Spark Therapeutics Receives Rare Pediatric Disease Designation for Investigational LUXTURNA™ (voretigene neparvovec) from F...
July 20 2017 - 7:30AM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene
therapy company dedicated to challenging the inevitability of
genetic disease, announced today that the Offices of Orphan
Products Development and Pediatric Therapeutics of the U.S. Food
and Drug Administration (FDA) have designated investigational
LUXTURNATM (voretigene neparvovec) as a drug for a rare pediatric
disease. Under FDA's Rare Pediatric Disease Priority Review Voucher
program, companies who receive approval for a new drug application
or Biologics License Application (BLA) for a rare pediatric disease
may be eligible to receive a voucher for a Priority
Review of a subsequent marketing application for a different
product. The Priority Review Voucher may be used by the
company or sold to a third party.
FDA’s Office of Tissues and Advanced Therapies currently is
reviewing the BLA for LUXTURNA for the treatment of patients with
vision loss due to confirmed biallelic RPE65 mutation-associated
retinal dystrophy under a Priority Review designation with a
Prescription Drug User Fee Act (PDUFA) action date of Jan. 12,
2018.
LUXTURNA has the potential to be both the first pharmacologic
treatment for an inherited retinal disease (IRD) and the first gene
therapy for a genetic disease approved in the United States. A
natural history study has shown that people with this IRD
eventually progress to complete blindness.
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, with proposed trade name
LUXTURNA™ (voretigene neparvovec), is currently under Priority
Review with FDA for the treatment of biallelic RPE65-mediated IRD
and has been designated for a rare pediatric disease. It previously
received breakthrough therapy and orphan product designations from
FDA and orphan product designations from the European
Medicines Agency (EMA). 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 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. For more
information, 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 candidate LUXTURNA™ (voretigene neparvovec).
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 for LUXTURNA to the FDA may not be approved; (ii) the
data from our Phase 3 clinical trial of LUXTURNA may not support
labeling for all biallelic RPE65 mutations other than Leber
congenital amaurosis (LCA); (iii) the improvements in functional
vision demonstrated by LUXTURNA in our clinical trials may not be
sustained over extended periods of time; and (iv) FDA may
ultimately determine not to award the Company a priority review
voucher. 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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