Press Release

April 19, 2021
1 min read

FDA grants orphan drug designation to CA-4948 for AML, myelodysplastic syndrome


Press Release

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The FDA granted orphan drug designation to CA-4948 for treatment of acute myeloid leukemia and myelodysplastic syndrome, according to the agent’s manufacturer.

CA-4948 (Curis) is a small-molecule inhibitor that targets IRAK4-L, the oncogenic isoform of IRAK4 preferentially expressed by most patients with AML and myelodysplastic syndrome (MDS).

Blast PC for AML
Source: Adobe Stock.

Results of phase 1 trials showed the agent has clinical activity in patients with relapsed or refractory disease.

“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," James Dentzer, CEO of Curis, said in a company-issued press release. “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 [hematologic] malignancies.”

CA-4948 also is being evaluated as monotherapy and in combination with ibrutinib (Imbruvica; Janssen, Pharmacyclics) for treatment of non-Hodgkin lymphoma.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.