NMPA Grants Conditional Approval to XOSPATA for FLT3mut+ AML


In an early February press release, pharmaceutical company Astellas Pharma Inc. (“Astellas”) shared that XOSPATA (gilteritinib) was given conditional approval in China for the treatment of patients with FLT3-mutated (FLT3mut+) acute myeloid leukemia (AML). The approval was granted by the National Medical Products Administration (NMPA) specifically for patients whose cancer has returned or whose cancer does not respond to treatment. Now, XOSPATA, approved under an accelerated pathway, offers to fill the unmet need for patients with AML in China.


Initially, a research collaboration between Astellas and Kotobuki Pharmaceutical Co. Ltd. (“Kotobuki”) discovered gilteritinib, now marketed as XOSPATA through Japan, some of Europe, and the United States. However, Astellas has exclusive commercialization, manufacturing, and development rights. XOSPATA is a FMS-like tyrosine kinase 3 (FLT3) inhibitor, which makes it an effective treatment for patients with FLT3 mutations.

Thus far, XOSPATA showed benefit for patients with FLT3-ITD and FLT3-TKD mutations. The former mutation is linked to an increased relapse risk and shorter survival rate; this specific mutation affects around 30% of patients with AML. Alternately, FLT3-TKD affects around 7% of patients with AML.

The NMPA’s conditional approval hinged on data from the Phase 3 COMMODORE trial and the Phase 3 ADMIRAL trial. In the COMMODORE clinical trial, researchers evaluated XOSPATA as opposed to salvage chemotherapy. While the trial remains ongoing, some pharmacokinetics insight is being drawn for review.

However, the Phase 3 ADMIRAL clinical trial offered a wealth of information on XOSPATA for patients with relapsed or refractory AML. 319 patients enrolled. Each day, patients received 120mg XOSPATA. Ultimately, the trial determined that XOSPATA increased overall survival rates in comparison to salvage chemotherapy. While the drug was relatively safe and well-tolerated, some adverse reactions did occur. These included:

  • Heightened ALT and AST levels
  • Anemia (low red blood cell count)
  • Thrombocytopenia (low platelet count)
  • Fever with neutropenia (low white blood cell count)
  • Nausea and diarrhea
  • Fatigue

Check out the full study findings in the New England Journal of Medicine.

Acute Myeloid Leukemia (AML)

Acute myeloid (or myelogenous) leukemia (AML) is a cancer of the blood and bone marrow. Normally, bone marrow, spongy tissue inside of our bones, is where our blood cells are formed. However, as these cells begin to develop in patients with AML, the cell DNA becomes damaged or harmed in some way. As a result, the bone marrow does not create healthy cells, but abnormal, immature, and leukemic myeloblasts (white blood cells), red blood cells, and platelets. When these unhealthy cells proliferate, it makes it difficult for healthy blood cells to survive. In many cases, AML can spread throughout the body, impacting the lymph nodes, spleen, or central nervous system (CNS).

AML symptoms include:

  • Pale skin
  • Frequent and recurrent infections
  • Easy and unusual bleeding and bruising
  • Fatigue
  • Fever
  • Bone pain
  • Shortness of breath / difficulty breathing
  • Night sweats
  • Appetite loss
  • Unintended weight loss

Unfortunately, AML diagnoses also come with a high 5-year mortality rate. One representative from Astellas notes that patients with FLT3mut+ AML typically only survive for around 6 months with chemotherapy. Healthline suggests that without the mutation, there is around a 27% 5-year survival rate.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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