Shares of Sage Therapeutics Inc jumped 5% in premarket trading following the announcement that the US Food and Drug Administration (FDA) has granted orphan-drug status to SAGE-718, a treatment for Huntington's disease.
The Cambridge, Massachusetts-based biopharmaceutical firm revealed that it is progressing with phase II clinical trials for SAGE-718, involving placebo-controlled studies in various disease domains, including its primary focus on cognitive impairment linked to Huntington's disease.
The FDA's orphan-drug designation (ODD) is a special classification that benefits medications and biologics targeting conditions that impact fewer than 200,000 individuals in the United States.
This status offers extended marketing exclusivity, protecting the drug from direct competition for a longer period. It's worth noting that the FDA had earlier fast-tracked SAGE-718 specifically for treating Huntington's disease, further underscoring the treatment's potential.
“Huntington’s disease is a devastating condition that often affects patients in their prime years, and it can significantly impact a patient’s ability to live independently," said Laura Gault, Sage's chief medical officer.
"Cognitive impairment is one of the most under-recognized aspects of this disease.
“There are currently no approved treatments to address cognitive impairment for people with HD and a growing sense of urgency among researchers and people living with HD to address cognitive impairment early so that patients can maintain independence longer.
"The ODD designation from the FDA provides continued momentum in our efforts to help patients and their families impacted by this aspect of HD.”
Ahead of the bell, the stock was changing hands for $20.53, up 89 cents.