ROCTAVIAN is First Gene Therapy for Hemophilia
to Receive Approved Federal Price in Germany
SAN
RAFAEL, Calif., Nov. 28,
2023 /PRNewswire/ -- BioMarin Pharmaceutical
Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to
transforming lives through genetic discovery, today announced an
agreement with the German National Association of Statutory Health
Insurance Funds (GKV-SV) on the reimbursement amount for
ROCTAVIAN® (valoctocogene roxaparvovec-rvox) for
people with severe hemophilia A. This is the first gene therapy for
hemophilia to receive an agreed federal price in Germany. ROCTAVIAN brings value to individuals
and the healthcare system in Germany, as a one-time, single-dose therapy
delivered as an intravenous infusion over 3-4 hours at a gross
price of €28,933.53 per vial. This vial price equates to
approximately $900,000 per patient in
net revenue to BioMarin at the current exchange rate, accounting
for average patient weight and number of vials required for the
one-time treatment, estimated reductions for customary rebates and
discounts.
"We are pleased to have reached an agreement on the
reimbursement amount for ROCTAVIAN with the GKV-SV, which is
responsible for health insurance that covers approximately 90% of
the German population," said Jeff
Ajer, executive vice president and chief commercial officer
at BioMarin. "This important progress will facilitate access to
ROCTAVIAN for eligible individuals among the roughly 2,000 adults
with severe hemophilia A in Germany and marks a key step forward for the
hemophilia community."
BioMarin and GKV-SV have agreed on an outcome-based prospective
cohort model for ROCTAVIAN. This will allow future reimbursement to
be increased or decreased, based on real-world data collected from
the German Haemophilia Registry of patients treated with ROCTAVIAN.
This agreement, with a minimum term of 3 years, ensures the supply
and reimbursement for eligible patients in Germany with ROCTAVIAN by the GKV-SV.
Two patients in Germany have
now received ROCTAVIAN commercially, and 60 people are eligible for
next steps based on companion diagnostic testing results for
adeno-associated virus serotype 5 (AAV5) antibodies.
Additionally, final price negotiations with the Italian
Medicines Agency are underway and expected to be formalized by
year-end 2023.
Significant Progress with U.S. Payers
BioMarin continues to work with private and public payers in the
U.S. to enable access, with the goal of ensuring that every
eligible adult interested in ROCTAVIAN is able to receive
treatment.
Numerous coverage policies that now include ROCTAVIAN have been
published by leading U.S. payers. While medical exception processes
provide a pathway to coverage, these policies representing millions
of lives will help increase access to treatment.
The company continues to partner with hemophilia treatment
centers (HTCs) across the country to educate them on how to
administer ROCTAVIAN, and many HTCs have readiness plans before the
end of this year.
It is estimated that there are approximately 6,500 adults living
with severe hemophilia A in the U.S. BioMarin expects approximately
2,500 of those adults to be eligible to receive ROCTAVIAN at this
time.
About Hemophilia A
Hemophilia A, also called factor VIII (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.
About ROCTAVIAN
ROCTAVIAN is an adeno-associated virus vector-based gene therapy
used for the treatment of adults with severe hemophilia A who do
not have antibodies to adeno-associated virus serotype 5 (AAV5),
which is determined by a blood 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.
The European Commission (EC) granted conditional marketing
authorization to ROCTAVIAN on August 24,
2022. The U.S. Food and Drug Administration (FDA) approved
ROCTAVIAN on June 29, 2023.
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 agreement with the German National Association of
Statutory Health Insurance Funds (GKV-SV) on the reimbursement
amount for ROCTAVIAN, including ROCTAVIAN's ability to bring value
to individuals and the healthcare system in Germany, the estimated per patient net revenue
of ROCTAVIAN to BioMarin in Germany, and BioMarin's ability to facilitate
access to ROCTAVIAN for eligible individuals in Germany; the impact of the outcome-based
prospective cohort model for ROCTAVIAN in Germany, including the ability to increase or
decrease future reimbursements and to ensure the supply and
reimbursement for eligible patients in Germany; BioMarin's expectations regarding the
number of patients who will be eligible to receive ROCTAVIAN in
Germany and the United States; BioMarin's expectations
regarding its negotiations with the Italian Medicines Agency for
the final price of ROCTAVIAN, including the expected timing of
formalization; BioMarin's ability to ensure that every eligible
adult interested in ROCTAVIAN is able to receive treatment; the
ability of the published U.S. coverage policies including ROCTAVIAN
to increase access to treatment; and 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, including, without limitation, the factors
contained under the caption "Risk Factors" in BioMarin's Quarterly
Report on Form 10-Q for the quarter ended September 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
|
View original content to download
multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-agreement-with-german-health-insurance-fund-on-reimbursement-amount-for-roctavian-valoctocogene-roxaparvovec-rvox-for-severe-hemophilia-a-in-germany-301999249.html
SOURCE BioMarin Pharmaceutical Inc.