History
2009 | 2008 | 2007 | 2006 | 2005 | 2004 | 2003 | 2000 | 1999 | 1998 | 1997
BioMarin Receives Notice of Allowance for Once Daily Dosing Patent for Kuvan
In March 2009, BioMarin received a notice from the U.S. Patent Office reporting allowance of claims covering once daily dosing methods for Kuvan® in the treatment of phenylketonuria (PKU). The company expects that the patent will be issued later in 2009. If issued, the patent’s 20-year term would expire in 2024. The company has a number of other pending patent applications covering various aspects of Kuvan compositions and dosing.
BioMarin’s Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA
In March 2009, BioMarin received formal acceptance from the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for its application for the clinical trial authorization (CTA) for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS Type IVA) or Morquio A Syndrome. A Phase 1/2 clinical trial is expected to begin soon.
BioMarin Appoints Dr. Henry J. Fuchs as Senior Vice President and Chief Medical Officer
In March 2009, BioMarin welcomed Dr. Henry J. Fuchs as Senior Vice President and Chief Medical Officer (CMO). Dr. Fuchs replaced longtime BioMarin CMO, Dr. Emil Kakkis.
Naglazyme Approved by Brazil’s National Health Surveillance Agency
In March 2009, BioMarin received marketing approval from ANVISA, Brazil’s National Health Surveillance Agency, for Naglazyme® (galsulfase) for the treatment of patients with Mucopolysaccharidosis VI (MPS VI). Estimates indicate that Brazil has the largest known number of affected MPS VI patients in the world.
Results From Phase 2 Clinical Study of 6R-BH4 in Peripheral Arterial Disease Not Statistically Significant
In February 2009, BioMarin announced results from its Phase 2 multi-center, randomized, double-blind, placebo-controlled clinical study of 6R-BH4 in patients with symptomatic peripheral arterial disease (PAD). There was no statistical significance observed between the 6R-BH4 treatment and placebo groups. BioMarin will determine the future of its 6R-BH4 cardiovascular program following the results of additional investigator-sponsored studies of 6R-BH4 including proteinuria, pulmonary arterial hypertension and 6R-BH4 plus vitamin C in patients with endothelial dysfunction.
Results of First Interim Efficacy Analysis for Riquent Phase 3 ASPEN Trial: Continuation of the Trial is Futile
In February 2009, BioMarin and La Jolla Pharmaceutical (LJP) announced that in the first interim efficacy analysis (IEA) for the Riquent® Phase 3 ASPEN trial, the Independent Data Monitoring Board (DMB) determined that the continuation of the trial was futile. Following this analysis, BioMarin and La Jolla discontinued the study and BioMarin opted-out of its agreement with La Jolla to develop and commercialize Riquent in the United States, Europe and all other territories of the world, excluding the Asia Pacific region.
BioMarin & La Jolla Pharmaceutical Sign Worldwide Development and Commercialization Agreement for Riquent
In January 2009, BioMarin and La Jolla Pharmaceutical entered into an agreement to develop and commercialize Riquent(R, La Jolla's investigational drug for lupus nephritus in the United States, Europe and all other territories of the world, excluding the Asia Pacific region. If the Phase 3 trial is successful and BioMarin opts-in the parties will share equally in all losses and profits. In the United States, BioMarin and La Jolla will share Riquent commercialization rights. In Europe and other territories outside of Asia, BioMarin will have exclusive rights to commercialize the product.
2008
Kuvan Approved By EMEA for Use in Europe
In December, the scientific committee of the European Medicines Agency (EMEA) approved Kuvan® (sapropterin dihydrochloride) as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency. This approval triggered a $30 million milestone payment to BioMarin from partner Merck Serono.
BioMarin Initiates Clinical Program for Morquio A Syndrome (MPS IVA)
In November, BioMarin initiated a clinical assessment program (MorCAP) for patients with MPS IVA (Morquio A Syndrome). Data collected will be used to augment current understanding of the disease by measuring endurance, respiratory in function and other parameters in affected patients. In 2009, the company plans to follow the program with a Phase 1b clinical trial to assess the safety and optimal dose of this enzyme replacement therapy.
Positive Results from Study of 6r-BH4 in Sickle Cell Disease
Positive results from a Phase 2a multi-center, open-label, dose-escalation clinical study of 6R-BH4 in patients with sickle cell disease (SCD) indicate that oral administration of 6R-BH4 is associated with improvements in endothelial dysfunction in sickle cell disease patients. Endothelial dysfunction was measured using the EndoPAT device to assess peripheral arterial tonometry (PAT), which is commonly used in assessing the sickle cell disease patient population.
