Mithridion has received orphan drug designation for its leading compound, MCD-386CR. The drug is intended to treat progressive supranuclear palsy, or PSP, a degenerative brain disease that affects balance, walking, speech, cognition and eye movements.
Getting orphan drug status gives MCD-386CR seven years of market exclusivity as a treatment for PSP, if it eventually is approved by federal regulators, said Mithridion chief executive Trevor Twose. It also provides eligibility for federal grants, tax credits and fee waivers.
Perhaps more importantly, this drug could potentially give some measure of hope for patients with PSP.
To learn more about orphan drugs and treatments for rare diseases check out the World Orphan Drug Congress USA.