Nilotinib(CAS: 641571-10-0)

Nilotinib (AMN107, trade name Tasigna), in the form of the hydrochloride

monohydrate salt, is a small molecule tyrosine kinase inhibitorapproved for

the treatment of imatinib-resistant chronic myelogenous leukemia.

Medical uses

It is FDA- (29 October 2007), EMA- (29 September 2009), MHRA- (19 November

2007) and TGA- (17 January 2008) approved for use as a treatment for Philadelphia

Chromosome (Ph+)-positive Chronic myelogenous leukaemia. In June 2006, a Phase

I clinical trial found nilotinib has a relatively favorable safety profile and shows activity

in cases of CML resistant to treatment with imatinib, another tyrosine kinase inhibitor

currently used as a first-line treatment. In that study 92% of patients (already resistant or

unresponsive to imatinib) achieved a normal white blood cell counts after five months of

treatment. The drug carries a black box warning for possible heart complications. The use

of low doses of nilotinib is being investigated for use for Parkinson’s and Alzheimer’s disease,

as well as for ALS, dementia and Huntington’s disease. Novartis announced on April 11, 2011

that it is discontinuing a Phase III trial of Tasigna® (nilotinib) for investigational use in the

first-line treatment of gastrointestinal stromal tumor (GIST) based on the recommendation of

an independent data monitoring committee. Interim results showed Tasigna is unlikely to

demonstrate superiority compared to Novartis’s Glivec® (imatinib)*, the current standard of care in this setting.