First Person Treated in Germany; Reimbursement Discussions on Track in
France and Italy
In the U.S., ROCTAVIAN Now Commercially
Available and Hemophilia Treatment Centers Have Begun Screening
Eligible Individuals
SAN
RAFAEL, Calif., Aug. 30,
2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc.
(Nasdaq: BMRN), a global biotechnology company dedicated to
transforming lives through genetic discovery, today announced that
an individual in Germany with
severe hemophilia A was treated with
ROCTAVIAN® (valoctocogene
roxaparvovec-rvox), marking the first time that the gene therapy
has been given commercially in Europe.
"Today represents an important milestone for the hemophilia
community, and for patients and physicians around the world seeking
access to ROCTAVIAN," said Jeff
Ajer, executive vice president and chief commercial officer
at BioMarin. "We look forward to more people gaining access to
ROCTAVIAN in the rest of Europe,
as well as the United States,
where ROCTAVIAN recently received FDA approval."
Hemophilia treatment centers (HTCs) in Germany are testing people with hemophilia to
determine treatment eligibility for the one-time gene therapy.
Before beginning treatment, individuals are evaluated clinically
and undergo testing. Currently, dozens of individuals are
undergoing screening to determine eligibility for treatment with
ROCTAVIAN. Final federal price negotiations in Germany are also ongoing.
"The burden of severe hemophilia A for people who are living
with the condition is substantial, and there remains a significant
unmet need for effective treatments that do not require chronic
therapy. This one-time infusion represents an important milestone,
offering new hope and potential, for eligible individuals in
Germany," said PD Dr. med.
Robert Klamroth, a treating
physician and chief physician of the Center for Hemophilia and
Hemostaseology at the Vivantes Klinikum in Berlin, Germany. "Bringing this therapy to all
those who can benefit is critical to improve outcomes for
individuals with severe hemophilia A."
Beyond Germany, the company's
applications seeking price and reimbursement approvals and other
launch preparation activities continue to progress in France and Italy. In Italy, ROCTAVIAN was awarded conditional
innovation designation, which is expected to facilitate pricing and
reimbursement.
U.S. Launch Activities Accelerating, including Eligibility
Testing at HTCs
In June, the FDA approved ROCTAVIAN for the treatment of adults
with severe hemophilia A (congenital factor VIII (FVIII) deficiency
with FVIII activity < 1 IU/dL) without antibodies to
adeno-associated virus serotype 5 (AAV5) detected by an
FDA-approved test.
ROCTAVIAN is now commercially available in the U.S. and HTCs
have begun screening people with severe hemophilia A to determine
eligibility. BioMarin has also seen an increasing inflow of patient
consent forms and a number of executed or in-process warranty
agreements that are expected to facilitate access and uptake of
ROCTAVIAN at HTCs across the U.S.
About Hemophilia A
Hemophilia A, also called FVIII deficiency or classic
hemophilia, is an X-linked genetic disorder caused by missing or
defective FVIII, a clotting protein. Although it is passed down
from parents to children, about one-third of cases are caused by a
spontaneous mutation, a new mutation that was not inherited.
Approximately 1 in 10,000 people have hemophilia A.
People living with hemophilia A lack sufficient functioning
FVIII protein to help their blood clot and are at risk for painful
and/or potentially life-threatening bleeds from even modest
injuries. Additionally, people with the most severe form of
hemophilia A (FVIII levels <1%) often experience painful,
spontaneous bleeds into their muscles or joints. Individuals with
the most severe form of hemophilia A make up approximately 50% of
the hemophilia A population. People with hemophilia A with moderate
(FVIII levels 1-5%) or mild (FVIII levels 5-40%) disease show a
much-reduced propensity to bleed. Individuals with severe
hemophilia A are treated with a prophylactic regimen of intravenous
FVIII infusions administered 2-3 times per week (100-150 infusions
per year) or a bispecific monoclonal antibody that mimics the
activity of FVIII administered 1-4 times per month (12-48
injections or shots per year). Despite these regimens, many people
continue to experience breakthrough bleeds, resulting in
progressive and debilitating joint damage, which can have a major
impact on their quality of life.
About ROCTAVIAN
ROCTAVIAN is an adeno-associated virus vector–based gene therapy
indicated for the treatment of adults with severe hemophilia A
(congenital factor VIII deficiency with factor VIII activity < 1
IU/dL) without antibodies to adeno–associated virus serotype 5
(AAV5) detected by an FDA–approved test. The one-time infusion
works by delivering a functional gene that is designed to enable
the body to produce FVIII on its own, reducing the need for ongoing
prophylaxis, demonstrated by results reported through a 3-year
study period.