Kuvan Receives Positive Opinion from CHMP for European Approval
In September, BioMarin and partner Merck Serono received a positive opinion for Kuvan® (sapropterin dihydrochloride) as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMEA). The CHMP recommendation will be considered by the European Commission, which will deliver its final decision on the granting of marketing authorization within 67 days. A positive opinion will trigger a $30 million milestone payment to BioMarin.
BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program
In July, BioMarin and Summit Corporation partnered in an exclusive worldwide licensing agreement for Summit's novel preclinical candidate SMT C1100 and all follow-on molecules, which are being developed to treat the fatal genetic disorder Duchenne muscular dystrophy (DMD).
Biopten (Sapropterin Dihydrochloride) Approved by Japanese Ministry of Health for the Treatment of PKU
In July, BioMarin partner, Asubio Pharma Co., Ltd., received marketing approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for a label extension of Biopten® (sapropterin dihydrochloride), which contains the same active ingredient as Kuvan® for the treatment of patients with phenylketonuria (PKU).
BioMarin Announces Program for ERT for Treatment of MPS IVA - Morquio A Syndrome
In June, BioMarin announced a new program for its third enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis IVA (MPS IVA), or Morquio A Syndrome. The company plans to initiate a Phase 1/2 clinical trial in the first quarter of 2009.
BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU
In May, the first patient initiated treatment in BioMarin's Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). The study is expected to conclude enrollment in the fourth quarter of 2008.
Naglazyme Approved by Japanese Ministry of Health
In March, AnGes MG, Inc., BioMarin's marketing and distribution partner in Japan, received approval for its Marketing Application for Naglazyme® (galsulfase) from the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with Mucopolysaccharidosis VI ( MPS VI).
BioMarin and Genzyme Restructure Aldurazyme 50/50 Joint Venture
In January, BioMarin and Genzyme announced a restructuring of their joint venture regarding Aldurazyme® (laronidase). Genzyme will continue to globally market and sell Aldurazyme for mucopolysaccharidosis I (MPS I) and BioMarin will continue to manufacture Aldurazyme. Under the revised structure, payments are projected to result in both BioMarin and Genzyme receiving approximately the same profit as under the original joint venture structure.
2007
Biomarin Re-Acquires Rights To Kuvan In Canada From Merck Serono
In December BioMarin re-acquired rights to Kuvan in Canada, which will enable the company to better coordinate commercialization efforts in the North American market. Terms of the agreement specify a reduction in royalties owed to BioMarin on Merck Serono sales outside the United States and Japan
Kuvan Approved By Fda; Launched Immediately In The U.S.
Approximately three years after filing the IND, BioMarin received FDA approval for KuvanTM (sapropterin dihydrochloride) Tablets, the first specific drug therapy for the treatment of phenylketonuria (PKU). The product was launched in the U.S. immediately following the FDA approval. Kuvan is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4) responsive PKU and is to be used in conjunction with a Phe-restricted diet.
Biomarin And Igan Collaborate On Development Of Enzyme Therapy To Treat Iga Nephropathy
BioMarin and IGAN Biosciences initiated a program to develop an IgA protease for treating IgA nephropathy, an orphan designated kidney disorder with few treatment alternatives. In the United States, approximately 800 patients per year develop end stage renal disease caused by IgA nephropathy. An estimated 40,000 are affected by the disorder.
Biomarin Files Ind For Peg-Pal For The Treatment Of Pku
In November, BioMarin filed an investigational new drug application (IND) with the FDA for PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), formerly known as PhenylaseTM, for the treatment of phenylketonuria (PKU). The company expects to initiate a clinical study of PEG-PAL in PKU patients in the first quarter of 2008. Preclinical data has demonstrated that PEG-PAL administered subcutaneously once weekly to PKU mice resulted in a sustained decrease in blood phenylalanine (Phe) levels in a twelve week study and has also shown potent Phe level reductions in primates.
Maa For Sapropterin For Hyperphenylalaninemia Submitted To Emea
November-BioMarin partner Merck Serono, a division of Merck KGaA, Darmstadt, Germany, submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for sapropterin dihydrochloride (known as KuvanTM in the U.S.) an oral treatment for patients suffering from significant hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency. Acceptance of the MAA filing by the EMEA will trigger a $15 million milestone payment to BioMarin. Sapropterin has received orphan medicinal product designation in the European Union.
