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FDA grants orphan drug designation to GPX-150 for sarcoma
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The FDA granted orphan drug designation to GPX-150 for the treatment of patients with soft tissue sarcoma, according to a press release from the drug’s manufacturer.
GPX-150 (Gem Pharmaceuticals), an anthracycline analog, is being evaluated in an open-label phase 2 clinical trial as first-line therapy for adults with advanced or metastatic soft tissue sarcoma.
The trial design calls for patients to receive GPX-150 infusions every 3 weeks for up to 48 weeks, a duration twice as long as the standard 24-week limit for doxorubicin administration, according to a description of the agent on Gem Pharmaceuticals’ website.
The trial is fully enrolled with 30 patients, and efficacy and safety data are expected by the end of this year. PFS rate at 12 months serves as the trial’s primary endpoint.
“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,” Arthur Klausner, CEO of Gem Pharmaceuticals, said in a company-issued press release.
The FDA 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.