 | |
| Company type | Public |
|---|---|
| Industry | Biotechnology |
| Founded | March 1997; 28 years ago (1997-03) |
| Founders |
|
| Headquarters | San Rafael, California, U.S. |
Key people | |
| Products | |
| Revenue |  US$2.419 billion[2] (2023) |
| US$158.1 million[2] (2023) | |
| US$167.65 million[2] (2023) | |
| Total assets | US$6,841 million[2] (2023) |
| Total equity | US$4,951 million[2] (2023) |
Number of employees | 3,401 (2023) |
| Website | biomarin |
| Footnotes / references [3][4][2] | |
BioMarin Pharmaceutical Inc. is an Americanbiotechnology company headquartered inSan Rafael, California. It has offices and facilities in theUnited States,South America,Asia, andEurope. BioMarin's core business and research are inenzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics formucopolysaccharidosis type I (MPS I), by manufacturing laronidase (Aldurazyme, commercialized byGenzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU).[5][6]
Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials.
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.[7] Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting.[8]
In 2002, BioMarin acquired Glyko Biomedical.[9]
In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of3,4-diaminopyridine (3,4-DAP),amifampridine phosphate.[10] In 2010, BioMarin was granted marketing approval by theEuropean Commission for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate for the treatment of the rare autoimmune diseaseLambert–Eaton myasthenic syndrome (LEMS). BioMarin launched the product under the name Firdapse.[11]
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 availablepoly (ADP-ribose) polymerase (PARP) inhibitor studied for the treatment of patients with rare, genetically defined cancers.[12] 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.[13] At its R&D day in October 2010, BioMarin also announced a new program for a peptide therapeutic,vosoritide (BMN-111), for the treatment ofachondroplasia.[14]
In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan metabolism.[15]
In 2014, BioMarin acquired ahistone deacetylase inhibitorchemical library fromRepligen for $2 million with the intention of advancing work toward therapies forFriedreich's ataxia and other neurological disorders.[16]
In November 2014, the company agreed to the acquisition ofProsensa for up to $840 million;[17] however, the range of treatments forDuchenne muscular dystrophy failed to attain FDA approval, and development ceased in May 2016.[18]
In October 2019 it was revealed that the group will open an office in Dublin to support further growth through Europe, the Middle East and Asia.[19]
The following is an illustration of the company's major mergers and acquisitions and historical predecessors (this is not a comprehensive list):
| BioMarin Pharmaceutical |
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As of 2022, BioMarin has six products on the market, each of which is anorphan drug.[20]
Biomarin is working to develop several new drugs.
In 2010, BioMarin became involved in controversy[22] surrounding3,4-diaminopyridine (3,4-DAP). BioMarin markets a phosphate salt of 3,4-DAP under the nameFirdapse. In 2010, BioMarin was granted exclusive licensing rights to Firdapse for 10 years. As a result, the price of a prescribedNational 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.[23]
In 2013, BioMarin Pharmaceuticals was at the center of a high profile debate regardingexpanded access of cancer patients to experimental drugs.[24] 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 unapprovedPARP inhibitor drug candidate that had exhibited promising activity in a smallPhase 1 clinical trial. The company declined, citing safety concerns.[25] Ms. Sloan eventually received a similar drug candidate from a different company.[26][failed verification]
In 2015, there was another controversy overexpanded 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.[27]
In April 2019, the BBC reported that patients who took part in a trial treatment for the drug Kuvan (sapropterin hydrochloride) were later denied access to it. The company was criticised by the NHS andStephen Hammond MP for patient profiteering. The company commented the following in response: "BioMarin is disappointed that the NHS England has not recognised the value of treating PKU patients with Kuvan, despite more than a decade of positive patient outcomes across 26 countries in Europe, Russia and Turkey"[28]
In June 2019, a Belgian court ordered BioMarin to continue supplying Vimizim to a young girl suffering fromMorquio syndrome free of charge. BioMarin stopped providing free Vimizim at the beginning of the year after negotiations with Belgian health authorities regardingreimbursement of the product repeatedly failed. This caused the parents to startlegal proceedings to force the company to keep providing the medicine free of charge. BioMarin was ordered in apreliminary injunction to keep doing so until a definitive judgment would be rendered, or until the medicine would be available on the Belgian market at a reasonable price.[29]
