BioMarin Pharmaceutical

Last updated
BioMarin Pharmaceutical Inc.
Company type Public
Industry Biotechnology
FoundedMarch 1997;27 years ago (1997-03)
Founders
  • Christopher Starr, Ph.D.
  • Grant W. Denison Jr.
Headquarters San Rafael, California, U.S.
Key people
Products
RevenueIncrease2.svg US$2.419 billion [2]  (2023)
Increase2.svg US$158.1 million [2]  (2023)
Increase2.svg US$167.65 million [2]  (2023)
Total assets Increase2.svg US$6,841 million [2]  (2023)
Total equity Increase2.svg US$4,951 million [2]  (2023)
Number of employees
3,401 (2023)
Website biomarin.com
Footnotes /references
[3] [4] [2]

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). [5] [6]

Contents

Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials.

History

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]

Business development

In 2002, BioMarin acquired Glyko Biomedical. [9]

In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate. [10] 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. [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 available poly (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 of achondroplasia. [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 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. [16]

In November 2014, the company agreed to the acquisition of Prosensa for up to $840 million; [17] however, the range of treatments for Duchenne 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]

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

Prosensa
(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)

Products

As of 2022, BioMarin has six products on the market, each of which is an orphan drug. [20]

Biomarin is working to develop several new drugs.

Controversies

In 2010, BioMarin became involved in controversy [22] 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. [23]

In 2013, BioMarin Pharmaceuticals was at the center of a high profile debate regarding expanded 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 unapproved PARP inhibitor drug candidate that had exhibited promising activity in a small Phase 1 clinical trial. The company declined, citing safety concerns. [25] Ms. Sloan eventually received a similar drug candidate from a different company. [26]

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. [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 and Stephen 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 from Morquio syndrome free of charge. BioMarin stopped providing free Vimizim at the beginning of the year after negotiations with Belgian health authorities regarding reimbursement of the product repeatedly failed. This caused the parents to start legal proceedings to force the company to keep providing the medicine free of charge. BioMarin was ordered in a preliminary 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]

Related Research Articles

<span class="mw-page-title-main">Lambert–Eaton myasthenic syndrome</span> Autoimmune disorder causing muscular weakness

Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder characterized by muscle weakness of the limbs. It is also known as myasthenic syndrome, Eaton–Lambert syndrome, and when related to cancer, carcinomatous myopathy.

<span class="mw-page-title-main">Biogen</span> Pharmaceutical company

Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, United States specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide. Biogen operates in Argentina, Brazil, Canada, China, France, Germany, Hungary, India, Italy, Japan, Mexico, Netherlands, Poland, Sweden, and Switzerland.

<span class="mw-page-title-main">Merck Group</span> German multinational science and technology company

The Merck Group, branded and commonly known as Merck, is a German multinational science and technology company headquartered in Darmstadt, with about 60,000 employees and a presence in 66 countries. The group includes around 250 companies; the main company is Merck KGaA in Germany. The company is divided into three business lines: Healthcare, Life Sciences and Electronics. Merck was founded in 1668 and is the world's oldest operating chemical and pharmaceutical company, as well as one of the largest pharmaceutical companies globally.

<span class="mw-page-title-main">Tetrahydrobiopterin</span> Chemical compound

Tetrahydrobiopterin (BH4, THB), also known as sapropterin (INN), is a cofactor of the three aromatic amino acid hydroxylase enzymes, used in the degradation of amino acid phenylalanine and in the biosynthesis of the neurotransmitters serotonin (5-hydroxytryptamine, 5-HT), melatonin, dopamine, norepinephrine (noradrenaline), epinephrine (adrenaline), and is a cofactor for the production of nitric oxide (NO) by the nitric oxide synthases. Chemically, its structure is that of a (dihydropteridine reductase) reduced pteridine derivative (quinonoid dihydrobiopterin).

<span class="mw-page-title-main">Pralatrexate</span> Chemical compound

Pralatrexate, sold under the brand name Folotyn, is a medication used for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL).

<span class="mw-page-title-main">Celgene</span> American biopharmaceutical company

Celgene Corporation is a pharmaceutical company that makes cancer and immunology drugs. Its major product is Revlimid (lenalidomide), which is used in the treatment of multiple myeloma, and also in certain anemias. The company is incorporated in Delaware, headquartered in Summit, New Jersey, and a subsidiary of Bristol Myers Squibb (BMS).

