From Wikipedia, the free encyclopedia
 |
|
| Type | Public |
|---|---|
|
Traded as |
|
| Industry | Biotechnology |
| Founded | March 1997; 26 years ago |
| Founders |
|
| Headquarters | San Rafael, California, U.S. |
|
Key people |
|
| Products |
|
| Revenue |  US$1.860 Billion (Fiscal Year Ended December 31, 2020)[1] |
|
Operating income |
–US$36.765 Million (Fiscal Year Ended December 31, 2020)[1] |
|
Net income |
US$0.859 Billion (Fiscal Year Ended December 31, 2020)[1] |
| Total assets | US$5.848 Billion (Fiscal Year Ended December 31, 2020)[1] |
| Total equity | US$4.106 Billion (Fiscal Year Ended December 31, 2020)[1] |
|
Number of employees |
2,581 (2017) |
| Website | biomarin.com |
| Footnotes / references [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).[3][4]
Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials.
History[edit]
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.[5] Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting.[6]
Business development[edit]
In 2002, BioMarin acquired Glyko Biomedical.[7]
In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate.[8] 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.[9]
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.[10] 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.[11] 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.[12]
In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan metabolism.[13]
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.[14]
In November 2014, the company agreed to the acquisition of Prosensa for up to $840 million;[15] however, the range of treatments for Duchenne muscular dystrophy failed to attain FDA approval, and development ceased in May 2016.[16]
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.[17]
Acquisition history[edit]
The following is an illustration of the company’s major mergers and acquisitions and historical predecessors (this is not a comprehensive list):
| BioMarin Pharmaceutical |
|
|||||||||||||||||||||||||||||||||
Products[edit]
As of 2022, BioMarin has six products on the market, each of which is an orphan drug.[18]
- Tetrahydrobiopterin (branded as Kuvan) (sapropterin dihydrochloride), a small molecule drug for phenylketonuria, introduced in 2007 as the first medication-based intervention to treat phenylketonuria[19]
- Arylsulfatase B (branded as Naglazyme) (galsulfase), a recombinant protein therapeutic for Maroteaux–Lamy syndrome (also called mucopolysaccharidosis type VI)
- Iduronidase (branded as Aldurazyme), a recombinant protein therapeutic for mucopolysaccharidosis I
- Amifampridine (branded as Firdapse), a small molecule drug for Lambert–Eaton myasthenic syndrome (as of 2013 approved in the EU only)
- Elosulfase alfa (branded as Vimizim), is the only enzyme replacement therapy to address the cause of Morquio A Syndrome (MPS IVA), which affects an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism.
- Cerliponase alfa (branded as Brineura), is an enzyme replacement treatment for Batten disease, which is a form of neuronal ceroid lipofuscinosis. It was approved in 2017.
- Valoctocogene roxaparvovec (branded as Roctavian) is an adeno-associated viral vector for treatment of hemophilia A that aims to transfer a working copy of the Factor VIII gene into patients who lack one. It was approved in the EU in August 2022.
Biomarin is working to develop several new drugs.
Controversies[edit]
In 2010, BioMarin became involved in controversy[20] 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.[21]
In 2013, BioMarin Pharmaceuticals was at the center of a high profile debate regarding expanded access of cancer patients to experimental drugs.[22] 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.[23] Ms. Sloan eventually received a similar drug candidate from a different company.[24]
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.[25]
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»[26]
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.[27]
References[edit]
- ^ a b c d e «BioMarin Pharmaceutical Inc. 2020 Annual Report». app.quotemedia.com. 31 December 2020. Retrieved 20 November 2021.
- ^ «US SEC: Form 10-K BioMarin Pharmaceutical Inc». U.S. Securities and Exchange Commission. Retrieved March 31, 2018.
- ^ BruceCockburnVEVO (2011-12-15), Bruce Cockburn — Call It Democracy, archived from the original on 2021-12-19, retrieved 2019-04-18
- ^ «‘A wonder drug helped me eat — then I had to go without’«. 17 April 2019.
- ^ «BioMarin». Life Sciences at CHF. Archived from the original on 2016-03-23. Retrieved 2016-07-09.
- ^ «Form S-1 Registration Statement». Biomarin Pharmaceuticals Inc. Retrieved 15 December 2015.
- ^ «BioMarin Pharmaceutical Acquires Glyko Biomedical». Deals. LEXPERT. Thomson Reuters. January 2003. Archived from the original on 2013-12-28.
