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Procysbi: Major Addition to Horizon’s Orphan Drug Portfolio

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Procysbi holds potential of more than $300 million in peak sales

Raptor Pharmaceuticals’ drug Procysbi had global net sales of $94.2 million in fiscal 2015. According to Raptor’s guidance, the drug’s global net sales should amount to $122 million in fiscal 2016. In 2016, Raptor expects to generate total net sales of $125 million–$135 million. This includes net sales of Procysbi and Quinsair.

According to Horizon Pharma’s (HZNP) estimates, Procysbi holds a peak sales potential of more than $300 million. In the next part, we’ll see how Procysbi is in line with Horizon’s existing rare disease portfolio.

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What are Procysbi’s revenue drivers?

Procysbi is a second-generation medicine. The first-generation therapy requires six-hour dosing, while the second-generation therapy has a 12-hour dosing. The transition of patients from first-generation medicine to Procysbi increased penetration in early onset patients. Efforts to expand the population base is one of the drivers for the drug in the United States. Additional country launches and an early access program for the drug in selected countries should drive the drug’s non-US sales.

How Procysbi helps cure NC

If left untreated, nephropathic cystinosis (or NC) causes irreversible cellular damage that leads to progressive multi-organ failure. This ultimately results in a patient’s death. When NC is treated with Procysbi, irreversible cellular damage can be prevented.

Horizon’s expansion in the orphan disease space might expand its margins. BioMarin Pharmaceutical (BMRN), Alexion Pharmaceuticals (ALXN), and Vertex Pharmaceuticals (VRTX) operate in the high-margin rare disease space.

The above expectations of Horizon Pharma might result in a rise in its share price. If you want exposure to aggressively growing Horizon, you can invest in the iShares Nasdaq Biotechnology ETF (IBB). IBB holds 0.54% of its assets in Horizon.

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