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news.The US Food and Drug Administration (FDA) has granted orphan drug designation for Verastem's orally available compound VS-5584 for use in the treatment of mesothelioma.
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Recently, the company started a Phase I clinical trial evaluating the combination of VS-5584 and VS-6063 to treat patients with relapsed or progressive malignant pleural mesothelioma.
The combination trial is supported by preclinical work showing the synergistic activity of VS-6063 and VS-5584 in mesothelioma models in vitro and in vivo.
Currently, VS-5584 is also being assessed in a Phase I trial in advanced solid tumors where the compound has been generally well tolerated and preliminary activity has been observed, including in mesothelioma.
The company said that some patients have been on trial for more than six months and the maximum tolerated dose of VS-5584 has not been reached.
Verastem president and chief executive officer Robert Forrester said: "This is an important regulatory milestone for Verastem and, together with our European orphan medicinal product designation, will facilitate our global development of VS-5584 to help improve the available treatment options for patients suffering from this highly aggressive cancer.
"We look forward to taking full advantage of the opportunities that orphan designation allows in order to bring this potential new treatment option to patients as rapidly as possible."
VS-5584 has already shown potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2.
In the European Union (EU), VS-5584 has already been granted orphan drug designation for use in mesothelioma.