Novartis’ myelofibrosis treatment approved in Europe

pharmafile | August 28, 2012 | News story | Sales and Marketing Incyte, Jakavi, Novartis, Orphan Drugs, myelofibrosis 

Novartis’ Jakavi, a new treatment for patients with myelofibrosis has been approved in Europe.

The drug was co-developed with Incyte, and the US company gained FDA approval to market the drug there in November last year under the name Jakafi.

Analysts expect peak sales in the US to reach $600 million and around $350 million in the rest of the world, which means the product is unlikely to join the ranks of orphan drugs which achieve blockbuster status with sales in excess of $1billion – sometimes called ‘nichebusters’.

The drug’s approval is nevertheless a big step forward in treating the very serious haematological cancer, which on average kills the patient within six years.

Jakavi is a JAK 1 and JAK 2 inhibitor for the treatment of disease-related splenomegaly or symptoms in adult patients with one of several different types of the rare disease myelofibrosis.

Myelofibrosis develops when the production of blood cells goes haywire, causing bone marrow scarring and faulty blood cell production, and resulting in an enlarged spleen and other severe complications.

Jakavi works by targeting the underlying mechanism of disease, the signaling in the JAK pathway which regulates blood cell production. The drug significantly reduces splenomegaly and improves symptoms regardless of JAK mutational status, disease subtype or any prior treatment, including hydroxyurea.

“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, Guy’s and St. Thomas’ NHS Foundation Trust, Guy’s 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.”

Jakavi has been approved for the condition despite its potential to cause can cause serious side effects, including a decrease in blood cell count and infections. The licence recommends that complete blood count monitoring is maintained for patients, and says dose reduction or interruption may be required in patients with severe hepatic or renal impairment or in patients developing haematologic adverse reactions such as thrombocytopenia, anemia and neutropenia.

Dose reductions are also recommended when Jakavi is co-administered with strong CYP3A4 inhibitors or fluconazole.

Myelofibrosis is an uncommon, life-threatening blood cancer characterised 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. In the EU, the disease affects about 0.75 out of every 100,000 people annually.

“This approval marks a significant milestone in addressing unmet treatment needs for patients in the European Union,” said Hervé 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.”

Two pivotal Phase III trials – COMFORT-I and COMFORT-II showed Jakavi altered the clinical course of myelofibrosis by reversing symptom progression and splenomegaly, and reducing the size of the spleen, thus improving quality of life and potentially impacting overall survival.

Patients with myelofibrosis have a decreased life expectancy, with a median 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 less than 5% of patients who are young and fit enough to undergo the procedure.

Jakavi is also being investigated in clinical trials for the treatment of polycythemia vera. 


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