Application submitted to MFDS for the designation of VascoStem, a stem cell treatment agent for Buerger’s disease, as an orphan drug

• Application submitted with new medical evidence
• A strategy to attract foreign patients

The Biostar Stem Cell Research Institute (President Ra Jeong-chan), co-operated by Nature Cell and R Bio, announced that an application was submitted to the Ministry of Food and Drug Safety (MFDS) of Korea on December 10, 2015 for VascoStem to be designated as an orphan drug for treatment of Buerger’s disease, or thromboangiitis obliterans.

VascoStem is an autologous adult stem cell treatment agent, which involves collecting 10 to 20cc of fat tissue from a Buerger’s disease patient and culturing 300 million stem cells high-purity adipose-derived mesenchymal stem cells (for an adult with a body weight of 60kg) to be administered to the areas of pain through intramuscular injections. A single treatment can help prevent severe pain, difficulty in walking and the risk of limb amputation.

Biostar commenced a research and development (R&D) project in 2005, and successfully developed autologous adult stem cell treatment agents including VascoStem after investing nearly KRW 100 billion in direct and indirect costs.

VascoStem was developed and clinically tested for treatment of Buerger’s disease, which is a rare disease, with the aim of obtaining a designation as an orphan drug for early commercialization. It has become Biostar’s first commercialized product.

An application for an orphan drug designation had been submitted to MFDS earlier this year on June 30, but it was voluntarily withdrawn by Biostar when there was a delay in receiving a positive response. An application was re-submitted recently, with supplementation of new medical evidence.

Biostar conducted a 2-year follow-up study after completing the first and second phases of its clinical trial. In addition to observing no adverse events among the participants, the results showed that the participants were able to cease the administration of related medications including analgesics and that none of the participants had their limbs amputated. Based on these results, Biostar deems that VascoStem satisfies the criteria for the designation of orphan drugs.

Meanwhile, Nishihara Clinic in Japan has obtained approval from the Ministry of Health, Labour and Welfare for regenerative treatment with the drug in question, based on the technical data provided by Biostar, and is currently using the drug for treatment of critical limb ischemia including Buerger’s disease and diabetic foot ulcers.

Ra Jeong-chan, the President of the Biostar Stem Cell Research Institute, said, “I don’t believe that human actions and practices can be completely free of flaws. A specialized review and consideration of patient health and national interests are necessary, in accordance with the purport of the orphan drug designation system.” He also revealed the future plans of the institute stating, “Once VascoStem is commercialized, we will make an aggressive effort to attract foreign patients to Korea as a means to generate national wealth and promote national prestige. This will dispel the misunderstandings related to the overseas procedural treatments that Koreans have received in the past.”