BioMarin sees GAAP break-even or better in 2020 assuming Valoctocogene launch
BioMarin Pharmaceutical updated the investment community on the company's research and development portfolio during its R&D day in New York, with CEO JJ Bienaime quoted as having stated: "BioMarin is at an inflection point where our combined understanding of the biology of many rare diseases and key technologies like gene therapy position us not only for exceptional growth over the next decade, but for GAAP profitability in 2020. We are excited about the possibilities of our two late-stage clinical programs in hemophilia A and achondroplasia potentially to transform the course of these conditions. We are also looking forward to leveraging our experience in gene therapy and genetic disease to bring a rich pipeline of new investigational therapies into the clinic." BioMarin noted that the Phase 3 study has now dosed 130 study participants, and that the company completed its process performance qualification campaign, which was the last step in the Chemistry, Manufacturing and Controls package, or CMC package, of the regulatory submissions. BioMarin is on track to submit marketing applications for investigational gene therapy, valoctocogene roxaparvovec, to health authorities in the United States and Europe before year end, the company noted in a press release recapping the event. The company also provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide, in children with achondroplasia, the most common form of disproportionate short stature in humans at its annual R&D day. In addition, BioMarin reaffirmed that it plans to provide top-line data on the randomized, placebo-controlled Phase 3 vosoritide study by the end of the year.