- ^ 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.
- ^ «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)
- ^ 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.
- ^ 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.
- ^ 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.
- ^ Brown, Steven (2013-01-07). «BioMarin Pharmaceutical buys Zacharon for $10 million». San Francisco Business Times. Retrieved 2013-01-07.
- ^ «BioMarin Nabs Repligen’s HDACi Compound Library». News: Discovery & Development. Gen. Eng. Biotechnol. News (paper). Vol. 34, no. 4. 15 February 2014. p. 14.
- ^ «BioMarin to Acquire Prosensa for Up to $840M». GEN — Genetic Engineering and Biotechnology News. 2014-11-24. Retrieved 2020-07-03.
- ^ «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.
- ^ Earley, Kelly (2019-10-02). «BioMarin opens new Dublin office to support EMEA growth». Silicon Republic. Retrieved 2019-10-07.
- ^ BioMarin Office Site: Products
- ^ 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.
- ^ Goldberg, Adrian (2010-11-21). «Drug firms accused of exploiting loophole for profit». BBC News. Retrieved 2010-11-21.
- ^ Hawkes, N.; Cohen, D. (2010-11-10). «What makes an orphan drug?». BMJ. British Medical Journal. 341: c6459. doi:10.1136/bmj.c6459. PMID 21081607. S2CID 2486975. Retrieved 2010-11-21.
- ^ «Andrea Sloan Faces Pharma Firm With History Of Indifference». Huffington Post. 2013-09-26. Retrieved 2013-12-24.
- ^ «In cancer drug battle, both sides appeal to ethics». CNN. 2013-09-20. Retrieved 2013-12-24.
- ^ 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
- ^ «Hannahs Kampf gegen Pharmafirma». B5 aktuell. 2015-05-13. Retrieved 2015-05-13.
- ^ «I helped test a wonder drug — then I was denied it». 2019-04-17. Retrieved 2019-04-18.
- ^ «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.
External links[edit]
- Business data for BioMarin Pharmaceutical Inc.:
- SEC filings
- Yahoo!
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).[3][4]
|
|
| Type | Public |
|---|---|
|
Traded as |
|
| Industry | Biotechnology |
| Founded | March 1997; 26 years ago |
| Founders |
|
| Headquarters | San Rafael, California, U.S. |
|
Key people |
|
| Products |
|
| Revenue | US$1.860 Billion (Fiscal Year Ended December 31, 2020)[1] |
|
Operating income |
–US$36.765 Million (Fiscal Year Ended December 31, 2020)[1] |
|
Net income |
US$0.859 Billion (Fiscal Year Ended December 31, 2020)[1] |
| Total assets | US$5.848 Billion (Fiscal Year Ended December 31, 2020)[1] |
| Total equity | US$4.106 Billion (Fiscal Year Ended December 31, 2020)[1] |
|
Number of employees |
2,581 (2017) |
| Website | biomarin.com |
| Footnotes / references [2] |
Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials.
HistoryEdit
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.[5] Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting.[6]
Business developmentEdit
In 2002, BioMarin acquired Glyko Biomedical.[7]
In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate.[8] 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.[9]
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.[10] 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.[11] 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.[12]
In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan metabolism.[13]
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.[14]
In November 2014, the company agreed to the acquisition of Prosensa for up to $840 million;[15] however, the range of treatments for Duchenne muscular dystrophy failed to attain FDA approval, and development ceased in May 2016.[16]
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.[17]
Acquisition historyEdit
The following is an illustration of the company’s major mergers and acquisitions and historical predecessors (this is not a comprehensive list):
| BioMarin Pharmaceutical |
|
|||||||||||||||||||||||||||||||||
ProductsEdit
As of 2022, BioMarin has six products on the market, each of which is an orphan drug.[18]
- Tetrahydrobiopterin (branded as Kuvan) (sapropterin dihydrochloride), a small molecule drug for phenylketonuria, introduced in 2007 as the first medication-based intervention to treat phenylketonuria[19]
- Arylsulfatase B (branded as Naglazyme) (galsulfase), a recombinant protein therapeutic for Maroteaux–Lamy syndrome (also called mucopolysaccharidosis type VI)
- Iduronidase (branded as Aldurazyme), a recombinant protein therapeutic for mucopolysaccharidosis I
- Amifampridine (branded as Firdapse), a small molecule drug for Lambert–Eaton myasthenic syndrome (as of 2013 approved in the EU only)
- Elosulfase alfa (branded as Vimizim), is the only enzyme replacement therapy to address the cause of Morquio A Syndrome (MPS IVA), which affects an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism.
