Strengthens and Expands BioMarin's Leadership
Position Serving PKU Patients with Rights to Both Kuvan®
(sapropterin dihydrochloride) and Pegvaliase Beyond North
America
2016 Full-year Guidance for Kuvan Expected to be
Between $320M-$350M including $70M-$80M in New Revenue from ROW
Territories
Starting January 1, 2016 BioMarin will
Commercialize and Record Sales of Kuvan in an Additional 55
Countries Already Served by BioMarin
Investor Call to be Held Today, October 1 at
8:30am ET/5:30am PT
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), today announced that it
has acquired all global rights to Kuvan® (sapropterin
dihydrochloride) and pegvaliase from Merck Serono (Merck). Under
the terms of the agreement, BioMarin will provide Merck with an
upfront payment of €340 million. An additional €60 million in
milestones will be paid to Merck if combined sales of Kuvan and
pegvaliase reach undisclosed cumulative sales thresholds. In
addition, €125 million will be paid to Merck for regulatory
milestones related to pegvaliase. Previously, BioMarin had
exclusive rights to Kuvan in the United States and Canada and to
pegvaliase in the United States and Japan. Under the terms of the
transaction, BioMarin will now have exclusive worldwide rights to
Kuvan and pegvaliase with the exception of Kuvan in Japan. Approved
in 2007 in the U.S., Kuvan is a commercialized product for the
treatment of patients with phenylketonuria (PKU). Pegvaliase is
currently in registration-enabling pivotal studies as a potential
therapeutic option for adult patients with phenylketonuria. With
the potential approval of pegvaliase, the two products combined
will expand and globalize BioMarin's leadership position by
offering a wider range of treatment options to patients worldwide
with PKU.
In 2005, Merck acquired from BioMarin the exclusive rights to
Kuvan and the option to develop pegvaliase in markets outside of
the U.S. and Japan. By regaining these rights to both products,
BioMarin has the opportunity to expand its commercial efforts
across the Company's global territories. Kuvan is currently
sold by Merck in many countries where BioMarin has a commercial
presence for both Naglazyme® (galsulfase) and Vimizim® (elosulfase
alfa). Kuvan has Orphan Drug exclusivity in Europe until
2020. Upon closing of the transaction in January 2016,
BioMarin will record all sales and profits of Kuvan.
"This is an excellent transaction for BioMarin as it provides
numerous operational and strategic synergies for the Company," said
Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of
BioMarin. "We will leverage our established worldwide
infrastructure and strong relationships within the PKU community to
ensure that patients globally have access to Kuvan, and potentially
pegvaliase upon approval. We look forward to expanding our PKU
franchise beyond the US and Canadian markets and into our existing
commercial footprint of about 60 countries where Kuvan is currently
sold."
2016 Full-year Kuvan Guidance
Starting in January 2016, BioMarin will begin commercializing
and recording revenue from all global sales of Kuvan, with the
exception of Japan. The addition of new Kuvan revenue in 2016
from territories previously held by Merck will result in increased
full-year revenue for BioMarin. Revenue from new ROW
territories outside of the US is expected to be between $70 million
to $80 million in 2016. Total Kuvan revenue to BioMarin for
full-year 2016 is expected to be between $320 million and $350
million, including revenue from new ROW territories. BioMarin
expects to transition commercialization activities from Merck over
the next 6-12 months, and will evaluate opportunities to leverage
its global commercial infrastructure to drive further growth of
Kuvan in these new territories. The transaction is expected
to be accretive to non-GAAP earnings beginning in 2016 and
accretive to GAAP earnings in 2018.
Conference Call Details
BioMarin will host a conference call and webcast to discuss the
acquisition of the global PKU franchise from Merck Serono on
October 1 at 8:30am ET/5:30am PT. This event can be accessed on the
investor section of the BioMarin website at www.BMRN.com.
U.S. / Canada Dial-in Number:
877.303.6313 |
International Dial-in Number:
631.813.4734 |
Conference ID: 50222632 |
|
Replay Dial-in Number: 855.859.2056 |
Replay International Dial-in Number:
404.537.3406 |
Conference ID: 50222632 |
About Phenylketonuria (PKU)
Phenylketonuria (PKU) is an autosomal recessive genetic disorder
caused by a defect or a deficiency of the enzyme phenylalanine
hydroxylase (PAH) or its cofactor tetrahydrobiopterin (BH4). PAH is
required for the metabolism of phenylalanine, an essential amino
acid found in all protein-containing foods. The prevalence of PKU
in BioMarin global commercial territories is estimated to be
approximately 50,000. If PKU patients are not treated with a
phenylalanine-restricted diet, phenylalanine will accumulate in the
blood and brain to abnormally high levels, thereby resulting in a
variety of complications including mental retardation and brain
damage, mental illness, seizures and tremors, and clinically
significant cognitive problems. Universal systematic newborn
screening programs were developed in the 1960s and early 1970s to
enable diagnosis of all patients with PKU patients at birth.
About Kuvan
Kuvan is the first oral therapy approved for the treatment of
hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) in
patients of all ages who have shown to be responsive to Kuvan or
due to tetrahydrobiopterin (BH4) deficiency. Kuvan was developed
jointly by BioMarin Pharmaceutical Inc. and Merck Serono. Kuvan is
to be used in conjunction with a phenylalanine-restricted diet.
Kuvan is the synthetic form of 6R-BH4, a naturally occurring
co-factor that works in conjunction with the enzyme phenylalanine
hydroxylase (PAH) to metabolize phenylalanine into tyrosine.
