Novartis announced that the European Commission (EC) approved Tasigna® (nilotinib) for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP) and pediatric patients with Ph+ CML-CP with resistance or intolerance to prior therapy including imatinib. Tasigna is the only second-generation tyrosine kinase inhibitor (TKI) currently approved in the European Union (EU) for the treatment of Ph+ CML-CP in children. The approval follows a positive opinion issued by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) on September 14, 2017 and applies to all EU member states.
"Treatment options for children with CML have historically been limited, and with this new indication an unmet need has been addressed," said Bruno Strigini, CEO, Novartis Oncology. "Data from two prospective studies demonstrated Tasigna is safe and effective in patients as young as two years old, which is consistent with the established safety profile of Tasigna in adults."
This expanded indication is based on two prospective studies of nilotinib in children with Ph+ CML-CP, which were part of a formal "pediatric investigation plan" agreed upon with the EMA. A total of 69 pediatric patients received nilotinib. These pediatric patients were aged 2 to 18 years and diagnosed with either newly diagnosed Ph+ CML-CP or Ph+ CML-CP with resistance or intolerance to prior therapy including imatinib. In the newly diagnosed CML patients, the major molecular response (MMR; BCR ABL/ABL <=0.1% International Scale [IS]) rate was 60.0% (95% confidence interval [CI]: 38.7, 78.9) at 12 cycles, with 15 patients achieving MMR. In patients with resistance or intolerance to prior therapy including imatinib, the MMR rate was 40.9% (95% CI: 26.3, 56.8) at 12 cycles, with 18 patients being in MMR. In newly diagnosed CML patients, the cumulative MMR rate was 64.0% by cycle 12. In patients with resistance or intolerance to prior therapy including imatinib, the cumulative MMR rate was 47.7% by cycle 12[1],[2].
In pediatric patients, adverse reactions observed were generally consistent with those observed in adults, with the exception of hyperbilirubinemia (Grade 3/4: 13.0%), a condition where there is too much bilirubin in the blood, and transaminase elevation (AST Grade 3/4: 1.4%, ALT Grade 3/4: 8.7%) which were reported at a higher frequency than in adult patients. No deaths were reported on treatment or after treatment discontinuation in both studies[1],[2].
This is the latest in a series of regulatory milestones for Tasigna. In June, Novartis achieved an important milestone for the Ph+ CML community when Tasigna became the first and only TKI to include information on stopping therapy in adult patients with Ph+ CML-CP in both the first-line setting and after switching from imatinib in the EU label and several other labels throughout the world. Novartis continues to follow the science to advance and reimagine the future of CML.
About Ph+ CML
CML is a type of cancer in which the body produces cancerous white blood cells. Almost all patients with CML have an abnormality known as the Philadelphia chromosome, which produces a protein called BCR-ABL. BCR-ABL causes malignant white blood cells to proliferate. Worldwide, CML is responsible for approximately 10% to 15% of all adult cases of leukemia, with an incidence of one to two cases per 100,000 people per year.
Novartis Commitment to CML
Novartis ongoing research in Ph+ CML has helped transform the disease from a fatal leukemia to a chronic condition in most patients. The company maintains an unwavering commitment to scientific innovation and access to care for patients worldwide. As an organization committed to patients, Novartis continues to reimagine CML by pursuing ambitious goals with courage, passion and commitment to the global CML community.
About Tasigna (nilotinib)
Tasigna (nilotinib) is approved in more than 122 countries for the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML) in adult patients resistant or intolerant to at least one prior therapy, including Glivec® (imatinib)*, and in more than 110 countries for the treatment of adult patients with newly diagnosed Ph+ CML in chronic phase. Tasigna is approved in the European Union (EU) for the treatment of Ph+ CML in the chronic phase in pediatric patients with resistance or intolerance to prior therapy including Glivec and for the treatment of pediatric patients with newly diagnosed Ph+ CML in the chronic phase.Please see full Prescribing Information including Boxed WARNING at www.tasigna.com.
About Glivec (imatinib)
Glivec (imatinib) is approved in more than 110 countries, for the treatment of adult patients in all phases of Philadelphia chromosome-positive (Ph+) CML, for the treatment of patients with KIT (CD117)-positive GIST, which cannot be surgically removed and/or have metastasized and for the treatment of adult patients following complete surgical removal of KIT+ GIST.Not all indications are available in every country.
Please see full Prescribing Information available at www.glivec.com.
* Known as Gleevec® (imatinib mesylate) tablets in the US, Canada and Israel.
About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 121,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world.
1. Hochhaus A, et al. Leukemia. 2016;30:1044-1054
2. Giles F, et al. Leukemia. 2013;27:107-112.