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Orphan Drug Approval Could Mean New Mesothelioma Treatment Option

26133630_mesothelinA drug designed to fight cancer by destroying the stem cells that give rise to new cancer cells has been granted orphan drug status for the treatment of malignant mesothelioma.

The designation paves the way for mesothelioma patients, who often have few treatment options, to get access to VS-5584 earlier than it would otherwise be available.

VS-5584 is a powerful inhibitor of a signaling pathway that regulates cancer progression and the survival of cancer stem cells. According to Verastem, the Boston-based maker of VS-5584, the drug has already received a similar designation for mesothelioma in Europe.

“This is an important regulatory milestone for Verastem and… will facilitate our global development of VS-5584 to help improve the available treatment options for patients suffering from this highly aggressive cancer,” Robert Forrester, Verastem President and Chief Executive Officer, said in a February news release.

VS-5584 and another Verastem drug called VS-6063 are the focus of a new Phase 1 clinical study in patients with pleural mesothelioma. Early laboratory and animal data suggest that the two drugs may work synergistically to destroy mesothelioma and other solid tumors. Six months into the Phase I human trial of the combination, the company reports it “has been well-tolerated” and that no one has had to stop taking it because of toxicity.

The orphan drug designation was created to encourage the development of drugs designed to treat people with rare diseases such as mesothelioma. Depending on the outcomes of the current research, orphan drug status for VS-5584 will enable Verastem to bring the drug to market faster and less expensively.

Although it is very rare, mesothelioma is one of the hardest cancers to treat. The irregular shape and difficult location of mesothelioma tumors, coupled with the cancer’s fast growth and resistance to chemotherapy, mean that many patients die of the disease within a year of diagnosis.


“Verastem Receives Orphan Drug Designation from FDA for VS-5584 in Mesothelioma”, February 12, 2015, News release, Veratsem website, http://phx.corporate-ir.net/phoenix.zhtml?c=250749&p=irol-newsArticle&ID=2016066

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