Beren Therapeutics' Drug Under FDA Review After Positive Long-Term Study
1 report, 1 independent
Updated Oct 15
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What happened
Beren Therapeutics announced new long-term findings for adrabetadex, an investigational cyclodextrin-based medicine for infantile-onset Niemann-Pick disease, type C. The drug is currently under review by the U.S. Food and Drug Administration. A case study presented at the Child Neurology Society Annual Meeting showed that treatment preserved neurological function for more than 10 years and was associated with improved survival and slower disease progression.
Why it matters
The positive long-term results regarding adrabetadex could influence the U.S. Food and Drug Administration's regulatory decision on the medicine.
Who's involved
- FDARegulatory agency reviewing the investigational medicine