Exclusive Rights To Kuvan Data Licensed To Asubio Pharma
September-BioMarin has licensed exclusive rights to data and intellectual property contained in the Kuvan (sapropterin dihydrochloride) NDA to long-standing partner, Asubio Pharma Co., Ltd. (a subsidiary of Daiichi Sankyo). Asubio will use this data to supplement its current filing to the Japanese Ministry of Health, Labour and Welfare for approval of its BH4 product for the treatment of PKU in Japan. This new data greatly expands the clinical data set on treatment of PKU and is expected to accelerate the timing for the label extension of Asubio's current BH4 product. BioMarin will receive a milestone payment for approval as well as double-digit royalties on net sales of BH4 for PKU in Japan.
Biomarin Licenses Cystic Fibrosis Technology From Ucsf
August-BioMarin has licensed from the University of California, San Francisco, intellectual property covering compounds demonstrated to improve cystic fibrosis transmembrane conductance regulator (CFTR) protein functionality. Lead compounds are expected to undergo additional animal testing and optimization, with the goal of filing an IND in 2009. Cystic fibrosis is a well-defined and relatively widespread orphan disease affecting an estimated 30,000 patients in the U.S./40,000 patients outside the U.S.
Bla For Naglazyme Submitted To Japanese Ministry Of Health
AnGes MG, Inc. (AnGes), BioMarin's marketing and distribution partner in Japan submitted a Biologics License Application (BLA) for Naglazyme® (galsulfase) to the Japanese Ministry of Health, Labour and Welfare. If approved, Naglazyme will be the first drug treatment option to MPS VI patients in Japan. Naglazyme has obtained an orphan designation in Japan.
Kuvan Receives Priority Review Status From Fda
July-Priority review status is an FDA designation granted to drugs that, if approved, will provide a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious or life-threatening disease.
Biomarin Initiates Expanded Access Program For Kuvan In The U.S.
In June, the first patient initiated treatment in BioMarin's expanded access program for Kuvan. Under an expanded access program, the FDA allows early access to investigational drugs being developed to treat serious diseases for which there is no satisfactory alternative therapy. BioMarin will provide Kuvan at no charge to up to 500 U.S. patients throughout the duration of the program.
Biomarin Submits Nda For Kuvan For Pku
May-The NDA filing contains data evaluating Kuvan in approximately 650 human subjects in six clinical studies and represents BioMarin's largest and most comprehensive filing to date. The fully electronic NDA filing includes a comprehensive set of preclinical, clinical and manufacturing related data on Kuvan. If the FDA accepts the NDA and grants the request for priority review, the FDA is expected to take action on the application within six months of its submission. Kuvan has received the orphan drug designation, which allows for seven years of market exclusivity within the United States following FDA approval.
Biomarin Initiates Phase 2a Clinical Study Of 6r-Bh4 In Sickle Cell Disease
In May, the first patient initiated treatment in the Phase 2a clinical study of 6R-BH4 (sapropterin dihydrochloride) for the treatment of sickle cell disease (SCD). The company expects to announce data from this study in the first half of 2008. SCD is an orphan disease with 70,000 to 100,000 patients in the U.S. It is well- diagnosed at birth, but there is only one approved drug treatment option currently available which is used by a minority of patients due to toxicity problems. The Phase 2a multi-center, open-label study will evaluate the safety of oral 6R- BH4 administered in escalating doses in patients with sickle cell disease, as well as changes in physiological and biochemical markers of endothelial function which underlie some key aspects of SCD.
Results From Phase 2 Clinical Study Of 6r-Bh4 In Poorly Controlled Hypertension
February-Results demonstrated that there was no statistically significant or clinically meaningful effect of 6R-BH4 on any efficacy or safety parameter measured, relative to placebo, despite encouraging pre-clinical and clinical studies of 6R-BH4 in diseases with endothelial dysfunction. BioMarin will analyze this data in detail to better understand the results.
Positive Results From Phase 3 Diet Study Of Phenoptin For Pku
January-All pre-specified efficacy and safety endpoints of the double-blind, placebo-controlled Phase 3 diet study of Phenoptin(TM) (sapropterin dihydrochloride) were met. Treatment resulted in a significant increase in patients' phenylalanine tolerance as well as a reduction in their blood phenylalanine levels. In addition, the data showed that Phenoptin was well tolerated in younger PKU patients under dietary control.
