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2013

FDA Advisory Committee Recommends Approval for BioMarin's Vimizim™ for the Treatment of Patients with Morquio A Syndrome

In November, BioMarin announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) voted in favor of approval of Vimizim for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).

BioMarin Announces French ATU Granted for Vimizim™ for the Treatment of Morquio A Syndrome

In November, BioMarin announced that the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use, for patient sales of Vimizim for the treatment of Morquio A Syndrome. An ATU is the regulatory mechanism used by the ANSM to make non-approved drugs available to patients in France when a genuine public health need exists.

BioMarin Initiates Phase 3 Trial for BMN 673 for the Treatment of Metastatic gBRCA Breast Cancer

In October, BioMarin announced that it dosed the first patient in its Phase 3 program to evaluate BMN 673, its poly ADP-ribose polymerase (PARP) inhibitor, in the treatment of metastatic germline BRCA mutated breast cancer.

BioMarin Doses First Patient in Phase 1/2 Trial with BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease

In September, BioMarin announced that it dosed the first patient in the Phase 1/2 trial for BMN 190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. This is the first time that a patient with Batten disease has been treated with an enzyme replacement therapy in a clinical trial setting.

BioMarin Appoints Richard Ranieri as Senior Vice President, Human Resources and Corporate Affairs

In September, BioMarin announced the appointment of Richard Ranieri as Senior Vice President, Human Resources and Corporate Affairs.

BioMarin Provides BMN 673 Program Update

In July, BioMarin provided an update on its ongoing Phase 1/2 study for its poly ADP-ribose polymerase (PARP) inhibitor BMN 673 for the treatment of solid tumors.

BioMarin Initiates Phase 3 Trial for PEG-PAL for the Treatment of PKU

In June, BioMarin announced that it initiated the Phase 3 program for PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU).

FDA Accepts Vimizim BLA and Grants Priority Review Designation

In May, BioMarin announced that the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

Vimizim MAA Validated by the EMA

In May, BioMarin announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Vimizim. Validation of the MAA confirms that the submission is complete and starts the EMA's formal review process.

BioMarin Advances BMN-701 for Pompe Disease to Next Phase of Development

In March, BioMarin announced results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of late-onset Pompe disease.

BioMarin Licenses Factor VIII Gene Therapy Program for Hemophilia A From University College London and St. Jude Children's Research Hospital

In February, BioMarin announced that it had licensed a Factor VIII gene therapy program for hemophilia A from University College London (UCL) and St. Jude Children's Research Hospital.

BioMarin Announces Kuvan Significantly Improves Inattentiveness in Kuvan Responding PKU Patients

In February, BioMarin announced results from the PKU-016 ASCEND study, the largest randomized controlled trial evaluating neuropsychiatric outcomes in phenylketonuria (PKU) patients treated with the approved drug Kuvan (sapropterin dihydrochloride).

BioMarin Acquires Zacharon Pharmaceuticals

In January, BioMarin announced that it acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego, focused on developing small molecules targeting pathways of glycan and glycolipid metabolism.

2012

BioMarin Phase 3 Study of GALNS for the Treatment of MPS IVA Meets Primary Endpoint

In November, BioMarin announced that the pivotal Phase 3 study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg.

BioMarin Announces Decision to Start Phase 3 Program for PEG-PAL

In September, BioMarin announced preliminary results from the Phase 2 program of PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU) demonstrating long-term retention, tolerability and providing evidence of efficacy. Based on these results, the company expects to start a pivotal Phase 3 study in the second quarter of 2013, following an anticipated end of Phase 2 meeting with the FDA in the first quarter of 2013.

BioMarin Announces Phase 1 Results for BMN-111 for Achondroplasia

In September, BioMarin announced the completion of a Phase 1 study for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia.

BioMarin Names Jeff Ajer Senior Vice President, Chief Commercial Officer

In September, BioMarin announced that Jeff Ajer, the previous Vice President, Commercial Operations, the Americas, was promoted to Senior Vice President, Chief Commercial Officer.