- Cerliponase alfa (branded as Brineura), is an enzyme replacement treatment for Batten disease, which is a form of neuronal ceroid lipofuscinosis. It was approved in 2017.
- Valoctocogene roxaparvovec (branded as Roctavian) is an adeno-associated viral vector for treatment of hemophilia A that aims to transfer a working copy of the Factor VIII gene into patients who lack one. It was approved in the EU in August 2022.
Biomarin is working to develop several new drugs.
ControversiesEdit
In 2010, BioMarin became involved in controversy[20] 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.[21]
In 2013, BioMarin Pharmaceuticals was at the center of a high profile debate regarding expanded access of cancer patients to experimental drugs.[22] 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.[23] Ms. Sloan eventually received a similar drug candidate from a different company.[24]
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.[25]
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»[26]
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.[27]
ReferencesEdit
- ^ a b c d e «BioMarin Pharmaceutical Inc. 2020 Annual Report». app.quotemedia.com. 31 December 2020. Retrieved 20 November 2021.
- ^ «US SEC: Form 10-K BioMarin Pharmaceutical Inc». U.S. Securities and Exchange Commission. Retrieved March 31, 2018.
- ^ BruceCockburnVEVO (2011-12-15), Bruce Cockburn — Call It Democracy, archived from the original on 2021-12-19, retrieved 2019-04-18
- ^ «‘A wonder drug helped me eat — then I had to go without’«. 17 April 2019.
- ^ «BioMarin». Life Sciences at CHF. Archived from the original on 2016-03-23. Retrieved 2016-07-09.
- ^ «Form S-1 Registration Statement». Biomarin Pharmaceuticals Inc. Retrieved 15 December 2015.
- ^ «BioMarin Pharmaceutical Acquires Glyko Biomedical». Deals. LEXPERT. Thomson Reuters. January 2003. Archived from the original on 2013-12-28.
- ^ 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.
- ^ «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)
- ^ 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.
- ^ 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.
- ^ 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.
- ^ Brown, Steven (2013-01-07). «BioMarin Pharmaceutical buys Zacharon for $10 million». San Francisco Business Times. Retrieved 2013-01-07.
- ^ «BioMarin Nabs Repligen’s HDACi Compound Library». News: Discovery & Development. Gen. Eng. Biotechnol. News (paper). Vol. 34, no. 4. 15 February 2014. p. 14.
- ^ «BioMarin to Acquire Prosensa for Up to $840M». GEN — Genetic Engineering and Biotechnology News. 2014-11-24. Retrieved 2020-07-03.
- ^ «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.
- ^ Earley, Kelly (2019-10-02). «BioMarin opens new Dublin office to support EMEA growth». Silicon Republic. Retrieved 2019-10-07.
- ^ BioMarin Office Site: Products
- ^ 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.
- ^ Goldberg, Adrian (2010-11-21). «Drug firms accused of exploiting loophole for profit». BBC News. Retrieved 2010-11-21.
- ^ Hawkes, N.; Cohen, D. (2010-11-10). «What makes an orphan drug?». BMJ. British Medical Journal. 341: c6459. doi:10.1136/bmj.c6459. PMID 21081607. S2CID 2486975. Retrieved 2010-11-21.
- ^ «Andrea Sloan Faces Pharma Firm With History Of Indifference». Huffington Post. 2013-09-26. Retrieved 2013-12-24.
- ^ «In cancer drug battle, both sides appeal to ethics». CNN. 2013-09-20. Retrieved 2013-12-24.
- ^ 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
- ^ «Hannahs Kampf gegen Pharmafirma». B5 aktuell. 2015-05-13. Retrieved 2015-05-13.
- ^ «I helped test a wonder drug — then I was denied it». 2019-04-17. Retrieved 2019-04-18.
- ^ «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.
