BioMarin Pharmaceutical Inc. (BioMarin) operates as a global biotechnology company.
Recent Developments
In the first half of 2024, the company focused on value creation through working to accelerate growth, optimize efficiencies and drive operational excellence, including progress in executing on key strategic priorities first outlined in January 2024. The company also completed a strategic portfolio assessment of research and development programs to determine which have the strongest combinat...
BioMarin Pharmaceutical Inc. (BioMarin) operates as a global biotechnology company.
Recent Developments
In the first half of 2024, the company focused on value creation through working to accelerate growth, optimize efficiencies and drive operational excellence, including progress in executing on key strategic priorities first outlined in January 2024. The company also completed a strategic portfolio assessment of research and development programs to determine which have the strongest combination of scientific merit, opportunity for commercial success and potential value creation for stockholders. In September 2024, the company held an Investor Day, during which it provided an overview of its new corporate strategy focused on innovation, growth, and value commitment. The company’s new strategy includes, among other things, its plans to expand VOXZOGO for the treatment of conditions beyond achondroplasia, its initiatives to drive sustained growth of the Enzyme Therapies portfolio (ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM), and the company’s decision to focus on the United States (U.S.), Germany and Italy with respect to ROCTAVIAN. The company also announced its updated commercial organizational model, which starting in 2025, is structured around three business units: Skeletal Conditions, Enzyme Therapies and ROCTAVIAN.
Commercial Products
VIMIZIM
VIMIZIM is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. MPS IVA is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS) causing excessive lysosomal storage of certain complex carbohydrates known as glycosaminoglycans (GAGs), such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected. VIMIZIM is approved for marketing in the U.S., the European Union (EU) and other international markets.
VOXZOGO
VOXZOGO is a once daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of disproportionate short stature in humans. In patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). VOXZOGO acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
VOXZOGO is approved for marketing in the U.S. and Japan for the treatment of achondroplasia in children with open growth plates of all ages, in the EU for the treatment of children with open growth plates aged four months and older, and in other markets, including Australia and Brazil, for patients in various age ranges.
The company continues to research VOXZOGO’s safety and effectiveness in children with achondroplasia while also advancing development across its CANOPY clinical program with VOXZOGO for the treatment of conditions beyond achondroplasia, including hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.
NAGLAZYME
NAGLAZYME is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) indicated for patients with MPS VI. MPS VI is a debilitating life-threatening genetic disease for which no other drug treatment exists and is caused by the deficiency of arylsulfatase B, an enzyme normally required for the breakdown of GAGs. Patients with MPS VI typically become progressively worse and experience multiple severe and debilitating symptoms resulting from the build-up of carbohydrate residues in tissues in the body. These symptoms include inhibited growth, spinal cord compression, enlarged liver and spleen, joint deformities and reduced range of motion, skeletal deformities, impaired cardiovascular function, upper airway obstruction, reduced pulmonary function, frequent ear and lung infections, impaired hearing and vision, sleep apnea, malaise and reduced endurance.
NAGLAZYME is approved for marketing in the U.S., the EU and other international markets.
PALYNZIQ
PALYNZIQ is a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations. PALYNZIQ is the company’s second approved treatment for PKU. PKU is caused by a deficiency of activity of an enzyme, phenylalanine hydroxylase (PAH), which is required for the metabolism of Phe. Phe is an essential amino acid found in all protein-containing foods. Without sufficient quantity or activity of PAH, Phe accumulates to abnormally high levels in the blood, resulting in a variety of serious neurological complications, including severe mental retardation and brain damage, mental illness, seizures and other cognitive problems. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients under the age of 40 in developed countries have been diagnosed at birth. PKU can be managed by a Phe-restricted diet, which is supplemented by nutritional replacement products, like formulas and specially manufactured foods; however, it is difficult for most patients to adhere to the strict diet to the extent needed for achieving adequate control of blood Phe levels.
PALYNZIQ is approved for marketing in the U.S. for adult patients with PKU who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. PALYNZIQ is also approved for marketing in the EU, Australia, and Brazil for patients ages 16 and older who have inadequate blood Phe control (blood Phe concentrations greater than 600 micromol/L) despite prior management with available treatment options.
