“Breakthrough Therapy” status for Portola’s andexanet alfa

USA-based Portola Pharmaceuticals (Nasdaq: PTLA) has been granted “Breakthrough Therapy” designation by the US Food and Drug Administration for andexanet alfa, its investigational Factor Xa inhibitor antidote.

The FDA designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. Portola is pursuing an accelerated approval pathway for andexanet alfa, a first-in-class agent, and plans to initiate registration-enabling studies in 2014. By the year 2020, Portola estimates that the number of patients presenting to the hospital who could benefit from an antidote could approach 500,000 in the USA, Japan and the five largest European Union countries alone.