FDA grants orphan drug designation to inecalcitol for AML

The FDA granted orphan drug designation to inecalcitol for the treatment of patients with acute myeloid leukemia, according to a press release from the drug’s manufacturer.

Inecalcitol (Hybrigenics) demonstrated in vitro and in vivo preclinical evidence of synergy with azacitidine (Vidaza, Celgene) and decitabine (Dacogen, Janssen-Cilag), two hypo-methylating anticancer drugs. This synergistic effect inhibited the growth of human AML cell lines in vitro and in vivo, prolonging survival in mice in two different experimental models of AML.

A phase 2 clinical trial showed single-agent inecalcitol demonstrated an inhibitory effect in half of treated patients with chronic lymphocytic leukemia, according to the press release.

Enrollment is ongoing for another phase 2 clinical trial that will evaluate inecalcitol combined with imatinib (Gleevec; Novartis) in patients with CLL.

“This orphan drug designation by the FDA will make the costs of clinical development of inecalcitol in the United States eligible to the so-called orphan drug tax credit,” Remi Delansorne, PhD, CEO of Hybrigenics, said in a press release. “This incentive will substantially support our strategy to undertake a significant part of this phase 2 clinical study in the United States, where the frequency of [AML] seems to increase. This leukemia remains the blood cancer with the lowest 5-year survival rate: 25% at best.”

The FDA’s 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.