In the U.S., PALYNZIQ is only available through the PALYNZIQ Risk Evaluation and Mitigation Strategy (REMS) program, which is required by the U.S. Food and Drug Administration (FDA) to mitigate the risk of anaphylaxis while using the product. Notable requirements of the company’s REMS program include the following: prescribers must be certified by enrolling in the REMS program and completing training; prescribers must prescribe auto-injectable epinephrine with PALYNZIQ; pharmacies must be certified with the REMS program and must dispense PALYNZIQ only to patients who are authorized to receive it; patients must enroll in the REMS program and be educated about the risk of anaphylaxis by a certified prescriber to ensure they understand the risks and benefits of treatment with PALYNZIQ; and patients must have auto-injectable epinephrine available at all times while taking PALYNZIQ.
BRINEURA
BRINEURA is a recombinant human tripeptidyl peptidase 1 (TPP1) for the treatment of patients with CLN2, a form of Batten disease. CLN2 is an incurable, rapidly progressive disease that typically ends in patient death by 10-12 years of age. Patients are initially healthy but begin to decline at approximately the age of three. BRINEURA is the first treatment approved to slow the progression of loss of ambulation in children with CLN2 disease and was one of the first therapies to go through an accelerated review procedure in the EU.
BRINEURA is administered via intracerebroventricular (ICV) infusion and intended to be used in combination with a delivery device, such as an injector or other delivery system.
BRINEURA is approved for marketing in the U.S. and in the EU for children of all ages and in other international markets.
ALDURAZYME
ALDURAZYME is a highly purified protein that is designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of GAGs. MPS I is a progressive and debilitating life-threatening genetic disease that is caused by the deficiency of alpha-L-iduronidase. Patients with MPS I typically become progressively worse and experience multiple severe and debilitating symptoms resulting from the build-up of carbohydrate residues in all tissues in the body. These symptoms include inhibited growth, delayed and regressed mental development (in the severe form of the disease), enlarged liver and spleen, joint deformities and reduced range of motion, impaired cardiovascular function, upper airway obstruction, reduced pulmonary function, frequent ear and lung infections, impaired hearing and vision, sleep apnea, malaise and reduced endurance.
The company developed ALDURAZYME through collaboration with Sanofi. Under the company’s collaboration agreement with Sanofi, the company is responsible for manufacturing ALDURAZYME and supplying it to Sanofi. Sanofi and BioMarin are members of BioMarin/Genzyme LLC, a 50/50 limited liability company (the BioMarin/Genzyme LLC) that: holds the intellectual property relating to ALDURAZYME and other collaboration products and licenses all such intellectual property on a royalty-free basis to Sanofi and BioMarin to allows the company to exercise its rights and perform its obligations under the agreements related to the BioMarin/Genzyme LLC; and engages in research and development activities that are mutually selected and funded by Sanofi and the company.
ALDURAZYME is approved for marketing in the U.S., the EU and other international markets.
KUVAN
KUVAN is a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for PAH, indicated for patients with PKU. KUVAN is the first drug for the treatment of PKU, which is an inherited metabolic disease.
KUVAN is approved for marketing in the U.S., the EU and other international markets (excluding Japan). In certain international markets, KUVAN is also approved for, or is only approved for, the treatment of primary BH4 deficiency, a different disorder than PKU.
Generic versions of KUVAN are available in several countries around the world, including multiple generic versions in the U.S. and the EU. Several generic versions of KUVAN have also been approved either centrally by the European Commission (EC) or on a country-by-country basis throughout the EU.
ROCTAVIAN
ROCTAVIAN is an adeno associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Hemophilia A, also called factor VIII deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. People living with hemophilia A are not able to form blood clots efficiently and are at risk for excessive bleeding from modest injuries, potentially endangering their lives. People with severe hemophilia often bleed spontaneously into their muscles or joints.
ROCTAVIAN was conditionally approved by the EC in August 2022 and approved by the FDA in the U.S. in June 2023.
In 2024, the company will focus commercial, research and manufacturing activities in three prioritized countries, including the U.S., Germany and Italy as part of its updated ROCTAVIAN strategy.
Research and Development Programs
The company has multiple clinical and preclinical product candidates in various stages of development that are intended to address the root causes of genetic conditions with a significant unmet medical need. Generally, the company’s development programs have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options.
VOXZOGO
The company is advancing development across its CANOPY clinical program with VOXZOGO for the treatment of hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. In 2024, the company began enrollment in the pivotal Phase 3 registration-enabling study for the treatment of hypochondroplasia.
BMN 333
BMN 333 is a longer-acting CNP in development for the treatment of multiple growth disorders, including achondroplasia and hypochondroplasia. The company initiated the first-in-human study of BMN 333 in January 2025.
