BioMarin Pharmaceutical | Wikipedia audio article

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). == 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. Seed investors were amongst others MPM Bioventures,
Grosvenor Fund and Florian Schönharting. == Business development ==
In 2002, BioMarin acquired Glyko Biomedical.In 2009, BioMarin acquired Huxley Pharmaceuticals,
Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine
phosphate. 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.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. 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. 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.In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology
company based in San Diego focused on developing small molecules targeting pathways of glycan
metabolism.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.In November 2014, the company agreed to the acquisition
of Prosensa for up to $840 million; however, the range of treatments for Duchenne Muscular
Dystrophy failed to attain FDA approval, and development ceased in May 2016. === Acquisition history ===
The following is an illustration of the company’s major mergers and acquisitions and historical
predecessors (this is not a comprehensive list): == Products ==
As of 2016, BioMarin has six products on the market, each of which is an orphan drug. Tetrahydrobiopterin (branded as Kuvan) (sapropterin
dihydrochloride), a small molecule drug for phenylketonuria, introduced in 2007 as the
first medication-based intervention to treat phenylketonuria
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.Biomarin is working
to develop several new drugs. Valoctocogene Roxaparvovec is an experimental
drug under investigation for treatment of hemophilia A. It is an adeno-associated viral
vector that aims to transfer a working copy of the Factor VIII gene into patients who
lack one. == Controversies ==
In 2010, BioMarin became involved in controversy 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.In 2013, BioMarin Pharmaceuticals
was at the center of a high profile debate regarding expanded access of cancer patients
to experimental drugs. 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. Ms. Sloan eventually received a similar drug
candidate from a different company.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

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