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FDA grants orphan drug designation to ASLAN003 for acute myeloid leukemia
The FDA granted orphan drug designation to ASLAN003 for the treatment of acute myeloid leukemia, according to the agent’s manufacturer.
ASLAN003 (ASLAN Pharmaceuticals) is an oral inhibitor of human dihydroorotate dehydrogenase (DHODH).
DHODH is the enzyme that controls the rate limiting step in the de novo synthesis of pyrimidines. It also contributes to the production of adenosine triphosphate, according a company-issued press release.
Increased levels of pyrimidines and adenosine triphosphate are necessary for tumor growth and survival. DHODH inhibition leads to induction of the tumor suppressor p53, which — at high levels — can trigger programmed cell death.
A phase 2 trial is underway to assess the agent for treatment of AML. Interim data are expected later this year.
Results of prior studies showed ASLAN003 demonstrated DHODH inhibition up to two orders of magnitude prior than first-generation DHODH inhibitors, according to the release. The agent did not appear associated with the toxicities observed with first-generation inhibitors or other novel AML therapies.