BioMarin Appoints Dan Spiegelman as Executive Vice President and Chief Financial Officer

In May, BioMarin announced the appointment of Dan Spiegelman as Executive Vice President and Chief Financial Officer (CFO).

BioMarin Completes Enrollment for Phase 3 Trial for GALNS for the Treatment of MPS IVA

In March, BioMarin announced that enrollment was complete for the pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN-110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

BioMarin Initiates Phase 1 Trial for BMN-111 for the Treatment of Achondroplasia

In February, BioMarin announced the initiation of a Phase 1 study in healthy volunteers for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of achondroplasia.

2011

BioMarin Receives Positive Opinion from European Regulatory Authorities for Expanded Biologics Manufacturing Facility

In December, BioMarin announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval for the company's manufacturing facility expansion in Novato, CA.

BioMarin Announces BMN-190 for Late infantile neuronal ceroid lipofuscinosis (LINCL) - Form of Batten Disease

In December, at its R&D Day, BioMarin announced a new clinical program, BMN-190 for LINCL, one form of Batten disease.

BioMarin Announces Buy Back of Naglazyme Royalties From Adelaide Health Authority

In November, BioMarin completed the buy back of certain intellectual property from SA Pathology, a unit of the Central Adelaide Local Health Network located in Adelaide, Australia for an upfront payment of $81 million. The intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI, which expire between 2022 and 2023.

BioMarin Initiates Phase 2 Study for GALNS in Patients Under Five Years of Age With MPS IVA

In November, BioMarin initiated a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA).

BioMarin Announces FDA Approval of Expanded Biologics Manufacturing Facility

In November, BioMarin announced tthat it had received approval from the U.S. Food and Drug Administration (FDA) for its manufacturing facility expansion in Novato, CA.

BioMarin Initiates Phase 1 Trial for BMN 673 in Patients With Advanced Hematological Malignancies

In July, BioMarin initiated a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies.

BioMarin Agrees to Acquire Biologics Manufacturing Plant in Ireland from Pfizer

In July, BioMarin announced that it had entered into a definitive agreement to acquire a bulk biologics manufacturing plant from Pfizer, located in Shanbally, Cork, Ireland. The transaction was completed in August of 2011.

BioMarin Initiates Phase 3 Trial for Amifampridine Phosphate for the Treatment of LEMS

In June, BioMarin initiated a Phase 3 trial for amifampridine phosphate (3,4-diaminopyridine phosphate) for the treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS).

BioMarin Initiates Pivotal Phase 3 Trial for GALNS for the Treatment of MPS IVA

In February, BioMarin initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease

In January, BioMarin initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease.

BioMarin Initiates Phase 1/2 Trial for BMN 673 for the Treatment of Genetically-Defined Cancers

In January, BioMarin initiated a Phase 1/2 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor in development for the treatment of genetically-defined cancers.

2010

BioMarin Announces Program for BMN-111 for the Treatment of Achondroplasia

In October 2010, BioMarin announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. BioMarin plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012.

BioMarin Appoints William Young and Kenneth Bate to Its Board of Directors

In September 2010, BioMarin announced the appointment of William D. Young and Kenneth M. Bate to its Board of Directors.

BioMarin Receives Orphan Drug Designation from the FDA for BMN-701 for the Treatment of Pompe Disease

In August 2010, BioMarin received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease.

BioMarin Acquires ZyStor Therapeutics, Inc.

In August 2010, BioMarin acquired ZyStor Therapeutics, Inc., a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders.

BioMarin Initiates Phase 3b Study to Evaluate the Effects of Kuvan on Neurophychiatric Symptoms in Subjects with PKU

In August 2010, BioMarin announced that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU).

BioMarin Halts Development of BMN-195 for Duchenne Muscular Dystrophy

In August 2010, BioMarin completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial results were not positive and the BMN 195 program was discontinued due to pharmaceutical and pharmacokinetic challenges.

BioMarin Reports Positive Results for Phase I/II Trial for BMN-110 for MPS IVA

In April 2010, BioMarin announced positive results for the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome.

