FDA grants Priority Review with Prescription Drug
User Fee Act (PDUFA) date of Jan. 12, 2018
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene
therapy company dedicated to challenging the inevitability of
genetic disease, announced today that the U.S. Food and Drug
Administration (FDA) has accepted for filing the Biologics License
Application (BLA) and granted Priority Review for voretigene
neparvovec, an investigational, potential one-time gene therapy
candidate for the treatment of patients with vision loss due to
confirmed biallelic RPE65-mediated inherited retinal disease (IRD).
The investigational gene therapy, which has the proposed trade name
LUXTURNA™ (voretigene neparvovec), has the potential to be both the
first pharmacologic treatment for IRD and the first gene therapy
for a genetic disease in the United States.
Priority Review is granted to therapeutics that would offer
major advances over existing therapies or would provide a treatment
where no adequate therapy exists. FDA’s goal for taking an action
on Priority Review applications is six months. For the review of
LUXTURNA, FDA has assigned a PDUFA date of Jan. 12, 2018.
“FDA acceptance for filing of our BLA for LUXTURNA is an
important development for people living with RPE65-mediated IRD, a
significant milestone for the gene therapy field, and a strong
testament to the dedication of our collaborators and employees,”
said Jeffrey D. Marrazzo, chief executive officer of Spark
Therapeutics. “As we work closely with FDA in the months ahead, we
will remain steadfast in our commitment to bring this important
investigational therapy to people living with RPE65-mediated IRD
who currently have no pharmacologic treatment options.”
Investigational LUXTURNA is intended to be administered one time
per eye in patients with vision loss due to biallelic
RPE65-mediated IRD. A natural history study has shown that people
with this IRD eventually progress to complete blindness.
“Today’s announcement is an important and exciting step forward
in the effort to treat blindness caused by inherited retinal
diseases,” commented Gordon Gund, co-founder and chairman emeritus,
Foundation Fighting Blindness (FFB), a nonprofit organization
focused on research for preventing and treating blindness caused by
IRDs. “While we await the FDA review process, it is fitting to note
that FFB is proud to have been a part of the early support of the
research that led to the development of this potential
therapy.”
LUXTURNA Clinical Trial OverviewThe safety and
efficacy of LUXTURNA were assessed in two open-label Phase 1
trials, which continue to follow participants who received LUXTURNA
between 2007 and 2012, and one open-label, randomized, controlled
Phase 3 trial. Following the one-year control period of the Phase 3
study, all control participants elected to cross over and received
LUXTURNA; long-term safety and efficacy continue to be assessed in
the Phase 3 participants who received LUXTURNA between 2013 and
2015. The clinical trial program included 41 participants with
vision loss aged four to 44 at the time of first administration.
Confirmed biallelic RPE65 mutations and the presence of sufficient
viable retinal cells were established in all participants.
LUXTURNA Phase 3 clinical trial data, including data from the
intent-to-treat population of all randomized participants through
the one-year time point, were published in The Lancet. Results
showed a statistically significant and clinically meaningful
difference between intervention (n=21) and control participants
(n=10) at one year, per the clinical trial’s primary endpoint, mean
bilateral multi-luminance mobility testing (MLMT) change score
(difference of 1.6; 95% CI, 0.72, 2.41; p=0.001). In addition,
participants who received LUXTURNA showed a marked difference
compared to control participants across the first two secondary
endpoints: full-field light sensitivity threshold (FST) testing
(p<0.001) and the mobility test change score for the first
injected eye (p=0.001). A third secondary endpoint, the change in
visual acuity (VA) averaged over both eyes, was not statistically
significant between intervention and control participants
(p=0.17).
On average, participants in the original Phase 3 intervention
group maintained functional gains observed by the day-30 visit
through at least two years, as measured by MLMT and FST. The more
than 100-fold (or greater than two log units) average improvement
in FST testing observed in the original intervention group at one
year, similarly, was maintained through at least two years.
In continuation of the trial to include crossover of the control
group to receive LUXTURNA, 93 percent (27 of 29) of all treated
Phase 3 trial participants saw a gain of functional vision as
assessed by bilateral MLMT over the follow-up period of at least
one year from administration of LUXTURNA to each eye. Additionally,
72 percent (21 of 29) of all Phase 3 trial participants receiving
LUXTURNA successfully completed MLMT at the lowest light level
evaluated (1 lux) at one year.
Data from a cohort of the Phase 1 clinical trial, in which
investigational LUXTURNA was administered to the contralateral, or
second previously uninjected eye, showed mean improvements in
functional vision and visual function. These improvements were
maintained through at least three years, as measured by both MLMT
and FST testing. This cohort of participants (n=8) received the
same dose of LUXTURNA that was administered in the Phase 3 trial
and would have met the Phase 3 eligibility criteria.
No serious adverse events (SAEs) associated with LUXTURNA or
deleterious immune responses have been observed. Two ocular SAEs
were reported in the clinical program. There was one SAE related to
the surgical procedure in one eye of a Phase 3 participant, in
which there was foveal thinning and a sustained reduction in VA.
One additional ocular SAE was reported in one eye of a Phase 1
participant in which the treatment for bacterial endophthalmitis
led to elevated intraocular pressure and subsequent optic atrophy.
There were three non-serious AEs of retinal deposit (subretinal
precipitate) in three participants (three eyes) that were
considered to be related to LUXTURNA. All three of these events
were mild in intensity, transient in nature and resolved without
consequences. The most common adverse reactions related to LUXTURNA
reported in 10 percent or greater of the combined Phase 1 and Phase
3 trial participants included conjunctival hyperemia, cataract,
intraocular pressure increased, and retinal tear.
About RPE65-mediated
Inherited Retinal Disease (IRD)Inherited retinal diseases
(also known as inherited retinal dystrophies) are a group of rare
blinding conditions caused by one of more than 220 different genes.
People living with IRD due to biallelic RPE65 gene mutations often
experience night blindness (nyctalopia) due to decreased light
sensitivity in childhood or early adulthood and involuntary
back-and-forth eye movements (nystagmus). As the disease
progresses, individuals may experience loss in their peripheral
vision, developing tunnel vision, and eventually, they may lose
their central vision as well, resulting in total blindness.
Independent navigation becomes severely limited, and
vision-dependent activities of daily living are impaired. There are
currently no approved pharmacologic treatment options for this
disease.
About Gene TherapyGene therapy is an
investigational approach to treat or prevent genetic disease by
seeking to augment, replace or suppress one or more mutated genes
with functional copies. It addresses the root cause of an inherited
disease by enabling the body to produce a protein or proteins
necessary to restore health or to stop making a harmful protein or
proteins, with the potential of bringing back function in the
diseased cells and slowing disease progression. To deliver the
functional gene into the cell, a vector is used to transport the
desired gene and is delivered either intravenously (IV) or injected
into specific tissue. The goal is to enable, through the one-time
administration of gene therapy, a lasting therapeutic effect.
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.
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); and (iii) the improvements in
functional vision demonstrated by LUXTURNA 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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