Editor's Note: Only responses from families of MPS and
important press releases are posted here. All medical expert
responses are posted on the Medical Expert
Page. Last updated: March 21, 2001.
Editor's Note:This is an excerpt from a press release from Genzyme dated February 16, 2001.
Genzyme and joint venture partner BioMarin Pharmaceutical Inc. are actively enrolling MPS I patients in the pivotal Phase 3 trial of Aldurazyme? (laronidase) enzyme replacement therapy. Enrollment in the six-month trial is expected to conclude by the end of the first quarter, and Genzyme and BioMarin anticipate filing for U.S. marketing approval by the end of this year. Approval is anticipated by mid-2002.
Genzyme and Biomarin Announce Phase 3 Trial of Aldurazyme™ for MPS-I
Date: November 22, 2000
Genzyme General (Nasdaq: GENZ) and BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX New Market: BMRN) announced on November 22 that they are initiating a pivotal Phase 3 trial of Aldurazyme™ (laronidase), and expect to treat the first patient in the trial by early December. The product, recombinant alpha-L-iduronidase, is an enzyme replacement therapy for patients with MPS-I, also known as Hurler, Hurler-Scheie and Scheie diseases. Genzyme and BioMarin have received Orphan Drug designation and Fast-Track status for Aldurazyme from the U.S. Food and Drug Administration.
In an initial clinical trial, Aldurazyme has shown safety and promising clinical results. If the Phase 3 trial confirms the initial safety and efficacy profile of the drug, the companies will submit for marketing approval in the U.S., Europe, Canada and other countries in order to provide Aldurazyme therapy to the thousands of patients who currently suffer from this life-threatening disease.
In 1998, Genzyme and BioMarin formed BioMarin/Genzyme LLC to develop and commercialize Aldurazyme throughout the world.
BioMarin specializes in the development and commercialization of therapeutic enzyme products. Since inception in 1997, BioMarin has applied its proprietary enzyme technology to develop products for lysosomal storage diseases and for the treatment of serious burns. Glyko, Inc., a BioMarin subsidiary, provides analytical and diagnostic products and services in the area of carbohydrate biology.
Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has three therapeutic products on the products in development focused on the treatment of genetic disorders. Genzyme General is a
division of the biotechnology company Genzyme Corporation.
This press release contains forward-looking statements, including statements regarding: the expected timing of dosing patients in a Phase 3 trial; the clinical potential of Aldurazyme; anticipated submissions for regulatory approvals of Aldurazyme™ in the U.S. and Europe; and the potential market introduction of Aldurazyme. Actual results may differ materially from those contained in these forward-looking statements as a result of a number of factors, including: the
actual safety and efficacy of Aldurazyme; the enrollment rates for the Phase 3 clinical trial; the results of clinical trials; the ability to manufacture sufficient amounts of Aldurazyme for development and commercialization activities; the timing and content of submissions to and decisions made by the FDA, the European Agency for the Evaluation of Medicine Products and other regulatory authorities; the continued funding of the joint venture; decisions made by physicians and third-party
payers regarding the use of, and reimbursement for, Aldurazyme; our ability to obtain and maintain adequate patent and other
proprietary rights protection for Aldurazyme; the scope, validity and enforceability of patents and other proprietary rights held by third parties related to therapies for MPS-1 and the actual impact of such patents and other rights on our ability to commercialize Aldurazyme; the competitive environment for therapies for MPS-1; and the risks and uncertainties described in Genzyme’s and BioMarin’s reports filed with the Securities and Exchange Commission under the Securities
Exchange Act of 1934, as amended, including Exhibit 99.2 to Genzyme’s Annual Report on Form 10-K for the year ended December 31, 1999, as amended. GENZ stock is a series of common
stock of Genzyme Corporation. Therefore, holders of GENZ stock are subject to all of the risks and uncertainties described in the aforementioned reports filed by Genzyme Corporation.
Editor's Note:This is an excerpt from a press release from Genzyme dated April 20, 2000.
