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Multiple Stargardt Contenders Progress In Japan Despite Small Market

Medical Need, Supportive Regulations

 
• By Lisa Takagi

With no approved therapies globally for Stargardt's, competition in the indication is increasing in Japan, where Kubota Vision is seeking for conditional approval for emixustat, while Belite Bio’s contender tinlarebant has received Sakigake designation as a local Phase III program continues.

Japan has two P3 candidates for Stargardt's Disease.
Japan has two candidates for Stargardt in Phase III, despite small local market • Source: Shutterstock

Against a background of the globally strengthening R&D focus on rare diseases, Japan is seeing unusually fierce competition in the relatively small indication of Stargardt disease, a rare ophthalmologic disorder where outstanding medical needs remain high, and helped by potential fast-track regulatory treatment.

With no approved therapeutics on the market anywhere worldwide, the most advanced candidate in the country is emixustat, an oral RPE65 inhibitor developed by Kubota Vision Inc

Key Takeaways
  • Despite a small market, Japan is seeing strong competition in the Stargardt disease segment, with two oral candidates now in Phase III. There are no approved products globally for the rare ophthalmic disorder.
  • Despite Kubota Vision’s emixustat missing its Phase III endpoints, the firm is pursuing a conditional approval pathway in Japan, backed by a push from patients' groups

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