FDA Orphan Drug Designation for Spinocerebellar – Ataxia

FDA Orphan Drug Designation for Spinocerebellar

FDA Orphan Drug Designation for Spinocerebellar

IntraBio, a U.K. based biopharmaceutical company, has received an Orphan Drug Designation from the US Food and Drug Administration (FDA) for its lead compound (IB1000) for the treatment of spinocerebellar ataxias (SCA), of which there are over 40 known subtypes.

Intrabio’s Senior-Vice President Taylor Fields told Ataxia UK “Although we have only investigated the clinical efficacy of IB1000 in a small number of SCA subtypes, we were pleased that the safety and efficacy data was so compelling that the compound was granted a single designation covering all SCAs.”

This designation is the third Orphan Drug Designation the FDA has granted to IB1000, along with the previous designations granted for Niemann-Pick type C (NPC) and GM2 Gangliosdosis (Tay-Sachs and Sandhoff).  In addition, IntraBio has been granted two Orphan Medicinal Drug Designations by the European Commission for IB1000 for the treatment of NPC and GM2 Gangliosdosis. In May, 2018, IntraBio filed an Orphan Medicinal Drug Application for SCA with the EMA, which they hope to receive between July and September 2018.  

These designations greatly support IntraBio’s plan to conduct a multinational, multicenter, placebo controlled clinical study for the treatment of inherited cerebellar ataxias, NPC, and Tay-Sachs disease. Details of the study, including the protocols and inclusion/exclusion, will be made available shortly. Enrollment in the studies are planned to begin in the early fall.

The designation has been posted on the FDA website, you can read it here.

For more information on the research see here.

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