Genzyme General and its joint venture partner BioMarin Pharmaceutical Inc. continue to progress toward initiating the confirmatory phase III clinical trial of Aldurazyme enzyme replacement therapy for patients with MPS I. The companies expect to begin enrolling patients later this year. In preparation for the start of the trial, a decision was made by the joint venture to use BioMarin’s new Galli Drive facility in Novato, California, to manufacture Aldurazyme for the confirmatory trial, as this will be the same material used for commercial launch. Production of Aldurazyme at BioMarin’s Carson Street clinical manufacturing facility in Torrance, California, will be suspended at the end of this month
BioMarin and Genzyme to Conduct Confirmatory Trial of Treatment for MPS-I Prior to Filing for FDA Approval
Date: November 17, 1999
BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX New Market: BMRN) and Genzyme General (Nasdaq: GENZ) today announced that they will conduct a confirmatory phase III clinical trial of Aldurazyme™enzyme replacement therapy for
mucopolysaccharidosis-I (MPS-I) prior to seeking marketing approval from the FDA. Following a positive meeting with the FDA yesterday, the companies and the agency agreed to build on the encouraging results of the initial clinical trial of Aldurazyme with additional data supporting the therapeutic benefit of the product.
BioMarin and Genzyme now expect to file a biologics license application (BLA) for Aldurazyme in the United States by the end of next year, with market introduction coming in 2001. Regulatory filing in Europe will quickly follow the U.S. filing and will be based on results from the confirmatory phase III trial, which will involve centers in both the United States and Europe. The FDA confirmedthat Aldurazyme maintains its Fast-Track Status.
"MPS-I is a chronic, life-threatening disease, for which no effective treatment options exist. Since inception, the goal of BioMarin's program for Aldurazyme has been to expeditiously meet the needs of MPS-I patients," said Grant W. Denison, Jr., chairman and chief executive officer of BioMarin. "We are very pleased with the clinical results we've collected to date, which have exceeded the expectations we had when we began the trial. Building on that foundation, we will continue our work with the FDA on this confirmatory trial."
Henri A. Termeer, chairman and chief executive officer of Genzyme Corporation, said: "Since we began this program, we have proceeded with a great sense of urgency, driven by our compassion for MPS-I patients. While today's decision extends the timing for the U.S. commercial launch of Aldurazyme, we expect that a confirmatory trial will strengthen our ability to transform the standard of care for MPS-I. By expanding the pool of available clinical data on Aldurazyme, we expect to accelerate acceptance of the product within the health care community. We are confident about Aldurazyme's potential, and we are working very closely with the FDA to make this product available as soon as possible."
In the initial clinical trial, Aldurazyme was administered on a weekly basis for 52 weeks to 10 patients with MPS-I ranging in age from 5 to 22 years. Patients met both of the primary endpoints prospectively defined in the clinical protocol. Specifically, patients experienced a normalization of liver size and showed a decrease in the excretion of urinary glycosaminoglycans, the carbohydrate substances that accumulate in patients with MPS-I. The patients who participated in the initial trial continue to receive treatment and are now approaching two years on therapy.
The confirmatory trial of Aldurazyme will be designed to collect additional data to supplement the positive results obtained in the initial clinical trial of the product. BioMarin and Genzyme are working with the FDA to finalize a protocol for the confirmatory trial. The companies expect trial enrollment to proceed quickly, given the number of MPS-I patients that they have identified through their physician and patient outreach. As in the initial trial of Aldurazyme, patients in the confirmatory trial are expected to represent the full spectrum of severity of MPS-I, including Hurler, Hurler-Scheie, and Scheie syndromes.
As part of the discussions with the FDA, the companies and the agency agreed that it is important to determine a protocol for treating the most severely ill MPS-I patients. BioMarin and Genzyme will work with the FDA to develop this protocol to allow
early access to treatment for terminally ill patients. The data from this clinical study is not required for the BLA filing.
BioMarin and Genzyme General formed a joint venture in
September 1998 for the development and commercialization of
Aldurazyme for the treatment of MPS-I.
