- Global Blood Therapeutics ( NASDAQ: GBT ) on Thursday said it had started an expanded access program (EAP) for its sickle cell disease treatment voxelotor in Brazil.
- An EAP allows medicines and drugs to be available before approval to treat a serious or life-threatening disease in patients with no alternative treatment option.
- Under the EAP, voxelotor will be available for the treatment of sickle cell disease in adults and children aged 12 years and older in Brazil. The drug will be supplied free of charge.
- The Brazilian drug regulator, ANVISA, has approved the EAP.
- "There are up to (100K) people estimated to have sickle cell disease in Brazil, yet there are no currently approved treatments that directly address the underlying cause of the disease," said GBT's executive VP and head of R&D Kim Smith-Whitley.
- GBT stock -1.5% at $34.81 in morning trading.
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Global Blood Therapeutics to expand access to its sickle cell disease treatment in Brazil