GI Innovation said on Thursday that it has received orphan drug designation (ODD) from the U.S. FDA for its Merkel cell cancer (MCC) drug, GI-101.

MCC is a rare skin cancer which appears as a flesh-colored or bluish-red nodule, on the face, head or neck with a tendency to spread quickly to other parts of the body. Long-term sun exposure or a weak immune system are cited as risk factors.

These modules usually appear at the dermis and epidermis of the skin, and 90 percent are elderly patients aged 50 or older. Additionally, about 5 to 12 percent of them are diagnosed with metastatic MCC, and the probability of survival for more than 5 years is less than 20 percent.

Pfizer’s Bavencio (ingredient: avelumab), the first immuno-cancer drug approved for MCC, was accelerated because it also received FDA ODD, reaching markets just three years after the start of clinical development. Other existing approved drugs for MCC include MSD’s Keytruda (pembrolizumab) but are not effective in more than half of the patients, leaving them with no treatment options after  failed treatment with these immune anticancer drugs.

"We have been discussing with the Memorial Sloan Kettering Cancer Center in the U.S. and key opinion leaders across the U.S. for more than a year to provide treatment options for rare diseases patients for whom there is no appropriate treatment," said GI Inno’s CSO Jang Myoung-ho. “This FDA designation will give momentum to our MCC clinical development.”

CEO Rhee Byung-geon added, "With the rapid growth of the rare drug market, pharmaceutical biocompanies around the world are fiercely competing to approve rare drugs."

Rhee emphasized that his company will pay more attention to the unmet medical needs and take the lead in developing new drugs.

The ODD is a permit system for intractable diseases with an annual prevalence of less than 100,000 among rare diseases. It includes benefits such as exemption from submitting clinical stage pediatric data, exemption from various licensing agency fees, exemption from taxes incurred during clinical trials, FDA clinical trial advice and market exclusivity for  seven years.