BioMarin Pharmaceutical Inc. specializes in the discovery, development and commercialization of carbohydrate enzyme therapeutics. Since inception in 1997, BioMarin has applied its proprietary enzyme technology to the development of products in five therapeutic areas: genetic diseases, burn debridement, fungal infections, male pro-fertility, and inflammation (psoriasis). Glyko, Inc., a BioMarin subsidiary, provides analytical and diagnostic services in the area of carbohydrate biology.
Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General currently has three therapeutic products on the market and a strong pipeline of therapeutic products in development focused on the treatment of
rare genetic disorders. A division of the biotechnology company Genzyme Corporation, Genzyme General has its own common stock intended to reflect its value and track its economic performance.
This press release contains forward looking statements regarding our expectations for filing for the biologics license application and market introduction for Aldurazyme; the filing of a regulatory dossier in Europe for Aldurazyme; the locations of, and the protocol for, and the patient population for the confirmatory clinical trial of Aldurazyme; our ability to obtain a compassionate use protocol in that clinical trial; our ability to transform the standard of care for MPS-I; our ability to accelerate market acceptance for Aldurazyme; and the clinical potential of Aldurazyme. Actual results may differ materially from those contained in these forward looking statements as a result of a number of factors, including: the timing and content of submissions to and decisions made by the FDA, the European Commission and other regulatory authorities; enrollment rates and results of clinical trials; the ability to manufacture sufficient quantities of Aldurazyme for development and commercialization activities; the continued funding of the joint venture; decisions made by physicians and third party payers regarding the use of, and reimbursement for, Aldurazyme; and the actual clinical potential of Aldurazyme.
Aldurazyme™ is a trademark of BioMarin/Genzyme LLC. All rights reserved.
Genzyme and BioMarin Report that Patients in Pivotal Trial of
a-L-iduronidase Show Marked Improvement
Data Presented at American Society of Human Genetics Meeting
CAMBRIDGE, Mass. and NOVATO, Calif., October 30, 1998.
MPS-I results from a genetic deficiency in the production of a
-L-iduronidase, a key enzyme required for the progressive
breakdown of complex carbohydrates found in all cells of the
body. Data from the trial demonstrated that a -L-iduronidase
replacement therapy reduced clinical measurements of the disease
and improved patients’ ability to perform normal functions
of daily life.
"The trial results are very encouraging," said Emil D.
Kakkis, M.D., Ph.D., the study’s principal investigator, who
presented the findings. "This clinical trial culminates 30
years of research and marks the first time enzyme replacement
therapy has been used effectively with MPS-I patients." Dr.
Kakkis is assistant professor, Division of Medical Genetics,
Department of Pediatrics, Harbor-UCLA Medical Center. He also
recently became president of BioMarin Genetics, a division of
BioMarin Pharmaceutical.
Trial Design and Results
The clinical trial, which was initiated in December 1997 and
conducted at the Harbor-UCLA Research and Education Institute in
Torrance, California, in collaboration with four other leading
medical centers, enrolled ten patients in a 26-week treatment
protocol. MPS-I strikes children, with initial diagnosis
typically at 2 or 3 years of age. The ages of those enrolled in
the trial ranged from 5-22 years.
Patients were evaluated before the trial began to establish a
clinical baseline. They were then given a weekly intravenous
infusion of 125,000 units (~0.5mg) of a -L-iduronidase per
kilogram of body weight. Patients were subsequently examined
after weeks 6, 12 and 26. Both quantitative data and
clinical/patient self-assessment data were collected and
analyzed.
Quantitative MRI studies demonstrated that treatment with a
-L-iduronidase was associated with a rapid 20 percent or more
reduction in liver size after 6-12 weekly doses in nearly all
patients, and that liver size returned to normal in the majority
of patients by 26 weeks. Patients also showed a decrease in the
excretion of urinary glycosaminoglycans, indicating the success
of a -L-iduronidase in breaking down these complex carbohydrate
materials, which otherwise will accumulate in patients with
MPS-I. Urinary glycosaminoglycan levels typically dropped by the
third or fourth dose of the enzyme, and all patients achieved a
60 percent or better reduction within 12 weeks.