The European Commission (EC) granted conditional marketing
authorization to ROCTAVIAN on August 24, 2022.
More information on testing to determine eligibility to receive
ROCTAVIAN can be found at https://www.ROCTAVIAN.com in the U.S. and
https://www.ROCTAVIAN.de in Germany.
U.S. Important Safety Information
Contraindications: Patients with active infections,
either acute (such as acute respiratory infections or acute
hepatitis) or uncontrolled chronic (such as chronic active
hepatitis B). Patients with known significant hepatic fibrosis
(stage 3 or 4 on the Batts-Ludwig scale or equivalent), or
cirrhosis, and patients with known hypersensitivity to
mannitol.
Infusion-related reactions including hypersensitivity
reactions and anaphylaxis, have occurred. Monitor during and for at
least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN
in a setting where personnel and equipment are immediately
available to treat infusion-related reactions. Discontinue infusion
for anaphylaxis.
Hepatotoxicity: The safety and effectiveness of ROCTAVIAN
in patients with hepatic impairment has not been established.
Perform liver health assessments prior to administration. The
majority of patients treated with ROCTAVIAN experienced ALT
elevations and required corticosteroids for ALT elevation. Assess
patient's ability to receive corticosteroids and/or other
immunosuppressive therapy that may be required for an extended
period. Live vaccines should not be administered to patients while
on immunosuppressive therapy.
Monitor ALT weekly for at least 26 weeks and as clinically
indicated, during corticosteroid therapy and institute
corticosteroid treatment in response to ALT elevations as required.
Continue to monitor ALT until it returns to baseline. Monitor
factor VIII activity levels since ALT elevation may be accompanied
by a decrease in factor VIII activity. One case of autoimmune
hepatitis was reported during third year follow-up in a patient
with history of hepatitis C and steatohepatitis.
It is recommended that patients abstain from consuming alcohol
for at least 1 year after administration and thereafter limit
alcohol use. Concomitant medications may cause hepatotoxicity,
decrease factor VIII activity, or change plasma corticosteroid
levels which may impact liver enzyme elevation and/or factor VIII
activity or decrease the efficacy of the corticosteroid regimen or
increase their side effects. Closely monitor concomitant medication
use including herbal products and nutritional supplements and
consider alternative medications in case of potential drug
interactions.
Thromboembolic events: Factor VIII activity above ULN has
been reported following ROCTAVIAN infusion. Thromboembolic events
may occur in the setting of elevated factor VIII activity above
ULN. Evaluate patients for risk of thrombosis including general
cardiovascular risk factors before and after administration
of ROCTAVIAN. Advise patients on their individual risk of
thrombosis in relation to their factor VIII activity levels above
ULN and consider prophylactic anticoagulation. Advise patients to
seek immediate medical attention for signs or symptoms indicative
of a thrombotic event.
Factor VIII inhibitors and Monitoring for inhibitors: The
safety and effectiveness of ROCTAVIAN in patients with prior or
active factor VIII inhibitors have not been established. Patients
with active factor VIII inhibitors should not take ROCTAVIAN.
Following administration, monitor patients for factor VIII
inhibitors (neutralizing antibodies to factor VIII). Test for
factor VIII inhibitors especially if bleeding is not controlled, or
plasma factor VIII activity levels decrease.
Monitor Factor VIII using the same schedule for ALT
monitoring. It may take several weeks after ROCTAVIAN
infusion before ROCTAVIAN-derived factor VIII activity rises to a
level sufficient for prevention of spontaneous bleeding episodes.
Exogenous factor VIII or other hemostatic products may also be
required in case of surgery, invasive procedures, trauma, or
bleeds. Consider more frequent monitoring in patients with factor
VIII activity levels ≤ 5 IU/dL and evidence of bleeding,
taking into account the stability of factor VIII levels since the
previous measurement.
Factor VIII activity produced by ROCTAVIAN in human plasma is
higher if measured with one-stage clotting assays compared to
chromogenic substrate assays. When switching from hemostatic
products prior to ROCTAVIAN treatment, physicians should refer to
the relevant prescribing information to avoid the potential for
factor VIII activity assay interference during the transition
period.
