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November 23, 2020
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FDA grants regulatory designations to PTC596 for sarcoma, glioma subsets

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The FDA granted fast track and orphan drug designations to PTC596 for treatment of leiomyosarcoma, according to the agent’s manufacturer.

The agency also granted rare pediatric disease designation and orphan drug designation to PTC596 (PTC Therapeutics) for treatment of diffuse intrinsic pontine glioma, a rare form of childhood glioma.

“We are very pleased with the FDA’s decisions to grant PTC596 these designations,” Stuart W. Peltz, PhD, CEO of PTC Therapeutics, said in a company-issued press release. “This brings us one step closer to providing truly novel therapeutic approaches to patients with devastating rare cancers seen in children and adults that do not have good treatment options.”

PTC596 is a small-molecule tubulin-binding agent designed to arrest tumor cells in G2/M phase by inhibiting tubulin polymerization, according to the release.

A phase 1b study is underway to evaluate the agent as treatment for leiomyosarcoma, a form of soft tissue sarcoma.

An estimated 4,000 people in the United States are diagnosed with the disease each year. About 40% of them will develop metastases, and only 13.6% of those with metastatic disease survive 5 years.

PTC596 also is under investigation as treatment for diffuse intrinsic pontine glioma, which accounts for 10% to 15% of pediatric brain tumors.

An estimated 300 people in the United States are diagnosed with the malignancy each year. Median OS for patients who receive standard radiation therapy is about 9 months, and less than 10% of patients survive 2 years.