New gene therapy may not be ‘one-and-done’ home run

May 29, 2019

BioMarin Pharmaceutical reported this week that the effects of its new gene therapy may diminish over time. 

The treatment, valoctocogene roxaparvovec, was created to help patients with hemophilia A produce needed blood-clotting proteins. The hope is that the infusion, which is being studied as a “one-and-done” cure with a potential price tag over $2 million, could replace the $400,000-per year regimen patients must currently undergo to produce blood-clotting proteins. 

But recent data has shown that the impact of the treatment plateaus after about two years and then becomes less effective over time — but still has some impact for up to eight years. The company said it is now looking into the potential impact of administering a second dose to patients receiving less benefit from the treatment over time. BioMarin also said that further study may show that the therapy lasts longer than eight years.

Read the full Reuters report.