Clinical data show that Kuvan produces significant reductions in
blood phenylalanine concentration in a large subset of
patients.
Kuvan is approved in 51 countries worldwide, including member
states of the European Union and the USA. Under the terms of the
former agreement with BioMarin, Merck Serono had received exclusive
rights to market Kuvan in all territories outside the USA, Canada
and Japan; all these rights have now been returned to BioMarin.
About Pegvaliase
Pegvaliase (PEGylated recombinant phenylalanine ammonia lyase)
is an investigational enzyme substitution therapy for the treatment
of phenylketonuria (PKU), an inherited metabolic disease caused by
a deficiency of the enzyme phenylalanine hydroxylase (PAH).
Pegvaliase is being developed by BioMarin as a potential
therapeutic option for adult patients with phenylketonuria.
Pegvaliase is currently in pivotal studies with data expected
in March/April of 2016.
Important Safety Information about Kuvan
It is not possible to know if Kuvan will work for you without a
trial of the medicine.
Your doctor will check your blood Phe levels when you start
taking Kuvan to see if the medicine is working.
Starting Kuvan does not eliminate the need for ongoing dietary
management. Any change to your diet may impact your blood Phe
level. Follow your doctor's instructions carefully. Your doctor and
dietitian will continue to monitor your diet and blood Phe levels
throughout your treatment with Kuvan to make sure your
blood Phe levels are not too high or too low. If you have
a fever, or if you are sick, your Phe level may go up. Tell your
doctor and dietitian as soon as possible so they can make any
necessary changes to your treatment.
Children younger than 7 years old treated with Kuvan doses of 20
mg/kg per day are at an increased risk for low levels of blood Phe
compared with children 7 years and older. Frequent blood monitoring
is recommended in this population to ensure that blood Phe levels
do not fall too low.
Tell your doctor if you have ever had liver or kidney problems,
have poor nutrition or have a loss of appetite, are pregnant or
plan to become pregnant, or are breastfeeding or plan to
breastfeed.
Kuvan is a prescription medicine and should not be taken by
people who are allergic to any of its ingredients. Kuvan and other
medicines may interact with each other. Tell your doctor about
all the medicines you take, including prescription
and over-the-counter medicines, vitamins, herbal and dietary
supplements.
If you forget to take your dose of Kuvan, take it as soon as you
remember that day. Do not take 2 doses in a day. If you take too
much Kuvan, call your doctor for advice.
The most common side effects reported when using Kuvan are
headache, runny nose and nasal congestion, sore throat, diarrhea,
vomiting, and cough. Additional adverse reactions reported in
connection with worldwide marketing include sore throat, heartburn
or pain in the esophagus, inflammation of the lining of the
stomach, indigestion, stomach pain, and nausea. These are not all
the possible side effects seen with Kuvan. Call your doctor for
medical advice about side effects. You may report side effects to
FDA at 1-800-FDA-1088.
Kuvan can cause serious side effects, including:
- Severe allergic reactions. Stop taking Kuvan
and get medical help right away if you develop any
of these symptoms of a severe allergic reaction:
- Wheezing or trouble breathing
- Nausea
- Flushing
- Lightheadedness or fainting
- Coughing
- Rash
- Inflammation of the lining of the stomach
(gastritis). Gastritis can happen with Kuvan and may be
severe. Call your doctor right away if you have
any:
- Severe upper stomach-area discomfort or pain
- Blood in your vomit or stool
- Nausea and vomiting
- Black, tarry stools
- Too much or constant activity (hyperactivity) can
happen with Kuvan. Tell your doctor if you have any signs
of hyperactivity, including fidgeting, moving around or talking too
much.
To access full Patient Information, go to
http://kuvan.com/patient-information/.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for patients with serious and
life-threatening rare and ultra-rare genetic diseases. The
company's portfolio consists of five commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.BMRN.com.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: the completion of the
acquisition of the rights to Kuvan and pegvaliase from Merck
Serono, the subsequent commercialization of Kuvan in the Merck
Serono territories and the development and commercialization of
pegvaliase. These forward-looking statements are predictions
and involve risks and uncertainties such that actual results may
differ materially from these statements. These risks and
uncertainties include, among others: clearance of the transaction
by relevant anti-trust authorities; our ability to transition
commercial activities related to Kuvan and our subsequent success
in executing those activities; actions by regulatory authorities,
particularly with respect to the transfer of the Kuvan marketing
authorizations and the development of pegvaliase; the timing and
results of ongoing and future clinical trials of pegvaliase, our
ability to manufacture both Kuvan and pegvaliase in the quantity
and configuration required for each jurisdiction and intended use;
and those factors detailed in BioMarin's filings with the
Securities and Exchange Commission, including, without limitation,
the factors contained under the caption "Risk Factors" in
BioMarin's 2014 Annual Report on Form 10-K, and the factors
contained in BioMarin's reports on Form 10-Q. Stockholders are
urged not to place undue reliance on forward-looking statements,
which speak only as of the date hereof. BioMarin is under no
obligation, and expressly disclaims any obligation to update or
alter any forward-looking statement, whether as a result of new
information, future events or otherwise. For additional
information, please visit www.BMRN.com. Information on BioMarin's
website is not incorporated by reference into this press
release.
BioMarin®, Naglazyme®, Kuvan®, Firdapse® and Vimizim® are
registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme
LLC.
CONTACT: Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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