External linksEdit
- Business data for BioMarin Pharmaceutical Inc.:
- SEC filings
- Yahoo!
|
|
| Тип | Общественные |
|---|---|
|
Торгуется как |
|
| Промышленность | Биотехнологии |
| Основан | Март 1997 г . ; 24 года назад |
| Учредители | |
| Штаб-квартира | Сан-Рафаэль, Калифорния , США |
|
Действующие лица |
|
| Продукты | |
| Доход | 1,31 миллиарда долларов США (2017) |
|
Операционная прибыль |
US $ −14,7 млн (2017) |
|
Чистая прибыль |
US $ −117,04 млн (2017) |
| Всего активов | 4,63 миллиарда долларов США (2017) |
| Общий капитал | 2,81 миллиарда долларов США (2017) |
|
Количество работников |
2581 (2017) |
| Веб-сайт | биомарин .com |
| Сноски / ссылки |
BioMarin Pharmaceutical Inc. — американская биотехнологическая компания со штаб-квартирой в Сан-Рафаэле, Калифорния . У него есть офисы и помещения в США , Южной Америке , Азии и Европе . Основным направлением деятельности и исследованиями BioMarin является заместительная терапия ферментами (ФЗТ). BioMarin была первой компанией, которая предоставила терапевтические средства от мукополисахаридоза типа I (MPS I), производя ларонидазу ( Aldurazyme , коммерциализируется Genzyme Corporation). BioMarin также была первой компанией, которая начала предлагать препараты для лечения фенилкетонурии ( PKU ).
На протяжении многих лет BioMarin критиковали за ценообразование на лекарства и за конкретные случаи отказа в доступе к лекарствам в клинических испытаниях.
История
Компания BioMarin была основана в 1997 году доктором философии Кристофером Старром. и Грант В. Денисон-младший с вложением 1,5 миллиона долларов от Glyko Biomedical, который стал публичным в 1999 году. Среди исходных инвесторов были MPM Bioventures, Grosvenor Fund и Флориан Шёнхартинг.
Развитие бизнеса
В 2002 году компания BioMarin приобрела Glyko Biomedical.
В 2009 году BioMarin приобрела Huxley Pharmaceuticals, Inc. (Huxley), у которой были права на патентованную форму 3,4-диаминопиридина (3,4-DAP), амифампридинфосфата . В 2010 году компания BioMarin получила разрешение Европейской комиссии на сбыт 3,4-диаминопиридина (3,4-DAP), амифампридинфосфата для лечения редкого аутоиммунного заболевания миастенического синдрома Ламберта-Итона (LEMS). Компания BioMarin выпустила продукт под названием Firdapse.
В 2010 году BioMarin приобрела LEAD Therapeutics, Inc. (LEAD), небольшую частную компанию по открытию и разработке лекарств на ранней стадии с ключевым соединением LT-673, перорально доступным ингибитором поли (АДФ-рибозы) полимеразы (PARP), изучаемым для лечения пациенты с редкими, генетически определенными формами рака. За этим приобретением последовала покупка ZyStor Therapeutics, Inc. (ZyStor), частной биотехнологической компании, занимающейся разработкой ERT для лечения лизосомных нарушений накопления, и ее ведущего продукта-кандидата, ZC-701, слияния инсулиноподобного фактора роста 2. и альфа-глюкозидаза (IGF2-GAA) в разработке при болезни Помпе. На своем дне исследований и разработок в октябре 2010 года компания BioMarin также объявила о новой программе пептидного терапевтического препарата восоритид (BMN-111) для лечения ахондроплазии .
В 2012 году BioMarin приобрела Zacharon Pharmaceuticals, частную биотехнологическую компанию, базирующуюся в Сан-Диего, которая занимается разработкой малых молекул, нацеленных на пути метаболизма гликанов.
В 2014 году BioMarin приобрела химическую библиотеку ингибиторов гистон-деацетилазы у Repligen за 2 миллиона долларов с намерением продвинуть работу по лечению атаксии Фридрейха и других неврологических расстройств.
В ноябре 2014 года компания согласилась на приобретение Prosensa за 840 миллионов долларов; однако ряд методов лечения мышечной дистрофии Дюшенна не получил одобрения FDA, и разработка прекратилась в мае 2016 года.
В октябре 2019 года стало известно, что группа откроет офис в Дублине для поддержки дальнейшего роста в Европе, на Ближнем Востоке и в Азии.
История приобретения
Ниже представлены основные слияния и поглощения компании, а также ее исторические предшественники (это неполный список):
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Продукты
По состоянию на 2016 год у BioMarin на рынке шесть продуктов, каждый из которых является орфанным препаратом .
