BioMarin mulling price of $2M – 3M for hemophilia gene therapy

In an interview at JPMorgan’s Healthcare Conference, BioMarin Pharmaceutical (BMRN +0.1%) CEO Jean-Jacques Bienaimé said that the company is exploring pricing its hemophilia A gene therapy, valoctocogene roxaparvovec (valrox), at $2M – 3M, a price range apparently acceptable to insurers considering the therapy’s benefits.

In clinical trials, treated patients experienced reductions in bleeding episodes from an average of 16.5 to zero, an effect sustained for three years.

Mr. Bienaimé also said that the company is open to alternative payment schemes such as pay-for-performance, an approach many other biotechs are using for pricey meds.

By comparison, Roche’s Hemlibra (emicizumab-kxwh), a bispecific antibody approved in the U.S. for hemophilia A in October 2018, costs almost $500K per year following the initial dose but is less expensive versus prophylactic treatments.

Barring something unexpected, BioMarin expects the FDA nod sometime in Q3.

#JPM20

https://seekingalpha.com/news/3532487-biomarin-mulling-price-of-2m-3m-for-hemophilia-gene-therapy