- Alnylam Pharmaceuticals is developing an alternate therapy for paroxysmal nocturnal hemoglobinuria (PNH) to compete with Alexion Pharma's treatment for the rare disease. Alexion's drug, Soliris, is the only approved medication for PNH.
- Currently, Soliris is considered the most expensive drug in the world at a hefty price tag of $440,000 a year.
- Alnylam's CEO, John Maraganore, plans to start final trials of ALN-CC5 in 2017.
Given the current climate and lack of tolerance for specialty drugs with seemingly arbitrary high price points, Maraganore's strategy to develop a lower priced PNH drug appears sound.
Clinically, however, Alnylam will have to prove its new drug will be as or more effective than Soliris, which has been on the market since its FDA approval in 2007. Head-to-head trials could be needed, according to analyst Ritu Baral, speaking to Bloomberg.
Bringing a new lower-priced drug into a a high-priced specialty market has proven successful. Hepatitis C treatments are a perfect example, with AbbVie and Gilead competing on price as well as efficacy.
Maragnore is confident in his drug as well as his strategy. In addition to taking on Alexion, Alnylam plans to challenge other companies working on rare disease treatments. It aims have five late-stage clinical trials for rare diseases by the end of 2017.