China has recently approved CStone Pharmaceuticals’ Tibsovo (ivosidenib tablets) for the treatment of adult patients with relapsed/refractory acute myeloid leukemia (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, providing a new precision therapy for this patient population. With multiple precision therapies now available, the cancer treatment arsenal is expanding for adult acute myeloid leukemia (AML) patients in the country, says GlobalData, a leading data and analytics company.

So far in China, only four other marketed products have been approved for AML. Zavedos (idarubicin), Vidaza (azacytidine), and Daunoblastin (daunorubicin) are  chemotherapies, and Xospata (gliteritinib) from Astellas is a precision therapy targeting FMS-like tyrosine kinase 3 (FLT3) mutations.

AML is a genetically heterogeneous disease characterized by recurrent gene mutations thereby limiting the treatment efficacy in AML patients. Although six recurrent gene mutations have been identified,  FLT3 and IDH 1/2 genetic mutations have emerged as the key targets for the development of precision therapies.

Precision medicine is an emerging and revolutionary approach of targeted therapy based on the genetic constitution of a patient and susceptibility to disease related mutations.

Sasmitha Sahu, Pharma Analyst at GlobalData, comments: “Chemotherapy is the first line of therapy for the treatment of AML in China. With an improved diagnostic landscape based on genetic assays, there is an increased need for the availability of precision therapies that target a specific mutation contributing to poor prognosis in AML. Therefore, the availability of a second precision therapy provides an expanded treatment option for AML patients.”

According to GlobalData, China has the highest incidence rates for AML among the eight major markets (8MM*). The total diagnosed AML incidence cases in China are expected to reach almost 40,000 in 2029, with nearly 7.1% cases accounting for IDH1 mutations. The treatment market for AML in China will expand from US$94.9m in 2021 to US$451.9m by 2029, representing a CAGR of 21.5%.

CStone is also evaluating ivosidenib in combination with azacitidine in a Phase III global trial, which also includes Chinese AML patients.

Sahu continues: “Due to limited safe and efficient therapies in newly diagnosed AML patients and those refractory/resistant to intensive chemotherapy, a combination of precision therapies with other therapies like azacitidine are being explored for improved survival rates and to address adverse outcomes.”

With continued prioritization of personalized medicine in its 14th Five-Year Plan, and Vision 2035, China is approving precision therapies on a priority basis. However, the implementation of precision medicines in Chinese medical practice is still in nascent stages due to cost and reimbursement barriers.

Sahu concludes: “China has a dominant visibility in the precision medicine field backed by  continued government vision and funding, which further accelerated the application of cost- and time-effective next-generation sequencing (NGS) technologies. Oral therapies are likely to reduce the economic burden associated with in-patient medical costs and treatment toxicities during chemotherapy in relapsed patients. Hence a comprehensive reimbursement policy framework around these therapies is needed to drive further uptake.”

*8MM: The US, France, Germany, Italy, Spain, the UK, Japan, China