dm+d

Unassigned

New Medicines

Stargardt's disease

Information

New molecular entity
Kubota Vision
Kubota Vision

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Stargardts is characterised by an excessive build-up of toxic vitamin A byproducts (e.g. A2E) in the retina. Emuxistat is expected to reduce this by inhibiting a critical enzyme in the visual cycle pathway (RPE65) [2]
Stargardts disease is an uncommon hereditary disorder of the eye that leads to progressive loss of sight. In most cases Stargardts disease is caused by abnormalities in a gene called ABCA4. At the time of designation of orphan drug status, the EMA stated Stargardts disease affects approximately 1 in 10,000 people in the European Union (EU) - equivalent to a total of 52,000 people [6].
Stargardt's disease
Oral