<span class="mw-page-title-main">Tetrahydrobiopterin deficiency</span> Medical condition

Tetrahydrobiopterin deficiency (THBD, BH4D) is a rare metabolic disorder that increases the blood levels of phenylalanine. Phenylalanine is an amino acid obtained normally through the diet, but can be harmful if excess levels build up, causing intellectual disability and other serious health problems. In healthy individuals, it is metabolised (hydroxylated) into tyrosine, another amino acid, by phenylalanine hydroxylase. However, this enzyme requires tetrahydrobiopterin as a cofactor and thus its deficiency slows phenylalanine metabolism.

<span class="mw-page-title-main">Astellas Pharma</span> Japanese pharmaceutical company

Astellas Pharma Inc. is a Japanese multinational pharmaceutical company, formed on 1 April 2005 from the merger of Yamanouchi Pharmaceutical Co., Ltd. and Fujisawa Pharmaceutical Co., Ltd..

Iduronidase, sold as Aldurazyme, is an enzyme with the systematic name glycosaminoglycan α-L-iduronohydrolase. It catalyses the hydrolysis of unsulfated α-L-iduronosidic linkages in dermatan sulfate.

<span class="mw-page-title-main">Amifampridine</span> Chemical compound

Amifampridine is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and Lambert–Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ad hoc forms of the drug, since there was no marketed form.

<span class="mw-page-title-main">PTC Therapeutics</span> Pharmaceutical company

PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.

<span class="mw-page-title-main">National Organization for Rare Disorders</span> U.S. nonprofit organization

The National Organization for Rare Disorders (NORD) is a nonprofit organization, based in Connecticut, aiming to provide support for individuals with rare diseases by advocating and funding research, education, and networking among service providers. It was founded in 1983 by Abbey Meyers, along with individuals and rare diseases leaders of rare disease support groups, and it is a 501(c)(3) tax exempt organization.

Emil Kakkis is an American medical geneticist known for his work to develop treatments for ultra rare disorders. He is the Founder of the Everylife Foundation for Rare Disease and Founder, CEO and President of Ultragenyx Pharmaceutical Inc.

Flexion Therapeutics, Inc. is an American biopharmaceutical company based in Burlington, Massachusetts that is focused on the discovery, development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis (OA), the most common form of arthritis.

<span class="mw-page-title-main">AbbVie</span> American pharmaceutical company

AbbVie Inc. is an American pharmaceutical company headquartered in North Chicago, Illinois. It is ranked sixth on the list of largest biomedical companies by revenue. In 2023, the company's seat in Forbes Global 2000 was 74, and rank 89 on the 2024 list. The company's primary product is Humira (adalimumab), administered via injection. It is approved to treat autoimmune diseases including rheumatoid arthritis, Crohn's disease, plaque psoriasis, and ulcerative colitis.

Elosulfase alfa, sold under the brand name Vimizim, is a medication used for the treatment of Morquio syndrome which is caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase. Elosulfase alfa is a synthetic version of this enzyme.

Catalyst Pharmaceuticals, Inc. is a biopharmaceutical company based in Coral Gables, Florida, United States. The company develops medicines for rare diseases, including the phosphate salt of amifampridine for the treatment of Lambert–Eaton myasthenic syndrome (LEMS). The drug is referred to under the trade name Firdapse, which was approved by the FDA for approved use in children 6 years and older with LEMS in addition to the prior approval for use in adults with LEMS on November 28, 2018. Firdapse commercially launched in January 2019.

Allergan plc is an American, Irish-domiciled pharmaceutical company that acquires, develops, manufactures and markets brand name drugs and medical devices in the areas of medical aesthetics, eye care, central nervous system, and gastroenterology. The company is the maker of Botox.

<span class="mw-page-title-main">Ultragenyx</span> American biopharmaceutical company

Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.

Relief Therapeutics is a Swiss biopharmaceutical company based in Geneva. The company focuses on developing drugs for serious diseases with few or no existing treatment options. Its lead compound, RLF-100, is a synthetic form of a natural peptide that protects the lung. The company was incorporated as Relief Therapeutics Holdings AG (RFLB.S) and listed on the SIX Swiss Exchange in 2016.

