Avidity Biosciences (RNA) Shares Soar 2.99% on Orphan Drug Designation

Avidity Biosciences (RNA) shares surged 2.99% today, marking a significant rebound after hitting a low not seen since March 2024, with an intraday decline of 10.67%.

Avidity Biosciences has recently received Orphan Drug designation in Japan for its investigational treatment delpacibart etedesiran (del-desiran) for myotonic dystrophy type 1 (DM1). This designation underscores the potential of del-desiran to address the root cause of DM1, a progressive neuromuscular disease with no approved therapies. The designation offers several benefits, including prioritized consultation regarding clinical development, tax incentives, and priority review of applications. The company is advancing its global commercialization preparations and expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025, aiming to submit marketing applications starting in 2026 in the U.S., European Union, and Japan. This development could positively impact the company's stock price due to the potential market expansion and regulatory progress.