MHRA Grants HAE Patients Early Access to BioCryst’s Berotralstat in United Kingdom
October 30 2020 - 8:15AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the United Kingdom’s Medicines and Healthcare products Regulatory
Agency (MHRA) has granted oral, once-daily berotralstat a positive
scientific opinion through the Early Access to Medicines Scheme
(EAMS).
Under the EAMS, hereditary angioedema (HAE)
patients in the UK aged 12 years and older can gain access to
berotralstat for the routine prevention of recurrent attacks of HAE
before the drug is granted marketing authorization by the European
Commission (EC).
HAE is a serious, and potentially
life-threatening, rare genetic illness characterised by periodic
episodes of acute swelling of the skin, pharynx, larynx,
gastrointestinal tract, genitals and/or extremities.
Medicines included in the EAMS are those that
have a high unmet need, are intended to treat, diagnose or prevent
seriously debilitating or life-threatening conditions where there
are no adequate treatment options, and are likely to offer
significant advantage over methods currently used in the UK. Under
the scheme, the MHRA provides a scientific opinion on the
benefit-risk balance of the medicine, based on the data available
when the EAMS submission was made.
“There are many patients in the UK that don’t
have a realistic option for effective HAE prophylaxis. The addition
of berotralstat through the EAMS will bring a much needed option
for HAE patients suffering with this debilitating disease,” said
Dr. Sorena Kiani, Consultant Immunologist at Royal London Hospital,
London.
“HAE patients around the world are waiting for
an oral, once-daily therapy to prevent attacks and reduce their
burden of therapy. With this decision by the MHRA, the wait for
many HAE patients in the UK can end sooner,” said Jon Stonehouse,
chief executive officer of BioCryst.
The European Medicines Agency (EMA) is reviewing
the marketing authorisation application (MAA) for berotralstat
under the centralized procedure. An opinion from the Committee for
Medicinal Products for Human Use (CHMP) is expected approximately
12 months from MAA validation, which the company announced on March
30, 2020.
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. BioCryst has several ongoing
development programs including ORLADEYO™ (berotralstat), an oral
treatment for hereditary angioedema, BCX9930, 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 future results, performance or achievements.
These statements are identified by use of terms such as “expect,”
“will,” and similar words, although some forward-looking statements
may be expressed differently. 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; developing and commercializing ORLADEYO (berotralstat)
or any HAE product candidate may take longer or may be more
expensive than planned; BioCryst may not be able to enroll the
required number of subjects in planned clinical trials of product
candidates; BioCryst may not advance human clinical trials with
product candidates as expected; the FDA, EMA, PMDA or other
applicable regulatory agency may require additional studies beyond
the studies planned for 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 product candidates, may impose a clinical hold with
respect to such product candidates, or may withhold market approval
for product candidates; product candidates, if approved, may
not achieve market acceptance; BioCryst’s ability to
successfully commercialize its product candidates, 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
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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