BioCryst Receives UK Approval of ORLADEYO™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary ...
May 12 2021 - 9:25AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the United Kingdom’s Medicines and Healthcare products Regulatory
Agency (MHRA) has granted marketing authorization for oral,
once-daily ORLADEYO™ (berotralstat) for the routine prevention of
recurrent hereditary angioedema (HAE) attacks in HAE patients 12
years and older.
“HAE UK welcomes the decision from the MHRA to
grant marketing authorization for berotralstat in the UK.
Hereditary angioedema is an unpredictable and life-threatening
condition which causes significant emotional and economic burdens
on people with HAE and their families and careers. These burdens
negatively impact their mental health and well-being, on top of the
physical demands of living with a chronic condition. An oral
therapy that shows effective control of HAE attacks will provide a
different treatment choice for clinicians and patients and will
assist in improving the quality of life of those living with the
condition,” said Laura Szutowicz, chief executive officer of HAE
UK.
“This is a significant milestone in furthering
our ability to meet the needs of more patients living with HAE. As
the first oral, once-daily therapy proven to reduce the number of
HAE attacks, today’s news has the potential to offer a convenient
new treatment option to improve the lives and outcomes of patients
with HAE,” said Dr. Sorena Kiani, consultant immunologist at Barts
Health NHS Trust.
A decision from the National Institute for
Health and Care Excellence (NICE) and Scottish Medicines Consortium
(SMC) for use of ORLADEYO under the UK’s National Health Service
(NHS) is anticipated in the fourth quarter of 2021.
“With the approval of the first oral, once-daily
treatment in the UK, BioCryst continues to bring ORLADEYO to HAE
patients and their families around the world,” said Jon Stonehouse,
president and chief executive officer of BioCryst.
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).
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 UK Summary of Product Characteristics (SPC)
and Patient Information Leaflet (PIL) for ORLADEYO will be
available from the MHRA website at
https://products.mhra.gov.uk/.
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, Japan and the United Kingdom for the prevention of HAE
attacks in adults and pediatric patients 12 years and older.
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, MHRA, 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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