BMN 349
BMN 349 is an oral therapeutic in development for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency. The company completed the single-ascending dose phase of the first-in-human study and dosing in the multiple-ascending dose phase of the study began in December 2024.
BMN 351
BMN 351 is the company’s next-generation oligonucleotide in development for the treatment of Duchenne Muscular Dystrophy (DMD). The company completed enrollment into the first and second dose cohorts in late 2024.
Manufacturing
The company manufactures the active pharmaceutical ingredients (API) for ALDURAZYME, NAGLAZYME, PALYNZIQ, VOXZOGO, and ROCTAVIAN in its production facilities located in Novato, California. The company also has a commercial-scale gene therapy manufacturing facility, located in Novato, California. The company manufactures the API for BRINEURA and VIMIZIM in its manufacturing facility in Shanbally, Cork, Ireland. The company’s Novato and Shanbally facilities have been inspected and have demonstrated compliance with current Good Manufacturing Practice (cGMP) to the satisfaction of the FDA, the EC and health agencies in other countries. The company also has installed aseptic filling and drug product packaging capabilities at the Shanbally site, which received EU approval in 2024. Additional regulatory inspections of this new drug product filling facility are planned and/or anticipated over the coming months.
Sales and Marketing
The company has established a commercial organization, which starting in 2025, is primarily structured around three business units: Skeletal Conditions, Enzyme Therapies and ROCTAVIAN. This organization, which includes a sales force, supports its product lines directly in the U.S., Europe, South America and certain other significant markets. For other selected markets, the company has signed agreements with other companies to act as distributors of all its products, other than ALDURAZYME. Most of these agreements generally grant the distributor the right to market the product in the territory and the obligation to secure all necessary regulatory approvals for commercial or named patient sales. Additional markets are being assessed at this time and additional agreements may be signed in the future.
Sanofi has the exclusive right to distribute, market and sell ALDURAZYME globally and is required to purchase its requirements exclusively from the company.
In the U.S., the company’s products (other than ALDURAZYME) are marketed through its commercial teams, including sales representatives and supporting staff members, who promote the company’s products directly to physicians in specialties appropriate for each product. Outside of the U.S., the company’s sales representatives and supporting staff members market its products (other than ALDURAZYME).
The company utilizes third-party logistics companies to store and distribute the company’s products. Moreover, the company uses third-party vendors, such as advertising agencies, market research firms and suppliers of marketing and other sales support-related services, to assist with the company’s commercial activities.
Customers
Customers for the company’s products (other than ALDURAZYME) include a limited number of specialty pharmacies and end-users, such as hospitals and non-U.S. government agencies. The company also sells its products (other than ALDURAZYME) to its authorized distributors and to certain larger pharmaceutical wholesalers globally, which act as intermediaries between the company and end-users and generally do not stock significant quantities of the company’s products. However, in certain countries, governments place large periodic orders for the company’s products. PALYNZIQ is distributed in the U.S. pursuant to the REMS program through a limited number of certified specialty pharmacies. During 2024, 25% of the company’s net product revenue was generated by two customers. Sanofi is its sole customer for ALDURAZYME and is responsible for distributing, marketing, and selling ALDURAZYME to third parties.
Trademarks
BioMarin, BRINEURA, KUVAN, NAGLAZYME, PALYNZIQ, ROCTAVIAN, VIMIZIM and VOXZOGO are the company’s registered trademarks. ALDURAZYME is a registered trademark of BioMarin/Genzyme LLC.
Government Regulation
The company’s products require approval from the FDA, the EC (based on the scientific opinions issued by the European Medicines Agency (EMA)) and corresponding agencies in other countries before they can be marketed.
The U.S. Foreign Corrupt Practices Act (FCPA), to which the company is subject, prohibits corporations and individuals from engaging in certain activities to obtain or retain business or to influence a person working in an official capacity.
The legislative and regulatory environments regarding privacy and data protection are continually evolving and developing, in response to increasing global attention. In the U.S., for example, the company is subject to the CCPA along with the California Privacy Rights Act of 2020 (CPRA).
The company is also subject to the EU’s General Data Protection Regulation GDPR, which requires that personal data is only collected for specified, explicit and legal purposes as set out in the GDPR or local laws, and the data may then only be processed in a manner consistent with those purposes.
History
BioMarin Pharmaceutical Inc. was incorporated in Delaware in 1996. The company began its operations in 1997.
