Sumitomo Pharma America (SMPA) announced that the FDA has granted Orphan Drug designation to its investigational therapy DSP-3077 for the treatment of retinitis pigmentosa (RP). DSP-3077 is described as an allogeneic regenerative cell therapy that uses retinal sheets derived from induced pluripotent stem (iPS) cells and is currently being evaluated in a Phase 1/2 clinical study in patients with RP.
According to SMPA, Orphan Drug designation is granted by the FDA to drugs or biologics intended to prevent, diagnose or treat rare diseases or conditions and can provide incentives such as tax credits for qualified clinical trials, exemption from user fees and the potential for seven years of market exclusivity following approval. RP is characterized as a rare, progressive, hereditary blinding condition affecting approximately 1 in 4,000 individuals and is associated with very limited, and in many cases no, demonstrated efficacious therapeutic options. The disease typically begins with loss of night and peripheral vision and in most cases progresses to affect central vision as photoreceptor cells in the retina degenerate over time.
SMPA noted that common pathology in RP includes progressive degeneration of rod photoreceptors, which are responsible for scotopic (low-light) vision, and cone photoreceptors, which support photopic (daytime) vision, visual resolution and color perception. The company stated that degenerative retinal conditions such as RP lack sufficient treatment options and can lead to profound vision loss, and characterized the Orphan Drug designation for DSP-3077 as recognition of the significant unmet medical need in this population. SMPA said it intends to work closely with the FDA to advance the clinical development of DSP-3077.
DSP-3077 is described as an investigational allogeneic iPSC-derived three-dimensional retinal organoid that is processed into retinal sheets with a multilayered retinal tissue structure containing abundant photoreceptor precursors. In the ongoing Phase 1/2 clinical study (NCT06891885), these retinal sheets are implanted into the eyes of patients with RP. SMPA and Sumitomo Pharma Co., Ltd. have been collaborating with RACTHERA Co., Ltd. on the research and development of DSP-3077, and the company said that technologies and expertise from RACTHERA were also utilized in the application for Orphan Drug designation.
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