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As the first and only FDA-approved treatment for two life-threatening ultra-rare disorders - paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), Alexion’s Soliris gained many benefits from its first-to-market position and orphan drug status. However, the breakthrough therapy faced challenges as well, most notably payer resistance to the drug's hefty price tag and competition from upcoming biosimilars.
This case study reviews how Alexion achieved blockbuster orphan drug status – recording net product sales worth $2.8 billion in 2016 - through its superior patient support programs, public relations campaigns and commercial expansion strategies. Additionally, the study also reviews how Alexion is preparing to counter biosimilar competition.
Using secondary research, Best Practices, LLC created this case study to highlight successful strategies and tactics for achieving blockbuster status for orphan drugs.
This case study was created using multiple secondary research sources.