Potential Mesothelioma Drug Wins Orphan Status from the European Commission

Verastem Inc. recently announced that they have received orphan drug status from the European Commission for a new drug that has been designed to treat mesothelioma. The goal of orphan status, both in Europe and in the United States, is to encourage the development of drugs for rare diseases such as this.

“Mesothelioma is a devastating disease with limited treatment options,” said Dr. Joanna Horobin, Verastem chief medical officer, in a recent press release. “We are working with investigators throughout Europe and internationally to bring a new treatment option for these patients.”

The name of the new drug candidate is VS-6063, announced the company. It will be studied in a Phase I clinical trial that is scheduled to begin later this summer. Currently, the drug maker, along with LabCorp, is developing a biomarker test to determine which mesothelioma victims are the best candidates for the trial. In addition, a Phase I/Ib trial using VS-6063 along with another chemotherapy drug is currently being offered to some ovarian cancer patients.

In Europe, Orphan Medicinal Product Designation allows for the drug developer to take advantage of up to 10 years exclusivity if the drug is approved for use in Europe. The status also allows U.S. companies to have greater interaction with the European drug regulatory agency in regards to clinical trials, notes an article in the Boston Business Journal.

Currently, the most widely-used drug in the United States for the treatment of malignant pleural mesothelioma is Alimta©, a chemotherapeutic agent developed by pharmaceutical giant Eli Lilly. It is often used in conjunction with other forms of mesothelioma treatment, including surgery and/or radiation. Still, the prognosis for most meso patients is not good, hence the need for better drugs that prolong lives.