Soliris: Translating Groundbreaking Science into a Successful Orphan Drug

STUDY OVERVIEW

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.

KEY TOPICS

• Soliris’ Trajectory to Achieving Blockbuster Status
• Benefits Availed by Alexion Owing to Soliris’ Orphan Drug Status
• Commercialization Strategies
• Alexion Uses Patient Support Programs
• Challenges Faced by Alexion’s Soliris
• Way Forward

KEY METRICS

• Soliris’ efficacy rate in PNH and aHUS
• Soliris’ net product sales from 2007 to 2016

METHODOLOGY

This case study was created using multiple secondary research sources.

If you purchase Best Practice Database document(s), you will have 30 days from the date of purchase to apply some or all of the cost of the document(s) toward the cost of a Full Access Individual, Pharma, Group or University Membership. Write us at DatabaseTeam@bestpracticesllc.com or call David Guinn at 919-767-9179 if you have any questions.