FDA grants orphan designation to Synageva drug
Synageva BioPharma Corp., a Waltham biopharmaceutical company, said it has been granted an orphan drug designation for one of its drug candidates.
The Food and Drug Administration grants orphan drug designations to drug candidates that aim to treat rare diseases. If the drug is ultimately approved for marketing, the drug's maker qualifies for a range of benefit, including seven years of market exclusivity, Synageva's press release noted.
The drug candidate in question is SBC-102, Synageva's enzyme replacement therapy in development to treat Lysosomal Acid Lipase (LAL) Deficiency, also known as Wolman Disease and Cholesteryl Ester Storage Disease, a condition for which there is currently no approved treatment.
People with LAL Deficiency are susceptible to liver problems and hardening of the arteries, according to the company's website.