On April 19, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for CA-4948, a first-in-class, small molecule inhibitor of IRAK4 and Curis’s most advanced therapeutic in clinical development. CA-4948 targets IRAK4-L, the oncogenic isoform of IRAK4 preferentially expressed by the majority of patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) and has shown broad clinical activity in Phase 1 trials in patients with relapsed or refractory (R/R) AML/MDS (Press release, Curis, APR 19, 2021, View Source [SID1234578178]).

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"We are pleased to take this important next step in unlocking the potential of CA-4948 to offer a safe and transformative, disease-modifying alternative treatment for patients on the AML/MDS spectrum," said James Dentzer, chief executive officer of Curis. "Receiving Orphan Drug designation for CA-4948 in AML and MDS represents a significant milestone in our mission of slowing or preventing the progression of disease in patients with these rare hematological malignancies."

Orphan Drug Designation is granted by the FDA to drugs intended to treat rare disorders that affect fewer than 200,000 people in the U.S. The designation can provide development and commercial incentives, including eligibility for seven years of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and exemption from FDA user fees.