Sumitomo Pharma America announced that its investigational therapy DSP-3077 has received orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of retinitis pigmentosa (RP). DSP-3077 is an allogeneic retinal sheet derived from induced pluripotent stem (iPS) cells, designed to replace degenerated photoreceptor cells in the retina. The therapy is currently being evaluated in a phase 1/2 study, which involves implantation of multilayered retinal sheets into patients with RP.

Orphan Drug designation is intended for therapies targeting rare conditions and provides benefits including clinical trial tax credits, user fee exemptions, and potential 7-year market exclusivity if approved. Sumitomo Pharma America is collaborating with Racthera Co, Ltd, whose technologies were incorporated into the investigational retinal sheets. RP