BioMarin Pharmaceutical
| Company type | Public |
|---|---|
| Industry | Biotechnology |
| Founded | 1997 |
| Founder | Christopher Starr Ph.D., Grant W. Denison Jr. |
| Headquarters | San Rafael, California , United States |
Area served | United States, South America, Asia, Europe |
| Products | Enzyme replacement therapies including Aldurazyme, Kuvan, Naglazyme, Firdapse, Vimizim, Brineura, Roctavian |
BioMarin Pharmaceutical Inc. is a biotechnology firm based in San Rafael, California, United States, with a presence spanning the United States, South America, Asia, and Europe. The company is recognized for its focus on enzyme replacement therapies (ERTs), developing innovative treatments for rare genetic conditions such as mucopolysaccharidosis type I (MPS I) using laronidase (Aldurazyme, marketed by Genzyme Corporation) and phenylketonuria (PKU).
The core of BioMarin's corporate strategy lies in creating breakthrough therapies for uncommon diseases, utilizing its international network and subsidiaries to broaden its market footprint. While the company has demonstrated significant growth and reported revenues of US$2.419 billion in 2023, it has also encountered scrutiny regarding its drug pricing strategies and limitations on patient access during clinical trials.
History
Established in 1997 by Christopher Starr Ph.D. and Grant W. Denison Jr., BioMarin began with a $1.5 million funding round from Glyko Biomedical and went public two years later in 1999. Early investors included MPM Bioventures, Grosvenor Fund, and Florian Schönharting.
Business development
BioMarin expanded its capabilities through several acquisitions over the years. In 2002, it purchased Glyko Biomedical. The acquisition of Huxley Pharmaceuticals came in 2009, bringing rights to amifampridine phosphate (3,4-DAP). By 2010, BioMarin had obtained European approval for 3,4-DAP (Firdapse) to treat Lambert–Eaton myasthenic syndrome (LEMS).
That same year, the company acquired LEAD Therapeutics, adding LT-673, a PARP inhibitor targeting rare cancers, to its portfolio. It also acquired ZyStor Therapeutics to advance enzyme replacement therapies addressing lysosomal storage diseases, including Pompe disease. During its 2010 R&D day, BioMarin unveiled progress on vosoritide (BMN-111), a peptide drug aimed at treating achondroplasia.
In 2012, BioMarin incorporated Zacharon Pharmaceuticals, which specializes in glycan metabolism pathways. Two years later, it acquired a chemical library of histone deacetylase inhibitors from Repligen to support development of treatments for Friedreich's ataxia and other neurological conditions.
Also in 2014, BioMarin agreed to acquire Prosensa for up to $840 million; however, after the failure of Duchenne muscular dystrophy therapies to gain FDA approval, this program was discontinued in 2016.
In October 2019, BioMarin established an office in Dublin to bolster its operations across Europe, the Middle East, and Asia.
Acquisition history
Key acquisitions contributing to BioMarin’s expansion include Prosensa (2014), Zacharon Pharmaceuticals (2012), ZyStor Therapeutics and LEAD Therapeutics (both in 2010), Huxley Pharmaceuticals (2009), and Glyko Biomedical (2002), underscoring a strategic approach centered on selective mergers and acquisitions.
Products
By 2022, BioMarin’s portfolio comprised six orphan drugs:
- Kuvan (sapropterin dihydrochloride), launched in 2007 as the first pharmacological treatment for phenylketonuria.
- Naglazyme (galsulfase), a recombinant enzyme therapy for Maroteaux–Lamy syndrome (mucopolysaccharidosis VI).
- Aldurazyme, a recombinant enzyme used in managing mucopolysaccharidosis I.
- Firdapse (amifampridine), a small molecule approved within the European Union for treating Lambert–Eaton myasthenic syndrome.
- Vimizim (elosulfase alfa), the sole enzyme replacement therapy available for Morquio A Syndrome (MPS IVA), a rare metabolic disorder affecting roughly 3,000 patients in developed countries.
- Brineura (cerliponase alfa), an enzyme replacement treatment approved in 2017 for Batten disease.
- Roctavian (valoctocogene roxaparvovec), an adeno-associated viral vector gene therapy for hemophilia A authorized in the EU as of August 2022.
BioMarin maintains substantial investment in research and development to continually broaden its pipeline of innovative therapeutic solutions.
Controversies
BioMarin has encountered multiple disputes that have affected its standing in the market and interactions with regulatory bodies. In 2010, after securing exclusive rights for a decade to 3,4-diaminopyridine phosphate salt (Firdapse), the medication's price escalated dramatically from $1,987 to $69,970 per treatment cycle, igniting discussions regarding pricing practices for orphan drugs. The firm justified this increase by referencing enhancements in quality assurance and compliance with regulatory standards.
In 2013, BioMarin refused requests for expanded access to BMN 673, an investigational PARP inhibitor used in cancer treatment, citing concerns about patient safety. This refusal drew public attention amid ongoing debates about compassionate use protocols.
Additional disputes over expanded access emerged in 2015 when supply complications arose involving a clinical trial drug intended for a patient outside the trial suffering from a neurological condition.
In 2019, criticism came from NHS England and UK Member of Parliament Stephen Hammond after patients involved in Kuvan clinical trials were denied continued drug access following the conclusion of the studies. BioMarin responded by emphasizing more than ten years of favorable patient outcomes across numerous countries to support its stance.
Also in 2019, a Belgian court mandated that BioMarin must keep supplying Vimizim at no cost to a young patient with Morquio syndrome after reimbursement discussions with Belgian health authorities reached an impasse. This legal ruling highlighted difficulties related to pricing and market entry within European healthcare frameworks.