Infantile Neuroaxonal Dystrophy (INAD)
Infantile neuroaxonal dystrophy (INAD) is an ultra-rare, infantile genetic neurological disorder and part of a spectrum of diseases called PLA2G6-associated neurodegeneration. Symptoms usually present between six and 18 months of age and there is often rapid onset of motor and intellectual regression. Later on, diminished muscle tone (hypotonia) and spasticity develop. The disease also leads to problems with vision and the eyes, the autonomic nervous system, and, in a minority of individuals, seizures. Usually, disease progression is rapid, and the disorder is invariably fatal in childhood. There are currently no approved treatments for INAD.
Retrotope is currently developing RT001, its lead clinical candidate, for the treatment of INAD. The company has completed enrolment and expects data to read out from its potentially pivotal Phase 2/3 trial of RT001 in the mid-2021.
RT001 has been granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of INAD. Additionally, the FDA has granted RT001 orphan drug designation for the treatment of PLA2G6-associated neurodegeneration, which includes INAD. The European Medicines Agency has also granted RT001 orphan drug designation for the treatment of INAD.
Additional patient and healthcare provider resources on Infantile neuroaxonal dystrophy are available in the Patient Resources section.