Drug for rare muscular dystrophy fast-tracked

Treatments for extremely rare medical conditions are few and far between. The number of cases of “orphan diseases” doesn’t justify the amount of cash needed to get a pharmaceutical developed, tested and approved.

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This is exactly the niche that Tel Aviv-based BioBlast Pharma was created to fill in 2012. Now its three experimental platforms are moving closer to market. Cabaletta, BioBlast’s lead product for treating two rare and currently untreatable conditions — oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar ataxia type 3 (SCA3) — received Fast Track approval in June from the US Food and Drug Administration (FDA) to expedite the drug’s development, review and potential approval specifically for treating OPMD. Cabaletta is a protein stabilizer that binds to proteins to prevent harmful aggregation. It is undergoing phase 2 trials in Israel and Canada for treating OPMD, and in Israel for treating SCA3. By year’s end, phase 3 trials are anticipated to start for OPMD in North America and for SCA3 in the US and Europe. More...

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