BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the European Commission (EC) has approved oral, once-daily
ORLADEYO™ (berotralstat) for the prevention of recurrent hereditary
angioedema (HAE) attacks in HAE patients 12 years and older.
“As the first targeted oral prophylactic therapy
approved in Europe, ORLADEYO represents a major advance in
treatment for HAE patients who have been waiting for a preventive
therapy. Physicians will be delighted to discuss this new option
with their patients,” said Emel Aygören-Pürsün, M.D., head of the
HAE Center at the University Hospital in Frankfurt.
“ORLADEYO offers people with HAE in Europe and
their physicians the first orally administered non-steroidal option
for preventing HAE attacks and represents a vitally important and
most welcome step in making more treatment options available,” said
Henrik Balle Boysen, executive vice president and chief operating
officer of HAE International, a global non-profit network of
patient associations dedicated to improving the lives of people
with HAE.
The EC approval of ORLADEYO is applicable to all
European Union member states plus Iceland, Norway and
Liechtenstein.
BioCryst has its European commercial team in
place and expects to launch ORLADEYO this quarter in Germany, with
launches in other European markets to follow. HAE patients in
France currently have access to ORLADEYO through an Autorisation
Temporaire d'Utilisation de cohorte (cohort ATU).
In the United Kingdom, HAE patients also
currently have access to ORLADEYO through an approved early access
to medicines scheme (EAMS). A marketing authorization application
(MAA) has been submitted to the Medicines and Healthcare products
Regulatory Agency (MHRA). Under the new European Commission
Decision Reliance Procedure, the MHRA will aim to complete the
review of the UK MAA as soon as possible following the EC approval
decision.
“Most European HAE patients today treat their
disease with on-demand therapy or androgens and we believe the
approval of oral, once-daily ORLADEYO provides an exciting new
opportunity for these patients to reduce their burden of therapy by
moving to prophylaxis with ORLADEYO,” said Jon Stonehouse,
president and chief executive officer of BioCryst.
“We saw tremendous enthusiasm and participation
from European HAE patients in our clinical trials and we have
invested in an experienced European commercial team that is excited
to bring ORLADEYO to HAE patients across Europe,” he added.
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.
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, 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). 1,2
About
ORLADEYO™
(berotralstat)ORLADEYO™ (berotralstat) is the
first and only oral therapy designed specifically to prevent
attacks of hereditary angioedema (HAE) in adult 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.
The full European Summary of Product
Characteristics (SMPC) for ORLADEYO will be available from the
European Medicines Association website at www.ema.europa.eu.
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, European
Union 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 United Kingdom. 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 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 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; risks relating to government actions,
including that decisions and other actions relating to pricing and
reimbursements 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; 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
1 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)
2 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)
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