J&J seeks US approval for leukemia drug

Tipifarnib, administered orally, is being investigated in patients 65 years of age and older with newly diagnosed acute myeloid leukemia (AML). As a drug intended to treat a life-threatening disease for which there is an unmet medical need, tipifarnib (R115777) was granted fast track status by the FDA in June 2004.

Johnson & Johnson Pharmaceutical R&D (J&JPRD) has submitted the tipifarnib new drug application (NDA) based on phase II clinical data, which use measurements of activity or responsiveness of a disease to the treatment rather than survival endpoints. In October 2004, J&JPRD initiated a phase III study to fully demonstrate the clinical benefit of tipifarnib in this condition.

Under the continuous marketing application (CMA) pilot-1 program, J&JPRD submitted reviewable units of the tipifarnib NDA as they were completed, receiving ongoing feedback from the FDA. Tipifarnib also was granted orphan drug status, a designation given to medications used to treat a rare disease or condition.

In addition to funding global clinical studies for tipifarnib, J&JPRD collaborated with the National Cancer Institute’s (NCI) cancer therapy evaluation program (CTEP) to conduct the pivotal phase II study that supports the tipifarnib NDA.

In October 2004, J&JPRD initiated the phase III international study. J&JPRD continues to investigate other potential uses of tipifarnib in solid and hematologic malignancies, including various stages of myeloid leukemia.

If approved, tipifarnib will be marketed in the US by Tibotec Therapeutics, a division of Ortho Biotech Products. J&JPRD will seek approval from regulatory authorities around the world for tipifarnib.