Specialty pharmaceutical firm Insys Therapeutics announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its Liposome Entrapped Paclitaxel Easy to Use (LEP-ETU) drug lead for gastric cancer.
Paclitaxel, a commonly used chemotherapeutic agent, is an anti-microtubular network agent, which is clinically active against a wide range of malignancies. However, due to its poor solubility, the drug is formulated along with or bound to a delivery vehicle, such as polyethoxylated castor oil or albumin nano-particles. Insys’ LEP-TEU is an enhanced formulation of paclitaxel. The company acquired the drug from its merger with NeoPharm in 2010.
The drug is being developed as treatment for gastric cancer, currently the fifth leading cancer in the world and the third most common cause of cancer-related death. An estimated 22,000 people will be diagnosed with gastric cancer in 2014.
Michael L. Babich, President and CEO of Insys Therapeutics, said, “We are pleased to have received orphan drug designation for LEP-ETU to treat gastric cancer, a devastating and difficult-to-treat disease. By entrapping the paclitaxel with liposomes, we believe our LEP-ETU formulation could potentially have reduced toxicity, while maintaining or enhancing efficacy of the cancer-fighting paclitaxel.”
Insys said that is studying the next development steps it should take for its orphan drug candidate.
The FDA’s orphan status is awarded to new drugs or biologics that target rare diseases or conditions present in less than 200,000 in the U.S. Insys stands to gain a seven-year period of marketing exclusivity in the U.S. upon approval of LEP-ETU for its orphan indication.
The company previously won an orphan designation from the FDA. In late August, the company received its second orphan drug status for its cannabidiol product as treatment for glioblastoma multiforme (GBM). The FDA also awarded orphan designation for its pharmaceutical cannabidiol (CBD) for rare pediatric-onset epilepsy, Dravet syndrome.