European orphan drug designation is granted for products intended to treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 persons.
The EC Commission concluded that Debio-0513 meets these requirements for the treatment of MG, as the disease only affects approximately 0.7 to 1.5 in 10,000 people in Europe.
In addition, orphan drug status of Debio-0513 in Europe can confer numerous benefits to its development, including clinical protocol assistance, reduced registration fees when filing for product approval and market exclusivity for a period of up to 10 years.
“Due to its unique mechanism of action, we hope that Debio-0513 will become an important novel treatment opportunity for MG sufferers. It has the potential to offer patients benefits such as modifying the progressive course of the disease, preventing relapses, and possibly working in combination with other available treatments. The sparing effect of Debio-0513 should decrease the risks usually related to current medications like steroids and other immunosuppressant,” said Loic Maurel, president and CEO of the Debiopharm Group Canadian subsidiary.