[td_text_with_title custom_title=”Company description”]BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. Approved products include Vimizim® (elosulfase alfa) for MPS IVA, a product wholly developed and commercialized by BioMarin; Naglazyme® (galsulfase) for MPS VI, a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for MPS I, a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution and Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include drisapersen, an exon skipping oligonucleotide, for which a marketing application has been submitted to FDA and EMA for the treatment of patients with Duchenne muscular dystrophy (DMD) with mutations in the dystrophin gene that are amenable to treatment with exon 51 skipping, pegvaliase (formerly referred to as BMN 165 or PEG PAL), PEGylated recombinant phenylalanine ammonia lyase, which is currently in Phase 3 clinical development for the treatment of PKU, talazoparib (formerly referred to as BMN 673), a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, reveglucosidase alfa (formerly referred to as BMN 701), a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase 3 clinical development for the treatment of Pompe disease, vosoritide (formerly referred to as BMN 111), a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia, BMN 044, BMN 045 and BMN 053, exon skipping oligonucleotides, which are currently in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (exons 44, 45 and 53), cerliponase alfa (formerly referred to as BMN 190), a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of CLN2 disease, a form of Batten disease, which is currently in Phase 1, BMN 270, an AAV-factor VIII vector, for the treatment of hemophilia A and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of MPS IIIB.[/td_text_with_title]


    Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering [...]
    [td_text_with_title custom_title=”Latest post”]

    Biomarin: FDA Accepts Drisapersen NDA

    SAN RAFAEL, Calif., June 29, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee […]


    Prosensa Update about Drisapersen

    [/td_text_with_title][td_text_with_title custom_title=”From Wikipedia”]

    BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase (Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU).


    BioMarin was founded in 1997 by Christopher Starr Ph.D. and Grant W. Denison Jr. with an investment of a $1.5 million from Glyko Biomedical and went public in 1999.[2][3] Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting.[4]

    Business development

    In 2002, BioMarin acquired Glyko Biomedical.[5]

    In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate.[6] In 2010, BioMarin was granted marketing approval by the European Commission for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate for the treatment of the rare autoimmune disease Lambert-Eaton myasthenic syndrome (LEMS). BioMarin launched the product under the name Firdapse.[7]

    In 2010, BioMarin acquired LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor studied for the treatment of patients with rare, genetically defined cancers.[8] This acquisition was followed by the purchase of ZyStor Therapeutics, Inc. (ZyStor), a privately held biotechnology company developing ERTs for the treatment of lysosomal storage disorders and its lead product candidate, ZC-701, a fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.[9] At its R&D day in October 2010, BioMarin also announced a new program for a peptide therapeutic, vosoritide (BMN-111), for the treatment of achondroplasia.[10]

    In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan metabolism.[11]

    In 2014, BioMarin acquired a histone deacetylase inhibitor chemical library from Repligen for $2 million with the intention of advancing work toward therapies for Friedreich's ataxia and other neurological disorders.[12]

    In November 2014, the company agreed the acquisition of Prosensa for up to $840 million,[13] the range of treatments for Duchenne Muscular Dystrophy however failed to attain FDA approval and in May 2016 development ceased.[14]

    Acquisition history

    The following is an illustration of the company's major mergers and acquisitions and historical predecessors (this is not a comprehensive list):

    BioMarin Pharmaceutical

    (Acq 2014)


    Zacharon Pharmaceuticals
    (Acq 2012)


    ZyStor Therapeutics, Inc.
    (Acq 2010)


    LEAD Therapeutics, Inc.
    (Acq 2010)


    Huxley Pharmaceuticals, Inc.
    (Acq 2009)


    Glyko Biomedical
    (Acq 2002)


    As of 2016, BioMarin has six products on the market, each of which is an orphan drug.[15]


    In 2010, BioMarin became involved in controversy[17][18] surrounding 3,4-diaminopyridine (3,4-DAP). BioMarin markets a phosphate salt of 3,4-DAP under the name Firdapse. In 2010, BioMarin was granted exclusive licensing rights to Firdapse for 10 years. As a result, the price of a prescribed National Health Service treatment course has increased from $1,987 for the unlicensed drug to $69,970 for Firdapse. The company states that prior to its licensing, there was no guaranteed quality control of the product and no way of formally monitoring for uncommon side effects through the regulatory process.[19]

