Alexion Pharmaceuticals

Building Support for an Orphan Drug

It was a make-or-break moment for a small company on the brink of approval for its first biotech drug after a 15-year, $800 million investment in research.

In early 2007, Alexion was awaiting FDA clearance of Soliris™ (eculizumab), the first and only treatment for paroxsysmal nocturnal hemoglobinuria (PNH), a very rare illness affecting fewer than 10,000 patients worldwide.

Just two months before the anticipated approval and launch, Alexion tapped Makovsky to lead what became the most successful ultra-orphan drug launch in history, generating 500 million media impressions over nine months. In just three months, Alexion’s valuation skyrocketed 80%.

The best reward? The impact of Soliris on patient lives. One USA Today cover story, which detailed one patient’s struggle with PNH and miracle recovery with Soliris, convinced another patient’s insurer to cover the cost of treatment with Soliris.