The company presents development portfolio to investors as key candidate advances to Phase III trials.

In a presentation to investors on October 18, BioMarin Pharmaceutical Inc. updated the community on the company’s gene-therapy development portfolio. Recognized as one of the country’s most innovative companies by Forbes’ Magazine in 2017, BioMarin offeredfresh insight into their rare genetic disease product portfolio which includes BMN 290 for fighting Friedreich’s Ataxia, Vosoritide for achondroplasia (dwarfism) in children, pegvaliase to treat adults with uncontrolled PKU , and an extremely promising gene therapy to treat hemophilia A, called BMN 270, soon to enter Phase III trials.

The company also announced that the FDA has finished their review of the IND for BMN 270. To support production of the therapy, BioMarin has constructed one of the largest gene therapy manufacturing facilities in the world, located in Novato, California. The facility can support approximately 2,000 patients per year.

"We are pleased to share the progress of our development programs in therapies to treat rare genetic diseases; hemophilia A, PKU, achondroplasia and our next IND into Friedreich's Ataxia," said Hank Fuchs, President Worldwide Research and Development of BioMarin. "In the near term, we are expecting an FDA decision on pegvaliase to treat adults with uncontrolled PKU in the first half of next year, and we continue to be rapidly and decisively developing the potential first gene therapy for severe hemophilia A."

BioMarin’s BMN 290, is a selective chromatin modulation therapy to treat Friedreich's Ataxia (FA), a rare disorder that leads to the progressive decline of the heart and neurologic system. According to the company, which expects to submit the IND in 2H 2018, there currently are no approved disease-modifying therapies for FA. “In preclinical models, BMN 290 increases frataxin expression in affected tissues more than two-fold,” said BioMarin. BMN 290, explained the company, “is a second-generation compound derived from a compound acquired from Repligen that had human clinical data demonstrating increases in frataxin in FA patients and selected for its ability to penetrate the central nervous system and cardiac target tissues, and its preservation of the selectivity of the original Repligen compound.”