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FDA Gives Orphan Drug Designation to Verastem for VS-5584 in Mesothelioma

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U.S. drugmaker Verastem, Inc. has been granted orphan drug status from the Food and Drug Administration (FDA) for the novel stem-cell targeting cancer drug VS-5584 in mesothelioma.

Orphan drug status is given to drugs that are intended to treat a rare disease or condition and provides certain incentives, such as U.S. market exclusivity and tax credits, for the drug’s sponsor. Mesothelioma, an asbestos-associated cancer affecting the lining of the lungs, heart, and abdomen is diagnosed in roughly 3,000 U.S. patient per year.

Stem-cell immunotherapy targeting cancer drugs such as VS-5584 seek to treat cancer at its root: the stem cells that allow cancer to survive and proliferate. They do this by inhibiting cancer stem cell signaling pathways.

VS-5584 targets class 1 P13K enzymes, which are involved in cellular functions like cell growth and survival, as well as mTORC1 (a protein complex that controls protein synthesis) and mTORC2 (a protein complex that regulates cellular metabolism). Verastem has patent protection of VS-5584 through 2029.

U.S. orphan drug designation for VS-5584 in mesothelioma comes less than a month after European orphan drug designation for VS-5584 in mesothelioma.

“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,” said Verastem Chief Executive Officer Robert Forrester in a company press release.

VS-5584 is being tested on mesothelioma patients in two clinical trials. A Phase 1 clinical trial that is evaluating VS-5584 in combination with VS-6063 (defactinib, another experimental cancer drug) in U.S. and U.K. mesothelioma patients recently got underway.VS-6063 is also being evaluated independently in the ongoing COMMAND trial in mesothelioma.

A second Phase I clinical trial is looking at the effectiveness of VS-5584 on advanced solid tumors, including mesothelioma tumors.

Patients with mesothelioma, which is resistant to chemotherapy, have few treatment options. Diagnosis of the incurable cancer can also prove difficult, further hampering treatment. Novel therapeutic, diagnostic, and prognostic approaches offer the best hope for beating not only mesothelioma, but all cancer types.

Verastem’s CEO says that the company looks 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.”

You can learn more about cancer stem cells and cancer treatment at Verastem.com.

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