MARLBOROUGH, Mass. — Sumitomo Pharma America, Inc. (SMPA) today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to its investigational therapy DSP-3077 for the treatment of retinitis pigmentosa (RP). DSP-3077 is an investigative regenerative cell therapy that leverages allogeneic retinal sheets derived from induced pluripotent stem (iPS) cells.
Orphan drug designation is granted by the FDA to a drug or biological product intended to prevent, diagnose, or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval.2
RP is a rare progressive blinding hereditary condition affecting approximately 1 in 4,000 individuals with very limited, or in many cases, no demonstrated efficacious therapeutic options available. RP first results in loss of night and peripheral vision but ultimately affects central vision in most cases. Although the progression of RP is highly variable among individuals, common pathology includes progressive degeneration (cell death) of rod photoreceptors (cells responsible for scotopic vision) and cone photoreceptors (cells responsible for photopic vision, visual resolution, and color vision).1,3,4
“Degenerative retinal conditions such as RP lack sufficient treatment options and can lead to profound vision loss,” said Tsutomu Nakagawa, Ph.D, President and Chief Executive Officer of SMPA. “Receiving Orphan Drug Designation for DSP-3077 recognizes the significant unmet needs of patients with RP and strengthens our commitment to advancing this program. We are dedicated to working closely with the FDA to progress the clinical development of this investigational treatment with the hopes of bringing a potentially innovative treatment option to people with RP.”
About DSP-3077
DSP-3077 is an investigational allogeneic iPSC-derived 3D retinal organoid, which is processed into retinal sheets with multilayered retinal tissue structure. Retinal sheets include abundant photoreceptor precursors. DSP-3077 is being investigated in a Phase 1/2 clinical study (NCT06891885). The study involves implanting the retinal sheets into the eyes of patients with RP. In the research and development of DSP-3077, SMPA and Sumitomo Pharma Co., Ltd. have been working closely with RACTHERA Co., Ltd. (RACTHERA), and the application of Orphan Drug designation to DSP-3077 also utilized RACTHERA’s technologies and expertise.
About Sumitomo Pharma
Sumitomo Pharma Co., Ltd. is a global pharmaceutical company based in Japan with key operations in the U.S. (Sumitomo Pharma America, Inc.) and Canada (Sumitomo Pharma Canada, Inc.) focused on addressing patient needs in oncology, urology, women’s health, rare diseases, cell & gene therapies and CNS. With several marketed products in the U.S., Canada, and Europe, a diverse pipeline of early- to late-stage assets, we aim to accelerate discovery, research, and development to bring novel therapies to patients sooner. For more information on SMPA, visit our website https://www.us.sumitomo-pharma.com or follow us on LinkedIn.
The Sumitomo corporate symbol mark is a trademark of Sumitomo Pharma Co., Ltd., used under license. SUMITOMO PHARMA is a trademark of Sumitomo Pharma Co., Ltd., used under license. SUMITOMO is a registered trademark of Sumitomo Chemical Co., Ltd., used under license. Sumitomo Pharma America, Inc. is a U.S. subsidiary of Sumitomo Pharma Co., Ltd.
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About RACTHERA
RACTHERA is a joint venture between Sumitomo Chemical Co., Ltd. (Head Office: Chuo‑ku, Tokyo, Japan; “Sumitomo Chemical”) and Sumitomo Pharma Co., Ltd (“Sumitomo Pharma”). The company began operations on February 1, 2025, after inheriting intellectual property and other assets related to the regenerative and cellular medicine business from Sumitomo Pharma. RACTHERA serves as the core organization for research and development in the Sumitomo Chemical Group’s regenerative and cellular medicine business and is engaged in the development of innovative therapies utilizing iPS cells and other modalities.
References
1 O’Neal TB, Tripathy K, Luther EE. Retinitis pigmentosa. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024. Updated February 12, 2024. Accessed March 16, 2026. https://www.ncbi.nlm.nih.gov/books/NBK519504/
2 US Food and Drug Administration. Designating an orphan product: drugs and biological products. Updated August 12, 2024. Accessed March 16, 2026. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
3 Frick KD, Roebuck MC, Feldstein JI, McCarty CA, Grover LL. Health services utilization and cost of retinitis pigmentosa. Arch Ophthalmol. 2012;130(5):629-634. doi:10.1001/archophthalmol.2011.2820
4 Parmeggiani F, Sato G, De Nadai K, Romano MR, Binotto A, Costagliola C. Clinical and rehabilitative management of retinitis pigmentosa: up-to-date. Curr Genomics. 2011;12(4):250–259. doi:10.2174/138920211795860125
Contact
Dave Murdoch – Sumitomo Pharma America, Inc.
Email: [email protected]
Phone: +1 (774) 405-5570