Retrotope Receives FDA Clearance to Commence RT001 Phase 2/3 Clinical Trial in Patients with Infantile Neuroaxonal Dystrophy (INAD)
Retrotope announced today that the U.S. Food and Drug Administration (FDA) has granted the company approval to conduct an open-label Phase 2/3 clinical trial of its investigational drug RT001 to evaluate
efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD). RT001 is the
first-in-class of a new category of drugs called D-PUFAs (deuterated polyunsaturated fatty
acids), which are designed to protect against free radical damage resulting in cell death that
is a hallmark of numerous neurodegenerative diseases including INAD. To date, Retrotope
has enrolled two INAD patients in two separate Expanded Access trials, the first patient
having begun treatment in March 2017 and the second patient having enrolled in November
2017. Promising results from the first study were presented at the recent annual meeting of
the American Academy of Neurology, and treatment of both children remains ongoing.
RT001 has been granted U.S. FDA orphan drug designation for the treatment of PLA2G6
associated neurodegeneration (PLAN), which includes INAD.
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