Sumitomo gets orphan drug tag for iPSC-based cell therapy

The U.S. FDA has granted orphan drug designation to Sumitomo Pharma America’s investigational therapy, DSP-3077, for a group of genetic eye disorders known as retinitis pigmentosa (RP).

DSP-3077 is a regenerative cell therapy that leverages allogeneic retinal sheets derived from induced pluripotent stem (iPS) cells, currently being investigated in a phase 1/2 clinical study in retinitis pigmentosa. The study involves implanting the retinal sheets into the eyes of patients with RP. Sumitomo Pharma America — U.S. operations for Japan-based Sumitomo Pharma — is developing the therapy with Tokyo-based Racthera.

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.

Back in February, a panel of experts gave the green light for Japan's Ministry of Health, Labour and Welfare to approve the commercialization of two regenerative medical products that use induced pluripotent stem cells — one of which was another one of Sumitomo’s therapies, known as Amchepry. Amchepry, also developed in collaboration with Racthera, consists of allogeneic iPS cell-derived dopaminergic neural progenitor cells, transplanted into the brains of people with Parkinson’s disease for the improvement of motor functions during the off-time period of patients with advanced disease. It was tested in a small phase 1/2 trial.