In addition, patients reported improvements in physical abilities
and quality of life. Joint pain was reduced and mobility
improved, allowing patients to do more activities that had been
difficult before, such as walk, run, swim, hang from monkey bars,
or swing a bat. Patients also had substantial improvements in
endurance and decreased fatigue. Difficulties in breathing were
much improved and contributed to the improved endurance. For
several patients, limited half days at school were extended to
full days. The severe headaches with occasional vomiting that
plagued some patients up to several times a week prior to
treatment, were resolved after several weeks of therapy. Hearing
and vision improved in certain patients.
Adverse events such as hives were noted in a minority of patients
but were manageable and did not significantly affect the
patients’ health. All patients who were enrolled in the
trial continue to receive therapy and will be evaluated at 12 and
18 months following the initiation of treatment.
"From its inception, BioMarin has been dedicated to
developing therapies which have the potential to change the
practice of medicine and improve patients’ lives," said
Grant W. Denison, Jr., chairman and chief executive officer of
BioMarin. "Today’s trial results represent a step
toward realizing that potential, showing that this treatment can
have a major positive impact on the lives of MPS-I patients. We
look forward to continuing the development of therapies which
address unserved medical conditions."
Henri A. Termeer, chairman and chief executive officer of Genzyme
Corp., said: "Genzyme has a deep commitment to helping
patients with rare genetic disorders such as MPS-I. We are very
excited by the potential this therapy has shown to improve
patients’ lives. Through our joint venture with BioMarin, we
will move forward immediately with our program to obtain
regulatory approval for this treatment, which we hope to make
available to patients very rapidly."
Genzyme and BioMarin Joint Venture
Alpha-L-iduronidase is being developed and commercialized through
a joint venture of Genzyme and BioMarin. Genzyme and BioMarin
expect to submit the data from this clinical trial in a Biologics
License Application (BLA) for a -L-iduronidase, which the
companies intend to file with the U.S. Food and Drug
Administration (FDA) during the first quarter of 1999. The FDA
has designated a -L-iduronidase a fast-track product. BioMarin
received orphan drug designation for the enzyme in September
1997, giving the product market exclusivity for seven years
following FDA approval.
Background on MPS-I MPS-I encompasses three
syndromes—Hurler, Hurler-Scheie, and Scheie
syndromes—that define ranges in a spectrum of severity. All
types of MPS-I are caused by a deficiency in an active enzyme, a
-L-iduronidase, which results in a build-up of carbohydrate
materials called glycosaminoglycans in all tissues of the body.
This build-up of stored material leads to cell, tissue and organ
dysfunction. The debilitating effects of MPS-I lead to early
death--often before the age of ten. Symptoms can include
enlargement of the liver and spleen, joint pain and immobility,
skeletal deformity, vision impairment, stunted growth, hearing
loss, obstruction of airways, severe headaches and
cardiomyopathy.
Approximately 2,000-3,000 patients in the developed world have
been diagnosed with MPS-I. As with other lysosomal storage
diseases, there are believed to be additional undiagnosed
patients. There is currently no known effective treatment for
MPS-I.
Genzyme General develops and markets therapeutic and surgical
products and diagnostic products and services. A division of the
biotechnology company Genzyme Corp., Genzyme General has its own
common stock intended to reflect its value and track its
performance.
BioMarin Pharmaceutical, Inc. is a privately held
biopharmaceutical company specializing in the discovery,
development and commercialization of carbohydrate enzyme
therapeutics. Since inception in 1997, BioMarin has applied its
proprietary enzyme technology to the development of products in
five therapeutic areas: genetic diseases, burn and wound care,
fungal infections, pro-fertility products and inflammation
(psoriasis). With its recent acquisition of Glyko, Inc., BioMarin
added analytical and diagnostic products and services in the area
of carbohydrate biology.
