Date: 2017-10-18
Type of
information: Initiation of development program
phase:
Announcement: initiation of development program
Company: Biomarin Pharmaceutical (USA - CA)
Product: BMN 290
Action
mechanism:
- histone deacetylase inhibitor. Friedreich's Ataxia is a rare autosomal recessive disorder with worldwide prevalence of approximately 15,000, which results in disabling neurologic and cardiac progressive decline. In preclinical models, BMN 290 increases frataxin expression in affected tissues more than two-fold. BMN 290 is a second generation compound derived from a compound acquired from Repligen that had human clinical data demonstrating increases in frataxin in FA patients.
Disease: Friedreich's Ataxia
Therapeutic
area: Rare diseases - Neurodegenerative diseases
Country:
Trial
details:
Latest
news:
- • On October 18, 2017, BioMarin Pharmaceutical provided an update on its development portfolio, which is focused on innovative therapies to treat rare and ultra-rare diseases. The company has selected BMN 290, a selective chromatin modulation therapy, for the treatment of Friedreich's Ataxia. The company expects to submit the IND in 2H 2018.
Is
general: Yes
