Drug (Brand / Generic)
Tasigna (Nilotinib hydrochloride monohydrate)
Company / Licensee
Tyrosine Kinase inhibitor
Chronic myeloid leukaemia
Approved in US, Switzerland, Japan and the EU
Tasigna (nilotinib) is indicated for treating Philadelphia chromosome positive chronic myeloid leukaemia (Ph+ CML). The drug was discovered by Novarits. The company obtained orphan drug designation for Tasigna from the US FDA on 22 May 2006 and received initial approval in 2007. The final approval was received from the FDA on 17 June 2010.
In August 2010, Novartis received approval for Tasigna from Swissmedic, Switzerland’s health authority. Approvals in the EU and Japan followed in December 2010.
The drug is undergoing first-line indication across the world. Phase III clinical trials on the drug began in 2007 and are expected to be complete by May 2013.
Chronic myeloid leukaemia (CML)
Chronic myeloid leukaemia (CML) is one of the four types of Leukaemia, a type of cancer that is characterised by the uncontrolled growth of leukocytes in the bone marrow and their accumulation in the blood. CML is bone marrow stem cell disorder that occurs due to excessive growth of granulocytes such as eosinophils, neutrophils and basophils. CML is associated with Philadelphia chromosome-positive (Ph+).
CML disease can affect adults and children, but in most cases it is found more in adults. The disease affects men than women. There were estimated to be about 5,050 CML patients diagnosed in the US in 2009 and, according to the National Cancer Institute, there were about 22,475 people surviving with CML disease in the US in 2009.
Almost 85% of the Ph+ CML patients were in the chronic phase of the disease at the time of diagnosis. During the chronic stage, the patients may be asymptomatic or may show mild disease symptoms.
Targeting Ph+ CML
Tasigna 150mg and 200mg capsules are used to treat Ph+ CML in adult patients in the chronic phase. The drug belongs to the antineoplastics group and contains a Tyrosine Kinase inhibitor. The drug assists in stopping the body from producing excessive or abnormal white blood cells and also disturbs the cancerous cells growth.
Tasigna is a selective inhibitor of the Bcr-Abl protein that causes production of cancer cells in a Ph+ CML patient. The drug is a first-line cancer treatment approved for treatment of CML after confirmed blood-related and unconfirmed cell-related response rates.
During Phase I clinical trials on Tasigna, 92% of the CML patients achieved normal white blood cell counts after using the drug for five months.
Novartis began Phase III clinical trials on the drug in 2007, which are scheduled to be complete by May 2013. During Phase III trials, the drug produced a faster and deeper response as a first-line treatment in Ph+ CML patients when compared with Gleevec. The FDA approval was, however, received based on ENESTnd Phase III clinical trial results published in The New England Journal of Medicine.
ENESTnd trials were randomised and open-label tests were conducted on 846 Ph+ CML patients. The trial aimed to compare the safety and efficacy of Tasigna with that of Gleevec, a similar drug discovered by Novartis in 2001 for treating Ph+ CML.
The ENESTnd results indicated that Tasigna showed improved treatment efficacy, and reduced the Ph+ factor and Bcr-Abl protein response much faster than Gleevec. The use of Tasigna resulted in lower rate of progression of cancer by at least 12 months. The safety was also tested as there were no sudden deaths after using Tasigna. During the trial Tasigna 300mg was administered twice a day and Gleevec 400mg was administered only once in a day. Adverse events were reported by some of the Ph+ CML patients using Tasigna.
The approval of Tasigna by US FDA was a major breakthrough in the treatment of the CML disease as well as expanding treatment options for patients in the chronic stage of the disease in the country. The drug has been approved in 80 countries across the world for treating chronic phase of Ph+ CML disease.