Pharma BD Deal Intelligence
Horizon Pharma's ~$800M acquisition of Raptor Pharmaceutical for Procysbi and Quinsair was a mixed bag: Procysbi endured as a durable orphan-drug contributor, but Horizon sold EMEA rights to both drugs to Chiesi for just $70M within eight months, and Quinsair's remaining sales collapsed roughly 90% after that carve-out.
One durable niche orphan drug (Procysbi) justified a fraction of the price; the rest of what was paid for (Quinsair, Raptor's pipeline) evaporated within a year or two of close.
Full analysis, sources & comparables →Fierce Biotech flagged that while the deal expanded Horizon's rare-disease franchise, Raptor's pipeline had suffered repeated clinical failures (notably the…
Pharmacy Times framed the deal as accelerating Horizon's transformation into a rare-disease specialist, with Procysbi adding a long-tail patent-protected…
The patient foundation that funded early Procysbi research criticized post-acquisition pricing — the drug launched at ~$300K/yr in 2013 and reached >$1M/yr for…
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Horizon Pharma agreed to acquire Raptor Pharmaceutical for $9.00/share cash (~$800M equity, ~$860.8M total including debt repayment). Adds PROCYSBI (cysteamine for nephropathic cystinosis) and QUINSAIR (inhaled levofloxacin for CF, EU only) to Horizon's rare-disease portfolio. Closed October 25, 2016.
One durable niche orphan drug (Procysbi) justified a fraction of the price; the rest of what was paid for (Quinsair, Raptor's pipeline) evaporated within a year or two of close.
Assessment window: 5yr post-close.
15 US cases/yr · $300M Procysbi peak sales projection (Zacks/analyst-cited)
Nephropathic cystinosis is an ultra-rare autosomal recessive lysosomal storage disorder caused by CTNS gene mutations that prevent cystine export from lysosomes. Cystine crystals accumulate in kidneys (causing Fanconi syndrome and progressive renal failure), eyes, thyroid, muscle, and CNS, typically presenting in infancy. Cysteamine depletes lysosomal cystine and is the only disease-modifying therapy.
The cystinosis therapeutic market at deal time was effectively a two-product duopoly within the same molecule class. Mylan's Cystagon (immediate-release cysteamine bitartrate, dosed every 6 hours including overnight) was the legacy generic standard but suffered from severe adherence problems due to its punishing dosing schedule and GI tolerability. Raptor's Procysbi (delayed-release cysteamine bitartrate, BID dosing) was approved by the FDA in April 2013 and EMA in 2013 — a reformulation rather than a novel mechanism, but one with patent protection through 2034 and clinical evidence of better compliance and cystine control. Quinsair (inhaled levofloxacin) added a CF lung-infection asset competing against Vertex-pivot drugs and inhaled tobramycin (TOBI/Bethkis) and aztreonam (Cayston, Gilead) in a market that was rapidly being remapped by Vertex's CFTR modulators (Kalydeco, Orkambi). The Horizon acquisition was less about competitive disruption and more about portfolio assembly — adding a high-priced orphan annuity (Procysbi at $300K-$1M+/yr) to a rare-disease platform that had been building through Hyperion (urea-cycle) and Crealta (Krystexxa). The transaction drew patient-advocacy criticism over Procysbi pricing escalation post-acquisition.
| Deal | Year | Value | Outcome |
|---|---|---|---|
| Horizon Pharma plc / Raptor Pharmaceutical Corp. (this deal) | 2016 | $860M | 46 |
| Horizon Pharma plc / Crealta Holdings LLC | 2015 | $510M | 87 |
| Horizon Pharma plc / Hyperion Therapeutics, Inc. | 2015 | $1.1B | 79 |
| Pfizer Inc. / BioNTech SE | 2020 | $748M | 100 |
| Gilead Sciences Inc. / Pharmasset Inc. | 2011 | $11.2B | 98 |
| Abbott Laboratories / Alere Inc. | 2016 | $5.8B | 95 |
| bioMerieux SA / BioFire Diagnostics Inc. | 2013 | $485M | 88 |
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