You’d Think We’d Care About Rare Diseases

But what we really care about are patients, the families who love them, and the doctors who treat them.

Raptor Pharmaceuticals (NASDAQ: RPTP) is a commercial-stage, global biopharmaceutical company committed to developing and commercializing life-altering therapeutics for orphan diseases. Our focus is to help patients with rare, debilitating, and potentially fatal diseases by leveraging our deep understanding of cellular metabolic pathways to develop medicines that address serious unmet medical needs.

As part of our commitment, we have extensively invested in our portfolio, including our two approved products, PROCYSBI® and QUINSAIR, in order to bring breakthrough therapies to patients in need of new treatment options.

We care about finding a way — a way around, through, and beyond rare diseases. We care about making a difference. And with like-minded investors and world-class employees, we do just that.


Twice-a-day PROCYSBI (cysteamine bitartrate) delayed-release capsules received FDA approval in April 2013 for the management of nephropathic cystinosis in adults and children six years and older and is the first product Raptor Pharmaceuticals, brought to market. The label was expanded for children as young as two in August 2015. In Europe, PROCYSBI gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate) received European Commission approval in September 2013 for the treatment of proven nephropathic cystinosis.

The PROCYSBI molecule, RP103, is currently in development for several additional indications:

Raptor holds U.S. orphan drug designation(s) for RP103 in Huntington’s disease and intellectual property for the use of cysteamine in Huntington’s disease and other neurodegenerative disorders including Parkinson’s disease, Rett Syndrome, and MeCP2-associated disorders. For additional information, please visit the Pipeline page.


The second Raptor product, QUINSAIR, received marketing authorization by the European Commission in March 2015 and Health Canada in June 2015 for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adults with cystic fibrosis. QUINSAIR, a twice-a-day treatment, contains levofloxacin, a proven antimicrobial active against a wide range of gram negative and gram positive bacteria. Raptor plans to launch QUINSAIR in Europe and Canada in the first half of 2016 and to discuss with the FDA the path to approval in the U.S. in 2016.

With activity against a broad spectrum of bacteria, QUINSAIR has the potential for development in additional orphan diseases with unmet need, including:

Preclinical and clinical development pipeline

Raptor has active clinical development programs in multiple therapeutic areas, in addition to those for RP103 and MP-376 noted above.

Convivia® (oral 4-methylpyrazole) is in Phase 2 development for the potential management of acetaldehyde toxicity due to ALDH2 deficiency, an inherited metabolic disorder that affects 40-50 percent of East Asian populations.Raptor has an exclusive license agreement with Uni Pharma Ltd., a Taiwanese pharmaceutical company, to develop and commercialize Convivia in Taiwan.

Our preclinical pipeline includes novel drug candidates to potentially treat cancers and other diseases.

Our pathway is clear: Leadership — with experience and understanding of risk — sets us on the path to achieve our goals. R&D stays focused on the science and evidence that will get us there. This is how we innovate, how we make a difference.