Verastem's VS-6063 gets orphan drug status

Hope seems to be on its way to mesothelioma patients. Good news because the European Commission has just granted Verastem, Inc.’s VS-6063 the orphan status.

According to News-Medical.net, Verastem, Inc., has been working hard to discover and develop drugs that will treat cancer aiming at cancer stem cells.  

VS-6063’s orphan designation only serves to encourage the development of the drug, which will provide benefit to patients with mesothelioma cancer, as well as other diseases.

The drug-maker wants to begin “the randomized, double-blind, placebo controlled trial of VS-6063 in mesothelioma” within this year.

"We are pleased that the EMA recognizes the significant unmet medical need in mesothelioma," said Christoph Westphal, M.D., Ph.D., Verastem Chairman and Chief Executive Officer. "This orphan drug designation provides us with a number of benefits in the development of VS-6063."

VS-6063 is described as an oral drug, which acts as a “small molecule inhibitor of focal adhesion kinase” (FAK). Research on FAK signaling pathway reveals that the VS-6063 plays a very important role in cancer stem cell survival and disease progression.

"Mesothelioma is a devastating disease with limited treatment options," Dr. Joanna Horobin, Verastem’s CEO, revealed.

 "We are working with investigators throughout Europe and internationally to bring a new treatment option for these patients."

The company is also working with LabCorp to develop a biomarker, which help identifies a subgroup of mesothelioma patients with low-marker called Merlin.

Almost half of mesothelioma patients have been discovered to be low with Merlin. A study conducted by Verastem and others have found out that Merlin-low mesothelioma cells and tumors are specifically reactive to FAK inhibition.

The clinical study conducted by Verastem is aimed as an “adaptive, double-blind, placebo-controlled trial” to assess VS-6063’s effectiveness when use in mass market, as well as for patients tumors that are low of Merlin-low.

Robert Forrester, Verastem President and Chief Operating Officer said that the company is currently discussing with major drug regulatory agencies worldwide.

"We plan to start the randomized, double-blind, placebo controlled trial of VS-6063 in mesothelioma later this summer."

EMA guidelines provide that drugs with orphan status are granted with up to 10 years of market exclusivity.

When a drug has been declared in the orphan status, the drug company is given EMA assistance to improve the drug’s clinical development and gearing the drug for marketing.

Likewise, EMA-designated orphan drugs are eligible for discount on regulatory fees and European Union-funded research grant.

VS-6063 is also under evaluation for Phase 1/1b trial in conjunction with paclitaxel among ovarian cancer patients.