Capsida Biotherapeutics granted orphan drug designation for potential gene therapy for STXBP1-related disorders
Capsida Biotherapeutics, a biotech company, just announced that the US Food and Drug Administration (FDA) granted an orphan drug designation for a potential gene therapy for STXBP1-related disorders. Very exciting news for our community.
What is an orphan drug designation?
An orphan drug designation can be granted for potential medicines that prevent, diagnose or treat a rare disease. In the US, a rare disease is a condition affecting fewer than 200,000 patients in the US, as defined in the Orphan Drug Act passed by Congress in 1983. The FDA’s orphan drug designation program provides incentives for biopharma and other drug developers to encourage development of medicines for rare diseases, including tax credits for qualified clinical trials. Orphan drug designation is a separate process from seeking approval from the FDA for a treatment. Capsida’s press release highlights its efforts to advance this gene therapy candidate, called CAP-002, to an IND filing in the first half of 2025.
Swati Tole, MD and Alberto Lopez, PhD from Capsida spoke at the STXBP1 Summit Research Roundtable in July 2024. The recording of their talk, “Disease-Modifying Treatment for STXBP1-Encephalopathy: IV Administration of rAAV CAP-002 Increases Brain Levels of STXBP1 Protein” is posted here. The talk starts at 1:09:15.
This Capsida announcement is exciting news, and complements the October 2023 news that Immedica was granted an orphan designation by the European Commission for glycerol phenylbutyrate in STXBP1-related disorders. More information on the Immedica news is provided in this news post.
These announcements also highlight the importance of our community continuing to develop clinical trial readiness including contributing to our registries and natural history studies including the STARR study and the soon-to-launch ESCO study. Our community's work is encouraging biopharma investment and enabling therapy development progress!