Spark Therapeutics Completes Rolling Biologics License Application Submission to FDA for Investigational Gene Therapy Voreti...
18 May 2017 - 9:30PM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy
company dedicated to challenging the inevitability of genetic
disease, today announced the completion of the rolling submission
of a Biologics License Application (BLA) with the U.S. Food and
Drug Administration (FDA) for voretigene neparvovec, an
investigational, one-time gene therapy for the treatment of
patients with vision loss due to confirmed biallelic RPE65
mutation-associated retinal disease. The BLA submission includes
data from three clinical trials that enrolled 41 participants with
RPE65-mediated IRD, including the first randomized, controlled
Phase 3 trial for a gene therapy for a genetic disease.
FDA will have 60 days to review the submission to determine if
it is complete. If deemed complete, the application will be
considered filed and the review period will begin.
“Completion of the rolling BLA is another step forward in our
goal to bring this investigational gene therapy to people living
with RPE65-mediated IRD who currently have no approved
pharmacologic treatment options,” said Jeffrey D. Marrazzo,
chief executive officer. “We look forward to working closely with
FDA, with the hope of bringing voretigene neparvovec, the first
potential gene therapy for a genetic disease in the U.S., to
patients as quickly as possible.”
About Spark TherapeuticsSpark Therapeutics, a
fully integrated company, strives to challenge the inevitability of
genetic disease by discovering, developing, and delivering gene
therapies that address inherited retinal diseases (IRDs),
neurodegenerative diseases, as well as diseases that can be
addressed by targeting the liver. Our validated platform
successfully has delivered proof-of-concept data with
investigational gene therapies in the retina and liver. Our most
advanced investigational candidate, voretigene neparvovec, in
development for the treatment of biallelic RPE65-mediated IRD, has
received orphan designations in the U.S. and European Union, and
breakthrough therapy designation in the U.S. The pipeline also
includes SPK-7001 in a Phase 1/2 trial for choroideremia, and two
hemophilia development programs: SPK-9001 (which also has received
both breakthrough therapy and orphan product designations by the
FDA, and access to the PRIority MEdicines (PRIME) Program by the
EMA) in a Phase 1/2 trial for hemophilia B being developed in
collaboration with Pfizer, and SPK-8011, in a Phase 1/2 trial for
hemophilia A to which Spark Therapeutics retains global
commercialization rights. To learn more about us and our growing
pipeline, visit www.sparktx.com.
Cautionary note on forward-looking
statementsThis release contains "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995, including statements regarding the
company's product candidates, including voretigene neparvovec,
SPK-7001, SPK-9001 and SPK-8011. Any forward-looking statements are
based on management's current expectations of future events and are
subject to a number of risks and uncertainties that could cause
actual results to differ materially and adversely from those set
forth in, or implied by, such forward-looking statements. These
risks and uncertainties include, but are not limited to, the risk
that: (i) our BLA submitted to the FDA may not be accepted, or, if
accepted, may not be approved; (ii) the data from our Phase 3
clinical trial of voretigene neparvovec may not support labeling
for all biallelic RPE65 mutations other than Leber congenital
amaurosis (LCA); and (iii) the improvements in functional vision
demonstrated by voretigene neparvovec in our clinical trials may
not be sustained over extended periods of time. For a discussion of
other risks and uncertainties, and other important factors, any of
which could cause our actual results to differ from those contained
in the forward-looking statements, see the "Risk Factors" section,
as well as discussions of potential risks, uncertainties and other
important factors, in our Annual Report on Form 10-K, our Quarterly
Reports on Form 10-Q and other filings we make with
the Securities and Exchange Commission. All information in
this press release is as of the date of the release, and Spark
undertakes no duty to update this information unless required by
law.`
Investor Contact:
Ryan Asay
Ryan.asay@sparktx.com
(215) 239-6424
Media Contact:
Monique da Silva
Monique.dasilva@sparktx.com
(215) 282-7470
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