This press release contains forward-looking statements about
potential therapeutic uses of a-L-iduronidase, the potential
market exclusivity of a-L-iduronidase, the size of the potential
market for a-L-iduronidase, the ability of the current clinical
trial of a-L-iduronidase to serve as a pivotal trial, and the
expected filing dates for a BLA.
Results may differ materially depending on the therapeutic effect
of a-L-iduronidase in commercial applications, the accuracy of
information regarding the number of patients suffering from
MPS-I, and decisions made by the FDA.
Genzyme’s releases are available at Genzyme and on the
company’s fax-on-demand service at 1-800-436-1443 in the
U.S. or 1-201-521-1080 elsewhere.
Editor's Note: This was copied with permission from the
Holland family from the University of Minnesota's MPS Discussion
Forum. With 3 children with Hurler Scheie Syndrome, they have
vast experience with this disorder. Their oldest child, Spencer,
had the chance to participate in the Enzyme Replacement Therapy.
Amy Holland (steve_amyholland@msn.com) February 12, 1999 (09:57)
Dear All,
Many parents have expressed an interest in Spencer's progress in
regards to the clinical trial for ERT. If you are not familiar
with our family, we have three children with hurler-scheie
syndrome;Spencer(9), Maddie(7), and Laynie(5). Spencer began
receiving enzyme treatments almost one year ago, as part of a
clinical trial with Dr. Emil Kakkis. I take Spencer one day each
week to a hospital that is an hour away and he receives an IV
infusion of the Enzyme. The whole process takes about 4-5 hours
to complete and Spencer now has a port-a-cath, which has made it
much easier to start an IV.
We are now very used to the routine of ERT. I have many friends
and relatives who pick up the girls from school each Tuesday
while I stay with Spencer. Spencer has never once shown even a
twinge of anxiety over receiving an IV treatment each week, he
still skips all the way to the treatment room. This is partly
because we use our time to watch movies, do crafts, and play
Nintendo, and partly because we have made so many friends at the
hospital who make each Tuesday something special. For those of
you who are considering ERT when it becomes available for your
child(I refuse to use the word "if")you can make this a
positive experience for your family, not one to be feared and
dreaded each week. There are several ways (Numby Stuff, EMLA
creme, or cold spray) to help with the pain of the IV start and
relieve emotional anxiety.
We have seen improvement in Spencer. This has been made evident
by Spencer's participation on a basketball team. We can clearly
see the changes in the stiffness of his joints when we watch him
play. He would not have been able to play a year ago because of
fatigue. He now keeps up with the other nine-year olds.
I know that there is concern over whether or not the Enzyme
crosses the blood brain barrier or can stop nuerologic
deterieration. I can not answer this question with scientific
information. I can only tell you what I have seen in Spencer. A
year ago, we were quite concerned about Spencer's IQ based on
testing done at the University of Minnesota. Although Spencer is
attending a private program for learning disabled children, he
continues to function above his IQ and has made extraordinary
gains in learning this year. We were concerned about his ability
to learn new things, but we have recently stopped worrying about
this. I am unsure whether or not this is an improvement because
of the ERT, but we were told at the U of M to expect a downward
trend in nuerologic function and that has not happened, in fact,
just the opposite.
We continue to see changes in our sweet little girls that show us
the ravages of MPS, but we have seen no further progression of
the disease in Spencer. His appearance is subtley changing and he
is showing less and less of the facial appearance of MPS. His
headaches have stopped and he is healthy and strong. He feels
that he is no different from any other kid and he is beginning to
feel more like that to me, too.
We are in a very difficult situation while we wait on FDA
approval. Our girls continue to decline while our little boy
continues to improve. Because of the delays with approval and the
changes we have seen in our seven year old, we too have been
forced into making the decision to do a Bone Marrow transplant
orwait for ERT to become available. Nothing about this is easy.