- Тетрагидробиоптерин (известный как Kuvan) (дигидрохлорид сапроптерина), низкомолекулярный препарат для лечения фенилкетонурии , представленный в 2007 году в качестве первого медикаментозного вмешательства для лечения фенилкетонурии.
- Арилсульфатаза B (торговая марка Naglazyme) (галсульфаза), рекомбинантный терапевтический протеин для лечения синдрома Марото-Лами (также называемого мукополисахаридозом типа VI)
- Идуронидаза ( торговая марка Aldurazyme), рекомбинантный протеин для лечения мукополисахаридоза I.
- Амифампридин (под торговой маркой Firdapse), низкомолекулярный препарат от миастенического синдрома Ламберта-Итона (с 2013 года одобрен только в ЕС)
- Элосульфаза альфа (торговая марка Vimizim) — единственная заместительная ферментная терапия, направленная на устранение причины синдрома Моркио А (MPS IVA), которым страдают около 3000 пациентов в развитых странах. Заболевание возникает в результате дефицита активности фермента, участвующего в метаболизме гликозаминогликанов (ГАГ).
- Cerliponase alfa (торговая марка Brineura) представляет собой ферментативную терапию для лечения болезни Баттена , которая является формой нейронального цероидного липофусциноза . Он был утвержден в 2017 году.
Биомарин работает над разработкой нескольких новых лекарств.
- Valoctocogene roxaparvovec представляет собой экспериментальный препарат под исследованием для лечения гемофилии А . Это аденоассоциированный вирусный вектор, который предназначен для передачи рабочей копии гена фактора VIII пациентам, у которых она отсутствует.
Споры
В 2010 году компания BioMarin была вовлечена в полемику вокруг 3,4-диаминопиридина (3,4-DAP). BioMarin продает фосфатную соль 3,4-DAP под названием Firdapse . В 2010 году BioMarin получила эксклюзивные лицензионные права на Firdapse на 10 лет. В результате цена прописанного Национальной службы здравоохранения курса лечения увеличилась с 1987 долларов за нелицензированный препарат до 69 970 долларов за Firdapse. Компания заявляет, что до ее лицензирования не было гарантированного контроля качества продукта и не было возможности формально отслеживать необычные побочные эффекты в рамках регулирующего процесса.
В 2013 году компания BioMarin Pharmaceuticals оказалась в центре громких дебатов о расширении доступа онкологических больных к экспериментальным препаратам. По совету своего врача Андреа Слоан, пациентка с распространенным раком яичников, попросила компанию предоставить ей доступ к BMN 673, неутвержденному кандидату в ингибиторы PARP, который продемонстрировал многообещающую активность в небольшом клиническом испытании фазы 1 . Компания отказалась, сославшись на соображения безопасности. В конце концов г-жа Слоан получила аналогичный кандидат на лекарство от другой компании.
В 2015 году возник еще один спор по поводу расширенного доступа , касающийся поставки лекарства, находящегося на клинических испытаниях, немецкому ребенку, страдающему заболеванием мозга, но не участвовавшему в исследовании.
В апреле 2019 года BBC сообщила, что пациентам, которые принимали участие в пробном лечении препаратом Куван (гидрохлорид сапроптерина), позже было отказано в доступе к нему. Национальная служба здравоохранения и член парламента Стивен Хаммонд раскритиковали компанию за терпеливую спекуляцию. В ответ компания прокомментировала следующее: «BioMarin разочарована тем, что Национальная служба здравоохранения Англии не признала ценность лечения пациентов с ФКУ с помощью Кувана, несмотря на более чем десятилетние положительные результаты лечения пациентов в 26 странах Европы, России и Турции».
В июне 2019 года бельгийский суд обязал BioMarin продолжать бесплатно поставлять Вимизим молодой девушке, страдающей синдромом Моркио . Компания BioMarin прекратила предоставлять Вимизим бесплатно в начале года после того, как переговоры с бельгийскими органами здравоохранения о возмещении стоимости продукта неоднократно терпели неудачу. Это заставило родителей подать в суд, чтобы заставить компанию продолжать предоставлять лекарства бесплатно. В предварительном судебном запрете BioMarin было приказано продолжать делать это до тех пор, пока не будет вынесено окончательное решение или пока лекарство не будет доступно на бельгийском рынке по разумной цене.
использованная литература
внешние ссылки
- Коммерческие данные BioMarin Pharmaceutical Inc .:
- Google Финансы
- Yahoo! Финансы
- Документы SEC