The FDA has granted Orphan Drug designation to Gem Pharmaceutical's GPX-150, the company’s lead product candidate that currently is undergoing a phase II clinical trial in patients suffering from soft tissue sarcoma.
The study is fully enrolled, with final safety and efficacy data expected to be available by this year’s fourth quarter.
Orphan drug designation is granted for novel drugs to treat rare medical diseases or conditions that affect less than 200,000 people in the U.S. The designation qualifies the sponsor for various incentives, including seven years of market exclusivity after the drug’s approval, tax credits for clinical research costs and reductions in FDA application fees.
“This Orphan Drug designation represents an important regulatory advance as we further our development of GPX-150, which has demonstrated intriguing pharmacological activity in sarcoma patients in our ongoing, open-label, phase II clinical study,” said Arthur Klausner, CEO of Gem Pharmaceuticals.
Soft tissue sarcoma (STS) includes a group of malignancies that affect tissues such as fat, muscles, nerves, and tendons. According to the American Cancer Society, 12,000 new cases of STS occur in the U.S. annually, with about 5,000 deaths.