    In 2013, BioMarin Pharmaceuticals was at the center of a high profile debate regarding expanded access of cancer patients to experimental drugs.[20] On the advice of her doctor, Andrea Sloan, a patient with advanced ovarian cancer, requested that the company provide her with access to BMN 673, an unapproved PARP inhibitor drug candidate that had exhibited promising activity in a small Phase 1 clinical trial. The company declined, citing safety concerns.[21] Ms. Sloan eventually received a similar drug candidate from a different company.[22]

    In 2015, there was another controversy over expanded access, concerning the supply of a drug on clinical trial to a German child who was suffering from a brain disorder but who was not part of the trial.[23]


    Use YYYY-MM-DD format for publication dates

    1. ^ "US SEC: Form 10-K BioMarin Pharmaceutical Inc". U.S. Securities and Exchange Commission. Retrieved March 31, 2018. 
    2. ^ "BioMarin". Life Sciences at CHF. Retrieved 2016-07-09. 
    3. ^ "History". Biomarin. Retrieved 2016-07-09. [self-published source]
    4. ^ "Form S-1 Registration Statement". Biomarin Pharmaceuticals Inc. Retrieved 15 December 2015. 
    5. ^ "BioMarin Pharmaceutical Acquires Glyko Biomedical". Deals. LEXPERT. Thomson Reuters. January 2003. Archived from the original on 2013-12-28. 
    6. ^ Stevens, Loralee (2009-10-27). "BioMarin Acquires Huxley Pharmaceuticals". North Bay Business Journal. Santa Rosa, California, United States. Archived from the original on 2011-01-04. Retrieved 2014-02-18. 
    7. ^ "BioMarin launches LEMS drug Firdapse in European Union; and Genmab gets conditional approval for Arzerra". The Pharma Letter. 2010-04-20. Retrieved 2014-02-18. (subscription required)
    8. ^ Brown, Steven E.F. (2010-02-04). "BioMarin to buy Lead Therapeutics for $18M". San Francisco Business Times. Archived from the original on 2011-06-29. Retrieved 2014-02-18. 
    9. ^ Leuty, Ron (2010-08-17). "BioMarin to buy ZyStor for up to $115M". San Francisco Business Times. Archived from the original on 2011-06-29. Retrieved 2014-02-18. 
    10. ^ Andersen, Karen (2010-10-21). "BioMarin Highlights Diverse Early-Stage Pipeline at R&D Day". Morningstar. Retrieved 2011-02-14. 
    11. ^ Brown, Steven (2013-01-07). "BioMarin Pharmaceutical buys Zacharon for $10 million". San Francisco Business Times. Retrieved 2013-01-07. 
    12. ^ "BioMarin Nabs Repligen's HDACi Compound Library". News: Discovery & Development. Gen. Eng. Biotechnol. News (paper). 34 (4). 15 February 2014. p. 14. 
    13. ^
    14. ^ "BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe (NASDAQ:BMRN)". Retrieved 2016-07-04. 
    15. ^ BioMarin Office Site: Products
    16. ^ Pollack, A (2007-12-14). "Agency Approves Drug to Treat Genetic Disorder That Can Lead to Retardation". The New York Times. Retrieved 2008-04-03. 
    17. ^ Goldberg, Adrian (2010-11-21). "Drug firms accused of exploiting loophole for profit". BBC News. Retrieved 2010-11-21. 
    18. ^ Martin, Daniel (2010-09-27). "Hospitals are forced to use unlicensed medicines to save millions". Daily Mail. London. Retrieved 2010-11-21. 
    19. ^ "What makes an orphan drug?". British Medical Journal. 2010-11-10. Retrieved 2010-11-21. 
    20. ^ "Andrea Sloan Faces Pharma Firm With History Of Indifference". Huffington Post. 2013-09-26. Retrieved 2013-12-24. 
    21. ^ "In cancer drug battle, both sides appeal to ethics". CNN. 2013-09-20. Retrieved 2013-12-24. 
    22. ^ Ed Silverman for Pharmalot. October 4, 2013 Andrea Sloan, 'Poster Child' For Compassionate Use, Gets A Drug
    23. ^ "Hannahs Kampf gegen Pharmafirma". B5 aktuell. 2015-05-13. Retrieved 2015-05-13.