
Investigational Sarcoma Drug GPX-150 Gets Orphan Drug Designation
The FDA granted orphan drug designation for a developmental agent known as GPX-150, an analog of doxorubicin currently being tested for use in advanced or metastatic soft-tissue sarcoma.
The US Food and Drug Administration (FDA) granted orphan drug designation for a developmental agent known as GPX-150, an analog of doxorubicin currently being tested for use in advanced or metastatic soft-tissue sarcoma.
Arthur Klausner, CEO of GPX-150’s developer Gem Pharmaceuticals, said in a
The new agent is modified in two locations on the molecule in an effort to reduce the cardiotoxicity commonly associated with doxorubicin. A
That trial used an accelerated dose escalation format, and determined the maximum tolerated dose to be 265 mg/m2; at that level, five of six patients required a dose reduction due to neutropenia. Along with neutropenia, common adverse events included anemia, fatigue, and nausea, but no patients experienced cardiotoxicity while on the study drug. Four patients achieved stable disease, the best response in the study.
As a result, the company began a
The orphan drug designation granted to GPX-150 is designed for agents targeting rare diseases or conditions. It qualifies the drug developer for tax incentives for clinical testing, exemption from prescription drug user fees, and reductions in FDA application fees.
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