Phase 2 Clinical Study Of 6r-Bh4 In Peripheral Arterial Disease Initiated
In January, the first patient initiated treatment in the Phase 2 clinical study of 6R-BH4 for the treatment of symptomatic peripheral arterial disease. The company expects to announce data from this study in the first half of fiscal year 2008. Peripheral arterial disease results from endothelial dysfunction and affects approximately eight million Americans, many of whom also suffer from intermittent claudication. The Phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study is designed to compare oral 6R-BH4 to placebo in subjects with intermittent claudication (IC) caused by peripheral arterial disease.
2006
Positive Results From Phase 3 Extension Study Of Phenoptin For Pku
December-Data has confirmed the long-term safety, tolerability and efficacy of Phenoptin to control blood Phe levels across a range of doses in PKU patients. The company is on track to file the NDA in the second quarter of 2007. A once daily dose regimen of Phenoptin was sufficient to maintain the reduction of blood Phe levels throughout a 24 hour period. The incidence and type of adverse events were comparable to that of the placebo group during the double-blind study and nearly all were mild or moderate in severity.
Aldurazyme Receives Marketing Approval In Japan
October-Japan's Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Aldurazyme® (laronidase), the first specific treatment for MPS I approved in Japan. Aldurazyme has been designated as an orphan drug in Japan.
Orapred Odt Launched By Alliant Pharmaceuticals
August-Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets), the first FDA-approved orally disintegrating tablet form of prednisolone, is now available in the United States. The Orapred product line, which includes Orapred ODT and Orapred® (prednisolone sodium phosphate oral solution) is marketed by Alliant Pharmaceuticals, Inc. Under the terms of the marketing agreement, BioMarin will receive milestone payments and royalties on Orapred products sales. BioMarin will retain commercial rights to the Orapred product line outside of North America.
Phase 2 Clinical Study Of 6r-Bh4 In Poorly Controlled Hypertension Initiated
In July, the first patient initiated treatment in the Phase 2 clinical study of 6R-BH4 for the treatment of poorly controlled hypertension. The company hopes to announce data from this study in early 2007 that will confirm results seen earlier in pilot clinical studies that demonstrated that oral administration of 6R-BH4 can reduce blood pressure in individuals who remain hypertensive despite treatment with other medications.
Biomarin And Alliant Pharmaceuticals Establish North American Licensing Agreement For Orapred
March-The licensing and acquisition agreement provides exclusive North American rights to Alliant for the Orapred® (prednisolone sodium phosphate oral solution) product line, including Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets). BioMarin will receive payments and royalties based on the product's approval, launch and level of sales. BioMarin will retain commercial rights outside of North America
Positive Results From Phase 3 Clinical Study Of Phenoptin For Pku
In March, positive results of a Phase 3, double-blind, placebo-controlled clinical study of Phenoptin™ (sapropterin dihydrochloride) confirmed that all pre-specified primary and secondary endpoints were met. Data demonstrates a statistically significant reduction at six weeks in blood phenylalanine (Phe) levels (p<0.0001) in patients receiving Phenoptin, compared with those receiving placebo.
Naglazyme Receives European Union Approval
January-The European Commission has granted marketing authorization for Naglazyme™ (galsulfase), the first treatment for MPS VI approved in the European Union. Naglazyme has been granted orphan drug status in the EU, which confers 10 years of market exclusivity. BioMarin will launch the product on a country-by-country basis.
Phenoptin For Pku Receives Fda Fast Track Designation
In January, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for Phenoptin™ (sapropterin dihydrochloride) for PKU, currently in Phase 3 clinical development.
The Fast Track program is designed to expedite the development and review process of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
Biomarin Establishes Commercial Operations In Europe
In anticipation of the pending European marketing approval for Naglazyme™, commercial operations have been established in Europe. BioMarin Europe Ltd., headquartered in London, with branch offices located in Spain, Switzerland and Italy, will be responsible for overseeing the sales and distribution of Naglazyme to the 25 member states of the European Union, Iceland and Norway.
2005
Biomarin Files New Drug Application For Orapred Odt
In August, BioMarin submitted a New Drug Application to the FDA for Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets), a new formulation of Orapred® (prednisolone sodium phosphate oral solution). Prednisolone is commonly used to reduce inflammation seen in numerous medical conditions including asthma, arthritis and cancer.
Orapred ODT may have the potential to provide individuals of all ages with a new formulation of prednisolone that is convenient and easy to administer
Biomarin Launches Naglazyme In The United States
June-Naglazyme® is the first drug therapy independently developed and commercialized by BioMarin. A team of U.S. based medical science liaisons will be responsible for providing support to infusion centers and physicians who administer Naglazyme.