BioMarin Launches Firdapse in the European Union

In April 2010 BioMarin  launched Firdapse(TM) (3,4-diaminopyridine) in the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

BioMarin Acquires LEAD Therapeutics

In February 2010, BioMarin entered into a stock purchase agreement to acquire 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 for the treatment of patients with rare, genetically defined cancers.

BioMarin Initiates Phase 1 Clinical Study of BMN-195 for Duchenne Muscular Dystrophy

In January 2010, BioMarin announced that the first subject had initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD).

Firdapse Receives Marketing Approval in the EU for LEMS

In January 2010, BioMarin announced today that the European Commission had granted marketing approval for Firdapse (3,4-diaminopyridine (amifampridine phosphate)), for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

 

2009

FDA Grants Orphan Drug Designation for 3, 4-DAP for LEMS

In November 2009, the Food and Drug Administration (FDA) granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS in Europe.

BioMarin Acquires Huxley Pharmaceuticals, Inc.

In October 2009, the company acquired Huxley Pharmaceuticals, Inc., which has rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). The company expects to launch the product in Europe in the first quarter of 2010, and is evaluating a development strategy for amifampridine phosphate in LEMS in the U.S. and for other indications in the U.S. and Europe.

BioMarin Initiates Phase II Clinical Study of PEG-PAL in PKU

In September 2009, the company initiated the Phase II clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Initial top-line results are expected in mid-2010. The primary objective is to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU. The secondary objectives are to evaluate the safety and tolerability, immune response and steady state pharmacokinetics of subcutaneous injections of multiple dose levels of PEG-PAL.

Kuvan Receives Priority Review Status From Health Canada

In June 2009, Kuvan® (sapropterin dihydrochloride) received priority review status from Health Canada. Priority review provides for a shortened submission review of 180 days versus the standard twelve months. BioMarin plans to file a marketing application for Kuvan in Canada in the third quarter of 2009, and with priority review status, a decision for marketing approval is expected in the first half of 2010.

BioMarin Initiates Phase I/II Clinical Trial for GALNS for Morquio A Syndrome (MPS IVA)

In April 2009, BioMarin initiated a Phase 1/2 clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies.

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® (sapropterin dihydrochloride) Tablets 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®, 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.

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2008

Kuvan Approved By EMEA for Use in Europe

In December, the scientific committee of the European Medicines Agency (EMEA) approved Kuvan® (sapropterin dihydrochloride) Tablets 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 function and other parameters in affected patients.

Positive Results from Study of 6R-BH4 in Sickle Cell Disease

In October, 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's partner Merck Serono received a positive opinion for Kuvan® (sapropterin dihydrochloride) Tablets 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.

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).

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.

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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.

In December, approximately three years after filing the IND, BioMarin received FDA approval for Kuvan® (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

In December, 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 Phenylase™, 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

In 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 Kuvan® 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

In September, BioMarin has licensed exclusive rights to data and intellectual property contained in the Kuvan (sapropterin dihydrochloride) Tablets 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.

BLA For Naglazyme Submitted To Japanese Ministry Of Health

In August, 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

In 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

In 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). 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

In 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.

Positive Results From Phase 3 Diet Study Of Phenoptin For PKU

In January, all pre-specified efficacy and safety endpoints of the double-blind, placebo-controlled Phase 3 diet study of Phenoptin™ (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.

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2006

Positive Results From Phase 3 Extension Study Of Phenoptin For PKU

In 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

In 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

In 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

In 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

In 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 January, 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.

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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


In 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

In 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

In May, BioMarin and Serono finalize an egreement to help 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, 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, 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.

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2004

BioMarin Files Marketing Applications for Naglazyme in the United States and Europe

In June, 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 February, 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.

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2003

Aldurazyme for MPS I Approved and Launched in the Unites States and Europe

In April and June, 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.

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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.

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1999

BioMarin Becomes a Publicly Traded Company (Nasdaq/SWX:BMRN)

In July, 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.

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1998

BioMarin/Genzyme LLC Formed to Support the Development and Commercialization of Aldurazyme

In September, 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.

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1997

BioMarin Initiates Clinical Trial of Aldurazyme for MPS I

In December, 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

In March, 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.

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