The European approval follows a series of international authorizations granted throughout the second half of the year, including clearances in Australia and Argentina in July, followed by U.K. regulators in September. CMP-002 is positioned as a potential first-in-class, disease-modifying treatment for patients struggling with the rare genetic condition.

Despite the immediate market enthusiasm, the stock remains down 9% over the past 12 months. CAMP4 management expects to reach a critical milestone by dosing the first patient before the end of the fourth quarter.