Biomarin Receives Fda Approval For Naglazyme
May-Naglazyme®, represents BioMarin's first independently developed and commercialized drug and the first FDA-approved treatment for MPS VI. The drug was granted the orphan drug designation in the United States, which confers seven years of market exclusivity.
BioMarin Forms Strategic Alliance With Serono for the Development and Commercialization of Phenoptin and Phenylase
This agreement helps fund late-stage development of BioMarin's PKU and 6R-BH4 programs. The companies will equally share Phase 3 development costs of Phenoptin and Phenylase for PKU and 6R-BH4 for the treatment of cardiovascular indications. Additionally, Serono will provide BioMarin up to $232 million in milestone payments in exchange for ex-U.S. commercialization rights (excluding Japan).
BioMarin Acquires Rights to 6R-BH4 for the Treatment of Cardiovascular Indications
In May 2005, BioMarin announced a partnership with Daiichi Suntory Pharma Co., Ltd. that gave the company exclusive worldwide rights (excluding Japan) for the use of 6R-BH4 to treat cardiovascular indications. This was the second agreement reached with Daiichi; the first, which was reached in November 2004, pertained to intellectual property, preclinical and clinical data on 6R-BH4 for genetic disorders including PKU, and to manufacturing and supply of 6R-BH4.
BioMarin Initiates Phase 3 Clinical Study of Phenoptin for PKU
In April 2005, BioMarin initiated a Phase 3 clinical study designed to evaluate the safety and efficacy of Phenoptin for the treatment of PKU. Phenoptin has been designated an orphan drug in the United States and Europe and assigned Fast Track status in the United States.
2004
BioMarin Files Marketing Applications for Naglazyme in the United States and Europe
In June 2004, BioMarin announced positive results of the Phase 3 clinical trial of Naglayzme (galsulfase) for MPS VI, keeping the company on track to file license applications in both the United States and European Union by the close of the year.
BioMarin Advances Phenoptin for PKU into Phase 2 Development
In 2004, BioMarin advanced Phenoptin™ (sapropterin dihydrochloride), an investigational small-molecule oral therapeutic for the treatment of phenylketonuria (PKU), from IND filing into Phase 2 development by the close of the year.
2003
Aldurazyme for MPS I Approved and Launched in the Unites States and Europe
In April and June 2003, respectively, the FDA and European Commission (EC) granted marketing authorization for Aldurazyme--the first approved enzyme replacement therapy for the treatment of MPS I. Aldurazyme received FDA approval in just over five and a half years after the investigational new drug application (IND) was filed.
2000
BioMarin Initiates Clinical Trial of Naglazyme for MPS VI
At the onset of clinical development, Naglazyme® (galsulfase) for MPS VI had been granted orphan drug and fast track designations by the U.S. Food and Drug Administration (FDA). The company manufactured the investigational enzyme at its cGMP manufacturing facility located near the large-scale facility where it is being manufactured today.
1999
BioMarin Becomes a Publicly Traded Company (Nasdaq/SWX:BMRN)
In July 1999, BioMarin completed an initial public offering, raising $67.3 million. Since a large number of the early investors were based in Europe, the company was listed on the Swiss SWX Exchange in addition to the Nasdaq National Market
1998
BioMarin/Genzyme LLC Formed to Support the Development and Commercialization of Aldurazyme
In September 1998, BioMarin and Genzyme Corporation established BioMarin/Genzyme LLC, a 50/50 joint venture for the worldwide development and commercialization of Aldurazyme for MPS I. Pursuant to the agreement, BioMarin is responsible for manufacturing the product and Genzyme is responsible for its commercialization. All expenses and profits and are shared equally between the companies.
1997
BioMarin Initiates Clinical Trial of Aldurazyme for MPS I
In December 1997, BioMarin initiated the first clinical trial of Aldurazyme® (laronidase) for the treatment of mucopolysaccharidosis I (MPS I). Dr. Emil Kakkis, then a fellow at Harbor-UCLA Medical Center, was the study’s principal investigator and Dr. Elizabeth Neufeld, Professor and Chair of the Biological Chemistry Department at UCLA, was the advisor. Together, Dr. Neufeld and Dr. Kakkis discovered how to produce a recombinant form of alpha-L-iduronidase that was later evaluated in the clinic.
BioMarin Pharmaceutical Inc. Founded
With a $1.5 million investment from Glyko Biomedical Ltd., BioMarin was open for business. The company s mission was to leverage its proprietary enzyme technology to develop therapies for the treatment of numerous diseases and conditions including genetic diseases, and burn and wound care. By the close of the year, the company had raised an additional $11.3 million from private investors.