ProductPROCYSBI

PROCYSBI^® is an orphan drug for the management of nephropathic cystinosis in adults and children 6 years and older. Nephropathic cystinosis is the most common form of cystinosis, a rare, inherited condition that affects every cell of the body by causing the buildup of a protein building block called cystine. PROCYSBI^® is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic levels of cystine in the cells. It offers patients a more tolerable cystinosis treatment with a less burdensome dosing schedule.

Background

Prior to its PDUFA date, Raptor was seeking non-dilutive financing to fund the launch of its lead product, PROCYSBI^®(RP-103), for the treatment of cystinosis. HCR provided $25 million prior to approval to fund the commercial buildout and then an additional $25 million coincident with the regulatory approval of PROCYSBI^®. This financing provided Raptor with sufficient capital to fund the U.S. and E.U. launches of PROCYSBI^® and avoided dilution to existing equity investors. Additionally, when Raptor required additional financing a few years later, HCR followed on with an additional $70 million financing. Raptor was acquired by Horizon Pharmaceuticals in 2016.

Interview with Julie Anne Smith, Former President and CEO, Raptor Pharmaceuticals