BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the Ministry of Health, Labor and Welfare (MHLW) in Japan has
granted marketing and manufacturing approval for oral, once-daily
ORLADEYO™ (berotralstat) 150 mg 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. One capsule of ORLADEYO per day works
to prevent HAE attacks by decreasing the activity of plasma
kallikrein.
ORLADEYO will be commercialized in Japan by
BioCryst’s partner, Torii Pharmaceutical Co., Ltd.
OrphanPacific, Inc. is BioCryst’s representative partner in Japan
and holds the marketing authorization.
Torii will launch ORLADEYO in Japan following
the successful completion of BioCryst’s pricing negotiations with
the Japanese National Health Insurance System (NHI).
“Until now, HAE patients in Japan had no
therapies approved to prevent attacks, so the approval of ORLADEYO
marks a significant advance in HAE treatment,” said Goichi Matsuda,
president of Torii. “We are pleased to have the opportunity to
bring the first oral treatment option to Japanese HAE patients and
are actively preparing for the commercialization.”
“Today’s approval of ORLADEYO in Japan
represents important progress towards our goal to bring an oral,
once-daily treatment to HAE patients around the world,” said Jon
Stonehouse, president and chief executive officer of
BioCryst. “Thank you to the HAE patients who
participated in our APeX-J trial, to the investigators who
conducted it, and to Torii and OrphanPacific for their partnership
to achieve this milestone to offer a much-needed new treatment
option to HAE patients and physicians in Japan.”
BioCryst received Orphan Drug and Sakigake
designation for ORLADEYO in Japan and the approval is based on data
from the APeX-J and APeX-2 clinical trials. 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.
In December 2020, the U.S. Food & Drug
Administration (FDA) approved ORLADEYO in the U.S. In Europe, the
European Medicines Agency (EMA) validated its marketing
authorization application (MAA) submission for ORLADEYO and formal
review of the MAA under the centralized procedure is underway. The
company expects an approval decision in Europe in the second
quarter of 2021.
With the approval in Japan, BioCryst is eligible
to receive an additional milestone payment of $15 million from
Torii upon receipt of a reimbursement price approval from Japan’s
National Health Insurance system in excess of the threshold
specified in the agreement with Torii. In addition, BioCryst will
receive tiered royalties ranging from 20 percent to potentially 40
percent of Japanese net sales.
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
INDICATIONORLADEYOTM
(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.
About Torii Pharmaceutical Co.,
Ltd.The corporate mission of Torii Pharmaceutical Co.,
Ltd. is to contribute to the improvement of human health and to
fulfill its responsibilities to customers, shareholders, society
and employees, by supplying world-class pharmaceutical products.
Torii Pharma focuses on Renal diseases, Hemodialysis, Allergy and
Skin diseases as its therapeutic areas of importance. Torii is a
member of Japan Tobacco Inc. (JT) group. Collaboration with JT
takes the form of functional focus, with JT undertaking R&D on
new compounds and Torii integrating manufacture and marketing. In
addition to Torii’s independent activities, Torii‘s partnership
with JT includes in-licensing of high-quality pharmaceuticals. More
details can be found on the corporate website
https://www.torii.co.jp/en/.
About OrphanPacific,
Inc.OrphanPacific is dedicated to providing orphan drugs
and also developing sales business of essential drugs in Japan.
OrphanPacific, a member of the CMIC Group, is pivotal to the
Group’s Innovative Pharma Model (IPM) strategy, designed to provide
support and expertise to global specialty pharmaceutical companies
that focus on acquiring the manufacturing and marketing rights of
prescription medicines across a broad range of therapeutic areas
worldwide but don’t have a license to manufacture and distribute
the pharmaceutical products in Japan. For more information, please
visit www.orphanpacific.com/en/.
Forward-Looking Statements This
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, performances 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; risks related to government actions, including that
decisions and other actions relating to pricing and exclusivity of
ORLADEYO in Japan may not be taken when expected or at all, or that
the outcomes of such decisions and other actions may not be in line
with 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 product candidates, including to support
the continued commercialization of ORLADEYO, may not provide
regulatory clearances which may result in delay of planned clinical
trials, may impose certain restrictions, warnings, or other
requirements on product candidates, including ORLADEYO, may
impose a clinical hold with respect to such product candidates,
or may withhold or withdraw market approval for such
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
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/3c7764e3-37b3-428c-8dbf-b81655b08a6c
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