BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the Committee for Medicinal Products for Human Use (CHMP) of
the European Medicines Agency (EMA) has adopted a positive
opinion recommending the approval of ORLADEYO™ (berotralstat)
for routine prevention of recurrent attacks of hereditary
angioedema (HAE) in adult and adolescent patients aged
12 years and older.
The European Commission (EC) will review the
CHMP recommendation and a final approval decision from the EC on
the marketing authorization application (MAA) for ORLADEYO is
expected in the second quarter.
If approved, ORLADEYO would be the first oral,
once-daily therapy in the European Union to treat patients with HAE
by preventing recurrent attacks. The CHMP positive opinion is based
on data from the pivotal APeX-2 clinical trial and supporting data
from the APeX-S trial. In APeX-2, ORLADEYO met its primary endpoint
(p<0.001) for ORLADEYO 150 mg compared to placebo. ORLADEYO
showed a positive safety profile and was generally well-tolerated
over 48 weeks in both APeX-2 and APeX-S.
“Following the recent approvals in the U.S. and
Japan, we continue to focus on bringing our oral, once-daily
treatment to HAE patients around the world,” said Jon Stonehouse,
president and chief executive officer of BioCryst. “The positive
CHMP opinion for ORLADEYO is an important step closer to delivering
a new option to HAE patients across Europe and our commercial team
is in place and ready to launch quickly upon final EC
approval.”
In December 2020, the U.S. Food & Drug
Administration (FDA) approved ORLADEYO in the U.S. In January 2021,
ORLADEYO was approved by the Ministry of Health, Labour and Welfare
in Japan. An Early Access to Medicines Scheme (EAMS) for HAE
patients has been approved by the Medicines & Healthcare
products Regulatory Agency in the United Kingdom.
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 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 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 periodically
files 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 commercial viability of ORLADEYO,
including its ability to achieve market acceptance; 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
Contact:InvestorsJohn Bluth+1 919
859 7910jbluth@biocryst.com
MediaCatherine Collier
Kyroulis+1 917 886 5586ckyroulis@biocryst.com
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