Raptor Pharmaceutical Corp. will launch an ultra-Orphan drug this quarter that it has shown to be non-inferior to a cheaper alternative. The company has yet to get any payers on board, but believes it can win them over based on the drug's ability to improve compliance and quality of life for patients with nephropathic cystinosis.

Raptor has set the U.S. list price of Procysbi (RP103) at $250,000 annually. The immediate-release formulation of the drug, Cystagon cysteamine, is marketed by Mylan Inc. for about $10,000 per year.