Taiwan Liposome Company, Ltd.,(TLC) (4152:TT) today announced that its oncology drug candidate TLC178 has been designated as a drug for a rare pediatric disease in the treatment of rhabdomyosarcoma (RMS) by the U.S. Food and Drug Administration (FDA).
This designation brings the company closer to receiving a Rare Pediatric Disease Priority Review Voucher, which can be used to expedite the review process or transferred to another company.
Rhabdomyosarcoma (RMS) is an aggressive and highly malignant form of cancer that develops from skeletal muscle cells that have failed to fully differentiate. RMS predominantly afflicts children. More than half of patients diagnosed with RMS are younger than 10 years of age and RMS accounts for approximately three to four percent of all cases of cancers diagnosed in childhood. About 350 new cases of RMS occur each year in the U.S.
RMS patients are typically treated with surgery to remove tumor tissue, which is followed by chemotherapy to decrease recurrence. Vincristine is currently the most common chemotherapeutic therapy for RMS. Vincristine is a vinca alkaloid similar to vinorelbine.
TLC178 consists of vinorelbine encapsulated using the NanoXTM nanotechnology platform to provide an improved safety profile relative to vinorelbine alone. Studies using preclinical models have demonstrated promise for TLC178 in the treatment of RMS.
The FDA’s Office of Orphan Products Development has designated TLC178 (vinorelbine tartrate liposome injection) for the treatment of RMS as a drug for a rare pediatric disease.
This designation may lead to an award of a priority review voucher, which can be redeemed to receive a priority review of a subsequent marketing application for a different product or sold to another company. A priority review voucher can shorten the FDA review process from at least ten months to six months.
A Phase 1/2 dose-escalation study of TLC178 is currently underway at sites in Taiwan and in the U.S. to evaluate the maximum tolerated dose (MTD). Once established, the MTD will be used in pivotal studies.