References

  1. ↑ "Leadership Team". Biomarin. Retrieved 5 December 2023.
  2. 1 2 3 4 5 6 "Biomarin Pharmaceutical Inc". reuters . Retrieved August 30, 2024.
  3. ↑ "US SEC: Form 10-K BioMarin Pharmaceutical Inc". U.S. Securities and Exchange Commission . Retrieved March 31, 2018.
  4. ↑ "BioMarin Pharmaceutical Inc. 2020 Annual Report". app.quotemedia.com. 31 December 2020. Retrieved 20 November 2021.
  5. ↑ BruceCockburnVEVO (2011-12-15), Bruce Cockburn - Call It Democracy, archived from the original on 2021-12-19, retrieved 2019-04-18
  6. ↑ "'A wonder drug helped me eat - then I had to go without'". 17 April 2019.
  7. ↑ "BioMarin". Life Sciences at CHF. Archived from the original on 2016-03-23. Retrieved 2016-07-09.
  8. ↑ "Form S-1 Registration Statement". Biomarin Pharmaceuticals Inc. Retrieved 15 December 2015.
  9. ↑ "BioMarin Pharmaceutical Acquires Glyko Biomedical". Deals. LEXPERT. Thomson Reuters. January 2003. Archived from the original on 2013-12-28.
  10. ↑ 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.
  11. ↑ "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)
  12. ↑ 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.
  13. ↑ 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.
  14. ↑ Andersen, Karen (2010-10-21). "BioMarin Highlights Diverse Early-Stage Pipeline at R&D Day". Morningstar. Archived from the original on 2011-07-06. Retrieved 2011-02-14.
  15. ↑ Brown, Steven (2013-01-07). "BioMarin Pharmaceutical buys Zacharon for $10 million". San Francisco Business Times. Retrieved 2013-01-07.
  16. ↑ "BioMarin Nabs Repligen's HDACi Compound Library". News: Discovery & Development. Gen. Eng. Biotechnol. News (paper). Vol. 34, no. 4. 15 February 2014. p. 14.
  17. ↑ "BioMarin to Acquire Prosensa for Up to $840M". GEN - Genetic Engineering and Biotechnology News. 2014-11-24. Retrieved 2020-07-03.
  18. ↑ "BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe (NASDAQ:BMRN)". investors.bmrn.com. Archived from the original on 2016-06-04. Retrieved 2016-07-04.
  19. ↑ Earley, Kelly (2019-10-02). "BioMarin opens new Dublin office to support EMEA growth". Silicon Republic. Retrieved 2019-10-07.
  20. ↑ BioMarin Office Site: Products
  21. ↑ 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.
  22. ↑ Goldberg, Adrian (2010-11-21). "Drug firms accused of exploiting loophole for profit". BBC News . Retrieved 2010-11-21.
  23. ↑ Hawkes, N.; Cohen, D. (2010-11-10). "What makes an orphan drug?". BMJ. 341. British Medical Journal: c6459. doi:10.1136/bmj.c6459. PMID   21081607. S2CID   2486975 . Retrieved 2010-11-21.
  24. ↑ "Andrea Sloan Faces Pharma Firm With History Of Indifference". Huffington Post. 2013-09-26. Retrieved 2013-12-24.
  25. ↑ "In cancer drug battle, both sides appeal to ethics". CNN. 2013-09-20. Retrieved 2013-12-24.
  26. ↑ Ed Silverman for Pharmalot. October 4, 2013 Andrea Sloan, 'Poster Child' For Compassionate Use, Gets A Drug Archived 2013-12-28 at the Wayback Machine
  27. ↑ "Hannahs Kampf gegen Pharmafirma". B5 aktuell. 2015-05-13. Retrieved 2015-05-13.
  28. ↑ "I helped test a wonder drug - then I was denied it". 2019-04-17. Retrieved 2019-04-18.
  29. ↑ "BioMarin moet verder levensreddend middel leveren aan patiënte" [BioMarin must continue to provide life-saving drug to patient]. www.tijd.be (in Dutch). De Tijd. 26 June 2019. Retrieved 26 June 2019.
This page is based on this Wikipedia article
Text is available under the CC BY-SA 4.0 license; additional terms may apply.
Images, videos and audio are available under their respective licenses.