BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the U.S. Food and Drug Administration (FDA) has approved oral,
once-daily ORLADEYO™ (berotralstat) for prophylaxis to prevent
attacks of hereditary angioedema (HAE) in adults and pediatric
patients 12 years and older.
“ORLADEYO offers people with HAE and their
physicians the first orally administered non-steroidal option for
preventing HAE attacks and represents an important and welcome step
in making more treatment options available to physicians and
patients,” said Anthony J. Castaldo, president and chief executive
officer of the US Hereditary Angioedema Association
(HAEA).
In the pivotal Phase 3 APeX-2 trial, ORLADEYO
significantly reduced attacks at 24 weeks, and this reduction was
sustained through 48 weeks. HAE patients who completed 48 weeks of
treatment (150 mg) saw reductions in their HAE attack rates, from a
mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks
per month after 48 weeks of therapy. In the long-term open label
APeX-S trial, patients completing 48 weeks of therapy (150 mg) had
a mean attack rate of 0.8 attacks per month.
ORLADEYO was safe and well tolerated in both
trials. The most frequently reported adverse reactions in patients
receiving ORLADEYO compared with placebo were gastrointestinal
reactions. These reactions generally occurred early after
initiation of treatment with ORLADEYO, became less frequent with
time and typically self-resolved.
“Patients and physicians acknowledge that HAE
treatments can add a burden to patients’ lives. As an oral,
once-daily option, ORLADEYO can provide significant attack
reduction and lessen the burden associated with injections and
infusions,” said Marc Riedl, M.D., professor of medicine and
clinical director, U.S. Hereditary Angioedema Association Center at
the University of California, San Diego, and an investigator in the
APeX-2 trial.
“With this new treatment option, physicians and
patients can continue to have
collaborative discussions to choose
the treatment that meets each patient’s needs, life
circumstances and preferences,” Riedl added.
HAE patients note a significant treatment burden
associated with existing prophylactic therapy. In addition to
reducing HAE attack rate, data from APeX-2 show that patients
reported meaningful improvements in both quality of life and
overall patient-reported satisfaction, and significant reductions
in their monthly use of standard of care on-demand medicine, while
taking oral, once-daily ORLADEYO (150 mg).2,3
“The FDA approval of ORLADEYO fulfills a promise
BioCryst made to HAE patients that we were committed to helping
them achieve the dream of an oral, once-daily medicine to prevent
and reduce the burden of their attacks,” said Jon Stonehouse,
president and chief executive officer of BioCryst.
“Thank you to the HAE patients who participated
in our clinical trials, to the investigators around the world who
conducted these trials, to the HAEA for their patient advocacy and
to our employees who never forgot that patients were waiting. We
will stay focused on enabling access and providing personalized
support to HAE patients and physicians,” Stonehouse added.
Commitment to Patient
Access
BioCryst is committed to supporting HAE patients
taking ORLADEYO through a new program designed to streamline access
to therapy. Through EMPOWER Patient Services, each HAE patient and
their healthcare provider will have a single point of contact for
access to ORLADEYO. A dedicated care coordinator will support
access for each patient with comprehensive financial support tools
and reimbursement support.
EMPOWER Patients Services is administered by
Optime Care Inc., the exclusive specialty pharmacy provider for
ORLADEYO. Physicians can begin writing prescriptions for ORLADEYO
immediately, with direct to patient shipments from Optime Care
expected to begin by the end of December 2020.
Additional information is available
at www.ORLADEYO.com and 1-866-5-EMPOWER (1-866-536-7693).
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 for the prevention
of HAE attacks in adults and pediatric patients 12 years and older,
and under regulatory review for approval in Japan and the European
Union. BioCryst has several ongoing development programs including,
an oral Factor D inhibitor for the treatment of complement-mediated
diseases, galidesivir, a potential treatment for COVID-19, Marburg
virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for
the treatment of fibrodysplasia ossificans progressiva. RAPIVAB®
(peramivir injection), a viral neuraminidase inhibitor for the
treatment of influenza, is BioCryst’s first approved product and
has received regulatory approval in the U.S., Canada, Australia,
Japan, Taiwan, Korea and the European Union. 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
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 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 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 2020
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
Contacts:John Bluth+1 919 859
7910jbluth@biocryst.com
Catherine Collier Kyroulis+1 917 886
5586ckyroulis@biocryst.com
________________________1 Radojicic, C, et al. Patient
Perspectives on the Treatment Burden of Injectable Medication
Administration for Hereditary Angioedema. Annual Scientific Meeting
of the American College of Allergy, Asthma &
Immunology; Poster 160, Nov (2020)
2 Johnston, D.T., et al. Berotralstat Improves
Patient-Reported Quality of Life Through 48 Weeks in the Phase 3
APeX-2 Trial; Annual Scientific Meeting of the American College of
Allergy, Asthma & Immunology; Poster 154, Nov
(2020)
3 Jacobs, J, et al. Berotralstat Positively Impacts
Patient-Reported Satisfaction: Results from the Phase 3 APeX-2
trial; Annual Scientific Meeting of the American College of
Allergy, Asthma & Immunology; Poster 158, Nov
(2020)
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/bf57a925-5da4-4c72-8a93-02e9c4308123
BioCryst Pharmaceuticals (NASDAQ:BCRX)
Historical Stock Chart
From Mar 2024 to Apr 2024
BioCryst Pharmaceuticals (NASDAQ:BCRX)
Historical Stock Chart
From Apr 2023 to Apr 2024