Please pray for quick FDA approval and availability of the Enzyme
for each of the MPS disorders,
Amy Holland
Recent Filings: May
1999 (S-1 Filing)
More filings for BMRN available from EDGAR Online | Access
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May 4, 1999
BIOMARIN PHARMACEUTICAL INC (BMRN)
S-1 Filing (SEC form S1)
BioMarin Pharmaceutical Inc. is a leading developer of
carbohydrate enzyme therapies for debilitating, life-threatening,
chronic genetic disorders and other diseases and conditions. In
October 1998, we completed the primary evaluation for the pivotal
clinical trial of our lead enzyme replacement product candidate,
BM101, for the treatment of mucopolysaccharidosis-I or MPS- I.
Based on the data from that trial, we intend to complete the
filing of a BLA with the FDA in the second half of 1999. We have
a joint venture with Genzyme Corporation for the worldwide
development and commercialization of BM101.
MPS-I is a life-threatening genetic disorder caused by the
lack of a sufficient quantity of the enzyme
(alpha)-L-iduronidase, which affects about 3,400 patients in
developed countries, including approximately 1,000 in the United
States and Canada. Patients with MPS-I have multiple debilitating
symptoms resulting from the buildup of carbohydrates in all
tissues in the body. These symptoms include delayed physical and
mental growth, enlarged livers and spleens, skeletal and joint
deformities, airway obstruction, heart disease and impaired
hearing and vision. If untreated, most children diagnosed with
MPS-I will die from complications associated with the disease
before adulthood.
BM101 is a specific form of (alpha)-L-iduronidase that is
intended to replace a deficiency of (alpha)-L-iduronidase in
MPS-I patients. In October 1998, we completed the primary
evaluation period for our pivotal clinical trial for BM101. This
clinical trial treated ten patients with MPS-I for a period of
six months at five medical centers in the United States. BM101
met the primary endpoints in its pivotal trial, reducing liver or
spleen sizes in eight of ten patients and lowering urinary
carbohydrate levels in all ten patients. In addition, various
secondary endpoints were reached in each of the patients. We
received notice from the FDA that our BLA will receive fast track
designation for the treatment of the more severe forms of MPS-I,
which account for approximately 60% of all cases. The FDA has
granted BM101 an orphan drug designation giving us exclusive
rights to market BM101 to treat MPS-I for seven years from the
date of FDA approval if BM101 is the first (alpha)-L-iduronidase
drug to be approved by the FDA for the treatment of MPS-I.
In September 1998, we established a joint venture with Genzyme
for the worldwide development and commercialization of BM101 for
the treatment of MPS- I. Our responsibilities within the joint
venture include obtaining U.S. regulatory approvals as well as
manufacturing and process development. Genzyme is responsible for
obtaining international regulatory approvals, worldwide sales and
marketing as well as pricing and reimbursement. We will share
expenses and profits from the joint venture equally with Genzyme.
Genzyme invested $8.0 million upon signing the agreement and has
agreed to purchase $10.0 million of common stock at the initial
public offering price in a private placement concurrent with this
offering. Genzyme has committed to pay us an additional $12.1
million upon approval of the BLA for BM101.
We are also developing enzyme replacement therapies for other
life-threatening genetic diseases. There are nine additional MPS
disorders caused by single enzyme deficiencies. We are developing
BM102 for the treatment of MPS-VI. We received an orphan drug
designation for BM102 to treat MPS-VI and intend to file an IND
for the use of BM102 to treat MPS-VI in the fourth quarter of
1999. We are also developing carbohydrate enzymes intended to
improve burn debridement and act as anti-fungals. Through a
wholly-owned subsidiary, we provide carbohydrate analysis to
research institutions and commercial laboratories.
Our strategy is to: (1) focus on drug candidates with known
biology and low technical risk, (2) target products that address
life-threatening conditions that may be developed and approved
quickly, (3) pursue well-defined, niche markets, (4) develop a
direct sales and marketing organization for select markets and
(5) enhance enzymatic expertise through our wholly-owned
subsidiary, Glyko, Inc.
Recent Filings: May
1999 (S-1 Filing)
More filings for BMRN available from EDGAR Online
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Editor's Note: The following is an excerpt from a
recent Genzyme Press Release.
