FDA Breakthrough Therapy Designation: 27 And Counting
On November 25, Protola Pharmaceuticals announces that the FDA grants the coveted Breakthrough Therapy Designation (BTD) for Andexanet Alfa (PRT4445), an investigational (first-in-class agent) Factor Xa inhibitor antidote. This is the 27th BTD that is announced by a sponsor company.
Portola Pharmaceuticals, a San Francisco-based biopharmaceutical company, focuses on the development and commercialization of therapies for thrombosis (blood clots), other hematologic disorders, and inflammation. The company is founded in 2003 and completes an Initial Public Offering (IPO) in May 2013. The company’s two main programs are in the area of thrombosis :
• Betrixaban – once-daily inhibitor of Factor Xa in Phase III trials
• Andexanet Alfa (PRT4445) – recombinant Factor Xa inhibitor antidote that reverses anticoagulant activity in patients treated with a Factor Xa inhibitor who experience uncontrolled bleeding.
Portola Pharmaceuticals plans on following the FDA’s Accelerated Approval route for Andexanet Alfa, with the start of registration-enabling studies in 2014. The company estimates that by 2020 :
“the number of patients presenting to the hospital who could benefit from an antidote could approach ½ million in the United States, Japan, and the 5 largest EU countries alone.”
Portola Pharmaceuticals forms clinical collaboration agreements with all of the following manufacturers of direct Factor Xa inhibitors:
• Bristol-Myers Squibb & Pfizer – Eliquis (Apixaban)
• Bayer HealthCare and Janssen Pharmaceuticals – Xarelto (Rivaroxaban)
• Daiichi Sankyo – Lixiana (Edoxaban).
The media, investors, and other stakeholders are still debating about the worth and importance of the FDA’s BTD. Since the first BTD is announced in January 2013 by Vertex Pharmaceuticals for its orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis :
• 34 BTDs (as of 11/22/13) are awarded to sponsor companies
• 2 BTDs (as of 11/22/13) receive FDA approval.
Please Note: FDA Official Logo from FDA website.