Ron Cooper, President and CEO of Albireo Pharma, discusses the effect the Orphan Drug Act has had on rare disease pharmaceutical companies.

As Mr. Cooper explains, the Orphan Drug Act of 1983, which facilitated the development of orphan drugs in the United States, provides financial incentives to companies like Albireo. The Orphan Drug Act also provides regulatory and intellectual property assistance, as well as tax credits. Additionally, companies developing orphan drugs are eligible for several years of marketing exclusivity compared to non-orphan drugs. 

Priority Review Vouchers are another incentive issued by the U.S. Food and Drug Administration (FDA) to increase orphan drug development. A company may receive a Priority Review Voucher from the FDA or purchase it from another company. Using a Priority Review Voucher on an FDA submission ensures that a company’s investigational drug will be reviewed by the FDA within six months instead of the standard length of ten months.

Albireo’s orphan drug, odevixibat, has received Rare Pediatric Disease Designation from the FDA and if approved for that pediatric indication, the company will be given a Priority Review Voucher. 

Odevixibat is under investigation to treat progressive familial intrahepatic cholestasis (PFIC), a rare life-threatening liver disease.

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