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# Biom Orphan Status for Angelman Syndrome Drug
- URL: https://www.fdaweb.com/biom-orphan-status-for-angelman-syndrome-drug/
- Published: 2021-03-30T12:00:00.000Z
- Updated: 2026-09-14T16:52:42.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149000

FDA has granted Biom Therapeutics an orphan drug designation for BIO017 and its use in treating Angelman syndrome. The company plans to begin a clinical trial involving BIO017 in the fourth quarter.

The company says Angelman syndrome is a subset of epilepsy. *Preclinical results, according to Biom, showed the drug’s ability to reduce seizures as well other behavioral improvements in a mouse model.* The rare genetic neurological disorder is characterized by developmental delays, learning disabilities, and severe speech impairment, it says. Other symptoms include the inability to coordinate voluntary movements, and tremulous, jerky movements of the arms and legs. It also includes a distinct behavioral pattern characterized by a happy disposition and unprovoked episodes of laughter and smiling, often at inappropriate times.