FDA Grants Fast Track Designation for BCX9930 in PNH
August 03 2020 - 7:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that
the U.S. Food and Drug Administration (FDA) has granted Fast Track
designation for its oral Factor D inhibitor, BCX9930, for the
treatment of paroxysmal nocturnal hemoglobinuria (PNH).
According to the FDA, the purpose of the Fast
Track designation is to get important new drugs to the patient
earlier by facilitating the development, and expediting the review,
of drugs to treat serious conditions and fill an unmet medical
need.
A drug that receives Fast Track designation is
eligible for some or all of the following:
- More frequent meetings with FDA to discuss the drug’s
development plan and ensure collection of appropriate data needed
to support drug approval.
- More frequent written communication from FDA about such things
as the design of the proposed clinical trials and use of
biomarkers.
- Eligibility for accelerated approval and priority review, if
relevant criteria are met.
- Rolling review, which means that a drug company can submit
completed sections of its new drug application (NDA) for review by
FDA, rather than waiting until every section of the NDA is
completed before the entire application can be reviewed.
“PNH patients have a tremendous need for therapy
improvements and it is exciting for patients that the FDA has
reviewed our proof of concept PNH data and granted Fast Track
status. This designation can significantly accelerate the
development timeline for BCX9930,” said Dr. William Sheridan, chief
medical officer of BioCryst.
“We look forward to working closely with the FDA
to fully utilize the opportunities presented by our Fast Track
designation to advance this important medicine to patients as
quickly as possible in PNH. In addition, we look forward to
regulatory discussions later this year on clinical trials for
BCX9930 in nephrology indications,” Sheridan added.
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 potential benefits
associated with an FDA Fast Track designation. These statements
involve known and unknown risks, uncertainties and other factors
which may cause the opportunities associated with the Fast Track
designation for BCX9930 to be materially different from those
expressed or implied by the forward-looking statements. These
statements reflect our current views 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: Fast Track designation by the FDA may not lead to a
faster development, regulatory review, or approval process with the
FDA and does not increase the likelihood that BCX9930 will receive
marketing approval; the ongoing COVID-19 pandemic 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; ongoing
and future preclinical and clinical development of BCX9930 may not
have positive results; 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; and the FDA 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 a clinical hold with respect to such
product candidates, or may withhold market approval for product
candidates. 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
projections and forward-looking statements.
BCRXW
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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