Nerviano Medical Sciences received European Medicines Agency (EMA) Orphan Drug Designation for its next-generation FLT3 inhibitor NMS-03592088 for treatment of Acute Myeloid Leukemia
March 4, 2024

NERVIANO, IT and BOSTON, Mass, 4 March, 2024 – Nerviano Medical Sciences S.r.l. (NMS), a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc. (NMS-US), a wholly owned subsidiary of NMS Group, focused on the discovery and development of oncology drugs and the largest oncological R&D company in Italy, announced that European Commission has granted Orphan Drug Designation (ODD) for NMS-03592088 for the treatment of Acute Myeloid Leukemia (AML), just a few weeks after the US Food and Drug Administration (FDA) approval.

NMS-0359288 is a next generation FLT3 inhibitor with superior activity and selectivity with respect to approved FLT3 inhibitor drugs and higher potency on the resistance mutation F691L opening opportunities for treatment of patients who failed prior FLT3 inhibitor treatment.  NMS-03592088 is being evaluated as monotherapy in a Phase I/II study in relapsed/refractory FLT3 positive AML in Europe and US. Preliminary evidence of clinical activity was observed during the dose escalation phase when administered as salvage line therapy including patients with prior FLT3 inhibitors (AACR2023). Based on these results the Committee for Orphan Medicinal Products released its positive opinion stating that “the drug showed responses in heavily pretreated patients with acute myeloid leukaemia including those who have failed treatment with the currently authorized medicinal product. The Committee considered that this constitutes a clinically relevant advantage.”

After having completed Phase I in Europe, the trial is currently enrolling patients in multiple Phase II cohorts (NCT03922100).

As we expand our focus to embrace the orphan drug destination in the EU for NMS-03592088, following the ODD approval by FDA last December, we are determined to bring new therapies to AML patients.  Our dedication to innovation for patients worldwide is strong .” said Lisa Mahnke, MD, PhD, CMO of NMS | CEO and Managing Director of NMS-US.

We are diligently implementing strategies to accelerate the development of NMS-03592088 as a promising new therapeutic option for patients battling AML with very few options. The Orphan Drug Designation is a key regulatory milestone that acknowledges the potential of NMS-03592088 for positioning as next generation FLT3 inhibitor and reinforces our commitment to expedite its development through the approval process” said Elena Ardini, MSc, Asset Leader of NMS.

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medical Association (EMA) Committee for Orphan Medicinal Products. The EMA grants orphan designation for the treatment, diagnosis or prevention of life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 persons in the EU. Drugs that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, reduced regulatory fees, access to protocol assistance from the EMA during development and access to centralized marketing authorization.

Link PR: 20240304_FLT3 Project_ODD_PR


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