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The FDA has given a rare disease an "orphan drug" designation, making it easier for a UK biotech company to get its drug approved in the US.
SiSaf says its drug, SIS-101-ADO, is the first treatment for Autosomal Dominant Osteopetrosis Type 2, or ADO2, a rare genetic disorder that causes a buildup of osteoclasts in children's bones, Bloomberg reports.
If approved, SIS-101-ADO would be the first treatment for the disorder, which has no approved treatments, and "could provide life-altering benefits for those who suffer from this debilitating disease," the company says.
The FDA's orphan drug designation provides incentives such as tax credits for clinical trials, exemption from user fees, and expanded marketplace exclusivity.
"Being granted Orphan Drug Designation and Rare Pediatric Disease Designation is a major milestone in our drive to move our revolutionary treatment forward to alleviate the pain and suffering that ADO2 inflicts," CEO Suzanne Saffie-Siebert says in a statement.
The company is currently preparing for first-in-human clinical trials.
SIS-101-ADO combines an siRNA that suppresses the expression of CLCN7, a mutant gene expressed by osteoclasts and other cell types responsible for causing ADO
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