Pharmosa Biopharm announced on June 24 that it has received official written notification from the European Commission, confirming that its investigational drug L608 has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the treatment of systemic scleroderma (SSc).

The company noted that L608 was previously granted Orphan Drug Designation by the U.S. FDA in December 2023. With this additional designation from the EMA, L608 is now eligible for a range of regulatory incentives in both the U.S. and EU, including administrative support, accelerated review processes, and market exclusivity of seven and ten years respectively upon approval—benefits that are expected to accelerate the drug’s global commercialization.

L608 is being developed specifically for the treatment of systemic sclerosis-associated Raynaud’s phenomenon and digital ulcers (SSc-RP/DU). Pharmosa Biopharm has completed consultations with the U.S. FDA regarding its clinical development plan and is now in the final stages of preparing for a combined Phase II/III trial application. Simultaneously, the company plans to engage with EMA and other regulatory agencies in Europe to coordinate its next steps in clinical development.

In addition to its development for SSc-RP/DU, L608 is also being investigated for the treatment of pulmonary arterial hypertension (PAH), among other potential indications. With the support of global orphan drug regulatory frameworks and its potential across multiple indications, L608 is expected to progress rapidly toward market approval. The company is actively seeking licensing and collaboration opportunities to advance its commercialization strategy.

According to research by Precedence Research, the global market for systemic scleroderma treatments is projected to grow from USD 2.6 billion in 2024 to USD 4.3 billion by 2034, representing a compound annual growth rate of over 5%.

Resource: 國邑新藥 L608獲歐盟委員會正式授予孤兒藥資格認定