Genzyme General Posts Second-Quarter EPS of $.46
Date: July 22, 1999
Therapeutics Clinical Development Programs
Genzyme and BioMarin Pharmaceutical Inc. are jointly preparing
a biologics license application (BLA) for alpha-L-iduronidase,
which is being developed as a treatment for patients with MPS I.
The companies are developing a protocol for a post-approval
clinical study, which has been requested by the Food and Drug
Administration. Post-approval—or phase IV—studies are
often required for products that are granted accelerated review.
As a result, the rolling filing of the BLA is now expected to
begin late in the third quarter, with product launch expected
mid-year 2000.
Editor's Note: The following is from a
recent Genzyme Press Release.
Genzyme and Biomarin Announce Phase 3 Trial of Aldurazyme™ for MPS-I
November 22, 2000
Genzyme General (Nasdaq: GENZ) and BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX New Market: BMRN) announced on November 22 that they are initiating a pivotal Phase 3 trial of Aldurazyme™ (laronidase), and expect to treat the first patient in the trial by early December. The product, recombinant alpha-L-iduronidase, is an enzyme replacement therapy for patients with MPS-I, also known as Hurler, Hurler-Scheie and Scheie diseases. Genzyme and BioMarin have received Orphan Drug designation and Fast-Track status for Aldurazyme from the U.S. Food and Drug Administration.
In an initial clinical trial, Aldurazyme has shown safety and promising clinical results. If the Phase 3 trial confirms the initial safety and efficacy profile of the drug, the companies will submit for marketing approval in the U.S., Europe, Canada and other countries in order to provide Aldurazyme therapy to the thousands of patients who currently suffer from this life-threatening disease.
In 1998, Genzyme and BioMarin formed BioMarin/Genzyme LLC to develop and commercialize Aldurazyme throughout the world.
BioMarin specializes in the development and commercialization of therapeutic enzyme products. Since inception in 1997, BioMarin has applied its proprietary enzyme technology to develop products for lysosomal storage diseases and for the treatment of serious burns. Glyko, Inc., a BioMarin subsidiary, provides analytical and diagnostic products and services in the area of carbohydrate biology.
Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has three therapeutic products on the market and a strong pipeline of products in development focused on the treatment of genetic disorders. Genzyme General is a division of the biotechnology company Genzyme Corporation.
This press release contains forward-looking statements, including statements regarding: the expected timing of dosing patients in a Phase 3 trial; the clinical potential of Aldurazyme; anticipated submissions for regulatory approvals of Aldurazyme™ in the U.S. and Europe; and the potential market introduction of Aldurazyme. Actual results may differ materially from those contained in these forward-looking statements as a result of a number of factors, including: the actual safety and efficacy of Aldurazyme; the enrollment rates for the Phase 3 clinical trial; the results of clinical trials; the ability to manufacture sufficient amounts of Aldurazyme for development and commercialization activities; the timing and content of submissions to and decisions made by the FDA, the European Agency for the Evaluation of Medicine Products and other regulatory authorities; the continued funding of the joint venture; decisions made by physicians and third-party payers regarding the use of!
, and reimbursement for, Aldurazyme; our ability to obtain and maintain adequate patent and other proprietary rights protection for Aldurazyme; the scope, validity and enforceability of patents and other proprietary rights held by third parties related to therapies for MPS-1 and the actual impact of such patents and other rights on our ability to commercialize Aldurazyme; the competitive environment for therapies for MPS-1; and the risks and uncertainties described in Genzyme’s and BioMarin’s reports filed with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including Exhibit 99.2 to Genzyme’s Annual Report on Form 10-K for the year ended December 31, 1999, as amended. GENZ stock is a series of common stock of Genzyme Corporation. Therefore, holders of GENZ stock are subject to all of the risks and uncertainties described in the aforementioned reports filed by Genzyme Corporation.
Aldurazyme™ is a trademark of BioMarin/Genzyme LLC. All rights reserved.
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