bone marrow scarring and faulty blood cell production, resulting in an enlarged spleen and other severe complications. Jakavi directly targets the underlying mechanism of disease, significantly reducing splenomegaly and improving symptoms regardless of JAK mutational status, disease subtype or any prior treatment, including hydroxyurea[4], [5].

"This approval marks a significant milestone in addressing unmet treatment needs for patients in the European Union, " said Herv Hoppenot, President, Novartis Oncology. "We are committed to the development of innovative treatments for orphan diseases, and are furthering research to assess the potential of targeted Jakavi therapy for other malignancies associated with a dysregulated JAK pathway."

The efficacy and safety of Jakavi in the treatment of patients with myelofibrosis was established in clinical studies, including the two pivotal Phase III trials - COMFORT-I and COMFORT-II. Chronic inflammation through elevated cytokine levels is one of the primary consequences of dysregulated JAK 1 and JAK 2 signaling, and may be a major contributor to morbidity and mortality of patients with myeloproliferative neoplasms such as myelofibrosis[6]. In one pivotal Phase III study, Jakavi was shown to alter the clinical course of myelofibrosis by reversing symptom progression and splenomegaly, thus improving quality of life and potentially impacting overall survival[4], [5].

COMFORT-I demonstrated that 41.9% of Jakavi-treated patients achieved at least a 35% reduction (roughly equivalent to a reduction in palpable spleen size by 50%) in spleen volume at 24 weeks from baseline compared to 0.7% of patients in the placebo group (p<0.001). An early analysis of COMFORT-I data at 51 weeks of treatment showed Jakavi treatment resulted in an overall survival benefit as compared to placebo (hazard ratio=0.50 [95% confidence interval: 0.25, 0.98])[5].

The most frequently reported grade 3 or higher adverse events were hematologic. One patient in each group discontinued treatment for thrombocytopenia or for anemia, respectively. The most common non-hematologic adverse events of any grade reported for patients receiving Jakavi or placebo, respectively, were fatigue (25% vs 34%), diarrhea (23% vs 21%), peripheral edema (19% vs 22%) and ecchymosis (19% vs 9%). One week after discontinuing Jakavi, these patients experienced a return of myelofibrosis symptoms that were present before initiating therapy; however, any symptoms they experienced as a result of treatment discontinuation subsided[5]. COMFORT-I was conducted in the US by Incyte under the worldwide collaboration and license agreement for INC424 (ruxolitinib).

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Novartis drug Jakavi® first medication to receive European Commission approval to treat patients with myelofibrosis