BioMarin Announces Presentations at 59th American Society of Hematology Annual Meeting & Exposition

SAN RAFAEL, Calif., Dec. 6, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company will present data in a late breaking abstract session at the 59th American Society of Hematology (ASH) Annual Meeting & Exposition being held December 9-12, 2017 in Atlanta, Georgia. The presentation will report interim results from a Phase 1/2 study of valoctocogene roxaparvovec, an AAV5-FVIII Gene transfer in severe hemophilia.

Listing of Posters and Presentations Related to BioMarin Products and Programs at the 59(th) American Society of Hematology Annual Meeting & Exposition

Oral Presentation - Late Breaking Abstract Session

    Title                                  Authors
    -----                                  -------

    Achievement of Normal Circulating       K. John Pasi, Savita Rangarajan,
     Factor VIII Activity Following BMN     Benjamin Kim, Will Lester,
     270 AAV5-FVIII Gene Transfer:          David Perry, Bella Madan,
     Interim, Long-Term Efficacy and        Fatemeh Tavakkoli, Ke Yang,
     Safety Results from a Phase 1/2        Glenn F. Pierce and Wing Yen
     Study in Patients with Severe          Wong
     Hemophilia A



    Presentation: December 11 at 7:30
     AM
    ---------------------------------

Poster Presentations

    Title                                Authors
    -----                                -------

    Impact of Pre-Existing                Brian Long, Krystal Sandza,
     Immunogenicity to AAV on Vector      Jennifer Holcomb, Juli
     Transduction By BMN 270, an AAV5-    Pherarolis, Lucy Crockett,
     Based Gene Therapy Treatment for     Lillian Falese, Greg Hayes,
     Hemophilia A                         Jeremy Arens, Charles A. O'Neill,
                                          Nancy Pryer, Carlos Fonck,
                                          Stephen Zoog and Christian
                                          Vettermann



    Presentation: December 10 at
     6:00-8:00 PM

    Poster/Presentation: #3332
    --------------------------

    Interim Analysis of Immunogenicity    Brian Long, Benjamin Kim, Wing Yen
     to the Vector Capsid and Transgene-  Wong, Ke Yang, Christian
     Expressed Human FVIII in a Phase-    Vettermann, Nancy Pryer, Romain
     1/2 Clinical Study of BMN 270, an    Hardet, Klaudia Kuranda, Philippe
     AAV5-Mediated Gene Therapy for       Veron, Federico Mingozzi, Glenn
     Hemophilia A                         F. Pierce and Becky Schweighardt



    Presentation: December 11 at
     6:00-8:00 PM

    Poster/Presentation: #4611
    --------------------------

About Hemophilia A

Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs much more frequently in males; incidence is estimated to be 1 in 4,000-5,000 male births. People born with hemophilia produce little or no clotting factors. The two main types of hemophilia are A and B. People with hemophilia A are missing or have low levels of clotting factor VIII.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com.

    Contact:


    Investors:                   Media:

    Traci McCarty                Debra Charlesworth

    BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.

    (415) 455-7558               (415) 455-7451

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SOURCE BioMarin Pharmaceutical Inc.