Pharmosa Biopharm announced on the 16th that its inhaled drug L608 for the treatment of systemic sclerosis (SSc) has been granted orphan drug designation by the European Medicines Agency (EMA), following a similar designation by the U.S. FDA.

Kan, Pei, General Manager of Pharmosa Biopharm, stated that the company is actively engaging in discussions for potential international licensing and partnership opportunities, leveraging supportive rare disease policies in both the U.S. and Europe to expand its global market presence through strategic alliances.

L608 is administered via home-based inhalation therapy, offering patients a more convenient treatment option.

With orphan drug designation granted in both the U.S. and Europe, the drug is now eligible for various incentives, including regulatory support, accelerated review processes, and market exclusivity of 7 to 10 years after approval.

Kan further noted that in addition to targeting SSc-RP/DU (systemic sclerosis-related Raynaud’s phenomenon and digital ulcers), L608 is also being developed for other indications such as pulmonary arterial hypertension. The company aims to accelerate global development and commercialization through orphan drug incentive programs and a regionally tailored combination product strategy.

Resource: 治療系統性硬化症新藥 國邑 獲歐盟孤兒藥認定