Malignancy: The integration of liver-targeting AAV vector
DNA into the genome may carry the theoretical risk of
hepatocellular carcinoma development. ROCTAVIAN can also insert
into the DNA of other human body cells. Monitor patients with risk
factors for hepatocellular carcinoma (e.g., hepatitis B or C,
non-alcoholic fatty liver disease, chronic alcohol consumption,
non-alcoholic steatohepatitis, advanced age) with regular liver
ultrasound (e.g., annually) and alpha-fetoprotein testing for 5
years following ROCTAVIAN administration. In the event that any
malignancy occurs after treatment with ROCTAVIAN, contact BioMarin
Pharmaceutical Inc. at 1-866-906-6100.
Most Common Adverse Reactions: Most common adverse
reactions (incidence ≥ 5%) were nausea, fatigue, headache,
infusion-related reactions, vomiting, and abdominal pain. Most
common laboratory abnormalities (incidence ≥ 10%) were
ALT, AST, LDH, CPK, factor VIII activity levels, GGT and
bilirubin > ULN. Patients also experienced adverse reactions
from corticosteroid use.
Isotretinoin, Efavirenz, and HIV Positive Patients:
Isotretinoin is not recommended in patients who are benefiting from
ROCTAVIAN. Efavirenz is not recommended in patients treated with
ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient
numbers of patients with HIV to determine whether the efficacy and
safety differs compared to patients without HIV infection.
Females and Males of Reproductive Potential: ROCTAVIAN is
not intended for administration in women. There are no data on the
use of ROCTAVIAN in pregnant women or regarding lactation. For 6
months after administration of ROCTAVIAN, men of reproductive
potential and their female partners must prevent or postpone
pregnancy using an effective form of contraception, and men must
not donate semen.
You may report side effects to the FDA at (800) FDA-1088 or
www.fda.gov/medwatch. You may also report side effects to BioMarin
at 1-866-906-6100.
Please see the ROCTAVIAN full Prescribing Information for
additional Important Safety Information.
About BioMarin
Founded in 1997, BioMarin is a global biotechnology company
dedicated to transforming lives through genetic discovery. The
company develops and commercializes targeted therapies that address
the root cause of genetic conditions. BioMarin's unparalleled
research and development capabilities have resulted in eight
transformational commercial therapies for patients with rare
genetic disorders. The company's distinctive approach to drug
discovery has produced a diverse pipeline of commercial, clinical,
and pre-clinical candidates that address a significant unmet
medical need, have well-understood biology, and provide an
opportunity to be first-to-market or offer a substantial benefit
over existing treatment options. For additional information, please
visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc. (BioMarin),
including, without limitation, statements about: the potential
impact of the first commercial treatment with ROCTAVIAN for severe
hemophilia A in Europe, including
(i) the potential benefits for the hemophilia community, and
patients and physicians in Germany
and around the world seeking access to ROCTAVIAN, (ii) BioMarin's
ability to commercially progress ROCTAVIAN in Europe and the
United States, and (iii) ROCTAVIAN's ability to improve
outcomes for individuals with severe hemophilia A; the
commercialization of ROCTAVIAN, including (i) BioMarin's
expectations regarding the number of patients who will be eligible
to receive ROCTAVIAN, including through patient eligibility testing
at HTCs in Germany and
the United States, (ii) BioMarin's
expectations regarding the ability to facilitate access and uptake
of ROCTAVIAN at HTCs across the United
States, and (iii) BioMarin's ability to obtain price and
reimbursement approvals in Germany, France and Italy; and the clinical development of
BioMarin's comprehensive gene therapy program for the treatment of
severe hemophilia A, including the ongoing clinical development of
ROCTAVIAN. 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: BioMarin's success in the commercialization
of ROCTAVIAN, including achieving adequate market share and
reimbursement levels; whether ROCTAVIAN will have the impacts and
benefits as anticipated; the results and timing of current and
planned preclinical studies and clinical trials of ROCTAVIAN and
the release of data from those trials, including continued
monitoring of the participants in the clinical trials and
post-approval studies; BioMarin's ability to successfully
manufacture ROCTAVIAN for the clinical trials and commercially; the
content and timing of decisions by the FDA, EU health authorities
and other regulatory authorities regarding ROCTAVIAN; and those
factors detailed in BioMarin's filings with the Securities and
Exchange Commission (SEC), including, without limitation, the
factors contained under the caption "Risk Factors" in BioMarin's
Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, as such factors may be updated by
any subsequent reports. 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.
BioMarin® is a registered trademark of BioMarin
Pharmaceutical Inc. ROCTAVIAN® is a trademark of
BioMarin Pharmaceutical Inc., with registration in Europe and pending in the U.S.
Contacts:
|
|
Investors
|
Media
|
Traci
McCarty
|
Andrew Villani
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(628)
269-7393
|
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SOURCE BioMarin Pharmaceutical Inc.