BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the Japanese National Health Insurance System (NHI) has approved
the addition of oral, once-daily ORLADEYO™ (berotralstat) to the
NHI drug price list on April 21, 2021.
Oral, once-daily ORLADEYO was approved in Japan
in January 2021 for prophylactic treatment of hereditary angioedema
(HAE) in adults and pediatric patients 12 years and older. ORLADEYO
is the first and only prophylactic HAE medication approved in
Japan.
ORLADEYO will be commercialized in Japan by
BioCryst’s partner, Torii Pharmaceutical Co., Ltd. Torii plans to
launch ORLADEYO following the NHI drug price listing.
“Our goal is to bring ORLADEYO to HAE patients
around the world who want a new oral, once-daily option to prevent
their attacks. In Japan, ORLADEYO is the first approved
prophylactic HAE medication, which has the potential to
significantly impact the lives of HAE patients,” said Jon
Stonehouse, president and chief executive officer of
BioCryst.
The NHI price listing triggers a $15 million
milestone payment from Torii to BioCryst. In addition, BioCryst
will receive tiered royalties ranging from 20 percent to 40 percent
of Japanese net sales.
BioCryst received Orphan Drug and Sakigake
designation for ORLADEYO in Japan. The APeX-J trial in Japan met
its primary endpoint (p=0.003) of a reduction in HAE attacks from
baseline for ORLADEYO 150 mg compared to placebo, and ORLADEYO was
safe and generally well-tolerated in the trial. In APeX-2, ORLADEYO
also met its primary endpoint (p<0.001) for ORLADEYO 150 mg
compared to placebo and was safe and generally well-tolerated.
About ORLADEYO™ (berotralstat)
ORLADEYO™ (berotralstat) is the first and only
oral therapy designed specifically to prevent attacks of hereditary
angioedema (HAE) in adults and pediatric patients 12 years and
older. One capsule of ORLADEYO per day works to prevent HAE attacks
by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety
Information
INDICATIONORLADEYO™ (berotralstat) is a
plasma kallikrein inhibitor indicated for prophylaxis to prevent
attacks of hereditary angioedema (HAE) in adults and pediatric
patients 12 years and older.
Limitations of useThe safety
and effectiveness of ORLADEYO for the treatment of acute HAE
attacks have not been established. ORLADEYO should not be used for
the treatment of acute HAE attacks. Additional doses or dosages of
ORLADEYO higher than 150 mg once daily are not recommended due to
the potential for QT prolongation.
IMPORTANT SAFETY INFORMATION An
increase in QT prolongation was observed at dosages higher than the
recommended 150 mg once-daily dosage and was concentration
dependent.
The most common adverse reactions (≥10% and
higher than placebo) in patients receiving ORLADEYO were abdominal
pain, vomiting, diarrhea, back pain, and gastroesophageal reflux
disease.
A reduced dosage of 110 mg taken orally once
daily with food is recommended in patients with moderate or severe
hepatic impairment (Child-Pugh B or C) and in patients taking
chronically administered P-glycoprotein (P-gp) or breast cancer
resistance protein (BCRP) inhibitors (eg, cyclosporine).
Berotralstat is a substrate of P-gp and BCRP.
P-gp inducers (eg, rifampin, St. John’s wort) may decrease
berotralstat plasma concentration, leading to reduced efficacy of
ORLADEYO. The use of P-gp inducers is not recommended with
ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate
inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a
narrow therapeutic index that are predominantly metabolized by
CYP2D6 or CYP3A4, appropriate monitoring and dose titration is
recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor.
Appropriate monitoring and dose titration is recommended for P-gp
substrates (eg, digoxin) when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in
pediatric patients <12 years of age have not been
established.
There are insufficient data available to inform
drug-related risks with ORLADEYO use in pregnancy. There are no
data on the presence of berotralstat in human milk, its effects on
the breastfed infant, or its effects on milk production.
To report SUSPECTED ADVERSE REACTIONS,
contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at
1-800-FDA-1088 or
www.fda.gov/medwatch.
Please see full
Prescribing Information.
About BioCryst
Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral,
small-molecule medicines that treat rare diseases in which
significant unmet medical needs exist and an enzyme plays a key
role in the biological pathway of the disease. Oral, once-daily
ORLADEYO™ (berotralstat) is approved in the United States and Japan
for the prevention of HAE attacks in adults and pediatric patients
12 years and older, and under regulatory review for approval in the
European Union. BioCryst has several ongoing development programs
including BCX9930, an oral Factor D inhibitor for the treatment of
complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the
treatment of fibrodysplasia ossificans progressiva, and
galidesivir, a potential treatment for Marburg virus disease and
Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase
inhibitor for the treatment of influenza, has received regulatory
approval in the U.S., Canada, Australia, Japan, Taiwan and Korea.
Post-marketing commitments for RAPIVAB are ongoing. For more
information, please visit the company’s website at
www.biocryst.com.
Forward-Looking StatementsThis
press release contains forward-looking statements, including
statements regarding BioCryst’s plans and expectations for
ORLADEYO. These statements involve known and unknown risks,
uncertainties and other factors which may cause BioCryst’s actual
results, performance or achievements to be materially different
from any future results, performance or achievements expressed or
implied by the forward-looking statements. These statements reflect
our current views with respect to future events and are based on
assumptions and are subject to risks and uncertainties. Given these
uncertainties, you should not place undue reliance on these
forward-looking statements. Some of the factors that could
affect the forward-looking statements contained herein include: the
ongoing COVID-19 pandemic, which could create challenges in
all aspects of BioCryst’s business, including without
limitation delays, stoppages, difficulties and increased expenses
with respect to BioCryst’s and its partners’
development, regulatory processes and supply chains, negatively
impact BioCryst’s ability to access the capital or credit
markets to finance its operations, or have the effect of
heightening many of the risks described below or in the
documents BioCryst files periodically with the
Securities and Exchange Commission; BioCryst’s ability to
successfully implement its commercialization plans for, and to
commercialize, ORLADEYO, which could take longer or be more
expensive than planned; the results of BioCryst’s partnerships with
Torii and OrphanPacific may not meet BioCryst’s current
expectations; the commercial viability of ORLADEYO, including its
ability to achieve market acceptance, which could impact the amount
of any related royalties BioCryst would be entitled to receive from
Torii; the FDA, EMA, PMDA or other applicable regulatory agency may
require additional studies beyond the studies planned for products
and product candidates, may not provide regulatory clearances which
may result in delay of planned clinical trials, may impose
certain restrictions, warnings, or other requirements on products
and product candidates, may impose a clinical hold with
respect to product candidates, or may withhold, delay, or
withdraw market approval for products and product
candidates; BioCryst’s ability to successfully manage its
growth and compete effectively; risks related to the international
expansion of BioCryst’s business; and actual financial results
may not be consistent with expectations, including that operating
expenses and cash usage may not be within management's expected
ranges. Please refer to the documents BioCryst files
periodically with the Securities and Exchange Commission,
specifically BioCryst’s most recent Annual Report on Form 10-K,
Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K,
all of which identify important factors that could cause the actual
results to differ materially from those contained in BioCryst’s
forward-looking statements.
BCRXW
Investors:John Bluth+1 919 859
7910jbluth@biocryst.com
Media:Catherine Collier
Kyroulis+1 917 886 5586ckyroulis@biocryst.com
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