In Massachusetts, a biopharmaceutical company dedicated to the development of new cancer drugs recently announced that it is seeking orphan drug designation in the U.S. and in Europe for the use of VS-6063 to treat mesothelioma.
If the drug is designated an orphan drug, exclusivity will be granted to the manufacturer for the use of their drug to treat a specific condition for seven years after the drug has been approved for marketing by the FDA. This gives pharmaceutical companies financial incentives to create drugs for rare diseases. A rare disease is defined as a disease that affects fewer than 200,000 people in the United States.
The company in question, Verastern, Inc. is a clinical-stage biopharmaceutical company that focuses on creating drugs that aid in the targeted killing of cancer stem cells, a major cause of cancer recurrence and the spread of malignant tumors.
Verastem drugs are designed to disrupt signaling pathways that allow cancer cells to proliferate. The drug VS-6063 has already demonstrated anti-cancer activity in early trials and is currently being evaluated in clinical trials with ovarian cancer patients. Later this year, the same drug will enter a pivotal mesothelioma study.