Despite bumetanide failure, promise remains in autism market, says analyst

French privately-held pharma major Servier and Neurochlore have recently announced that, following a lack of efficacy in two Phase III trials of bumetanide for autism spectrum disorder (ASD) in adolescents, they would be terminating these trials early.

These results are particularly disappointing given the promise bumetanide had shown in an earlier Phase IIb trial, and because there is a large unmet need for treatments for the core symptoms of ASD, says GlobalData. The data and analytics company notes that currently, the only FDA-approved drugs for autism are the atypical antipsychotics, which aim to treat the associated symptom of irritability.

The large unmet need for a treatment for ASD is reflected in a diverse pipeline, with numerous companies recognizing the market potential for such a product. GlobalData’s proprietary database shows that the autism pipeline consists of 47 drugs spanning all stages of development, including two drugs in Phase III, five drugs in Phase II, and five drugs in Phase I development.