Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading
late-stage biotechnology company advancing an integrated and
sustainable pipeline of genetic therapies for rare disorders with
high unmet need, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track and Orphan Drug
designations to RP-A601, the Company’s gene therapy candidate for
the treatment of plakophilin-2 related arrhythmogenic
cardiomyopathy (PKP2-ACM).
RP-A601 is Rocket’s adeno-associated virus (AAV.rh74)-based gene
therapy for PKP2-ACM, a devastating, inherited heart disease
associated with life-threatening arrhythmias, cardiac structural
abnormalities, and sudden cardiac death. The current standard of
care consists of medical therapy, implantable cardioverter
defibrillators (ICDs), and ablation procedures, none of which are
curative. Even with treatment, life-threatening arrhythmias and
progression of disease can still occur. PKP2-ACM affects
approximately 50,000 people in the U.S. and Europe.
Fast Track designation is granted to facilitate the development
and expedite the review of medicines that treat serious conditions
and fill an unmet medical need. The designation enables increased
communication with the FDA throughout the development of RP-A601,
offers the potential for accelerated approval and priority review
if criteria are met, and permits a Rolling BLA Review. Orphan Drug
designation is granted to support the development of medicines for
rare disorders that affect fewer than 200,000 patients in the
U.S.
Rocket recently announced FDA clearance of the Investigational
New Drug (IND) application for RP-A601 based on robust preclinical
proof of concept studies that demonstrated decreased arrhythmias
and increased survival. RP-A601 is the first gene therapy in
development for PKP2-ACM to receive IND clearance and the second of
three programs in the Company’s AAV-based cardiovascular gene
therapy franchise.
The Company is initiating a multi-center Phase 1, dose
escalation trial that will evaluate the safety and preliminary
efficacy of RP-A601 in at least six adult PKP2-ACM patients with
ICDs and who have overall high risk for life-threatening
arrhythmias. The study will assess the impact of RP-A601 on PKP2
myocardial protein expression, cardiac biomarkers, clinical
predictors of life-threatening ventricular arrhythmias, and sudden
cardiac death.
About FDA Fast Track Designation
Fast Track designation is designed to facilitate the development
and expedite the review of drug candidates that are intended to
treat serious conditions and for which nonclinical data has
demonstrated the potential to address unmet medical need. The
program enables increased communication with the FDA for the
purpose of expediting the product's development, review, and
potential approval. Therapies may qualify for accelerated approval
and priority review if criteria are met, in addition to Rolling
Review, which affords the opportunity to submit completed sections
of the Biologic License Application (BLA) for review by the FDA,
rather than waiting until every section is completed before the
entire application can be reviewed.
About FDA Orphan Drug Designation
Orphan Drug designation is granted by the FDA for the treatment,
prevention or diagnosis of diseases that are life-threatening or
chronically debilitating with a prevalence of fewer than 200,000
patients annually. The designation provides certain benefits,
including market exclusivity upon regulatory approval, exemption of
FDA application fees, and tax credits for qualified clinical
trials.
About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
PKP2-ACM is an inherited heart disease caused by mutations in
the PKP2 gene and characterized by life-threatening ventricular
arrhythmias, cardiac structural abnormalities, and sudden cardiac
death. PKP2-ACM affects approximately 50,000 adults and children in
the U.S. and Europe. Patients living with PKP2-ACM have an urgent
unmet medical need, as current medical, implantable cardioverter
defibrillator (ICD), and ablation therapies do not consistently
prevent disease progression or arrhythmia recurrence, are
associated with significant morbidity including inappropriate
shocks and device and procedure-related complications, and do not
address the underlying pathophysiology or genetic mutation. RP-A601
is being investigated as a one-time, potentially curative gene
therapy treatment that may improve survival and quality of life for
patients affected by this devastating disease.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an
integrated and sustainable pipeline of investigational genetic
therapies designed to correct the root cause of complex and rare
childhood disorders. The Company’s platform-agnostic approach
enables it to design the best therapy for each indication, creating
potentially transformative options for patients afflicted with rare
genetic diseases. Rocket's clinical programs using lentiviral
vector (LVV)-based gene therapy are for the treatment of Fanconi
Anemia (FA), a difficult to treat genetic disease that leads to
bone marrow failure and potentially cancer, Leukocyte Adhesion
Deficiency-I (LAD-I), a severe pediatric genetic disorder that
causes recurrent and life-threatening infections which are
frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare,
monogenic red blood cell disorder resulting in increased red cell
destruction and mild to life-threatening anemia. Rocket’s first
clinical program using adeno-associated virus (AAV)-based gene
therapy is for Danon Disease, a devastating, pediatric heart
failure condition. Rocket also has received IND clearance for the
AAV-based gene therapy program for PKP2-arrhythmogenic
cardiomyopathy (ACM) and is advancing a preclinical program for
BAG3-associated dilated cardiomyopathy (DCM). For more information
about Rocket, please visit www.rocketpharma.com.
Rocket Cautionary Statement Regarding Forward-Looking
Statements
Various statements in this release concerning Rocket’s future
expectations, plans and prospects, including without limitation,
Rocket’s expectations regarding the safety and effectiveness of
product candidates that Rocket is developing to treat Fanconi
Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate
Kinase Deficiency (PKD), Danon Disease (DD), PKP2-ACM and other
diseases, the expected timing and data readouts of Rocket’s ongoing
and planned clinical trials, the expected timing and outcome of
Rocket’s regulatory interactions and planned submissions, Rocket’s
plans for the advancement of its Danon Disease program, including
its planned pivotal trial, and the safety, effectiveness and timing
of related pre-clinical studies and clinical trials, may constitute
forward-looking statements for the purposes of the safe harbor
provisions under the Private Securities Litigation Reform Act of
1995 and other federal securities laws and are subject to
substantial risks, uncertainties and assumptions. You should not
place reliance on these forward-looking statements, which often
include words such as “aim,” “anticipate,” "believe," “can,”
“continue,” “design,” “estimate,” "expect," "intend," “may,”
"plan," “potential,” "will give," "seek," "will," "may," "suggest"
or similar terms, variations of such terms or the negative of those
terms. Although Rocket believes that the expectations reflected in
the forward-looking statements are reasonable, Rocket cannot
guarantee such outcomes. Actual results may differ materially from
those indicated by these forward-looking statements as a result of
various important factors, including, without limitation, Rocket’s
ability to monitor the impact of COVID-19 on its business
operations and take steps to ensure the safety of patients,
families and employees, the interest from patients and families for
participation in each of Rocket’s ongoing trials, patient
enrollment, trial timelines and data readouts, our expectations
regarding our drug supply for our ongoing and anticipated trials,
actions of regulatory agencies, which may affect the initiation,
timing and progress of pre-clinical studies and clinical trials of
its product candidates, our ability to submit regulatory filings
with the U.S. Food and Drug Administration (FDA) and to obtain and
maintain FDA or other regulatory authority approval of our product
candidates, Rocket’s dependence on third parties for development,
manufacture, marketing, sales and distribution of product
candidates, the outcome of litigation, our competitors’ activities,
including decisions as to the timing of competing product launches,
pricing and discounting, our integration of an acquired business,
which involves a number of risks, including the possibility that
the integration process could result in the loss of key employees,
the disruption of our ongoing business, or inconsistencies in
standards, controls, procedures, or policies, our ability to
successfully develop and commercialize any technology that we may
in-license or products we may acquire and any unexpected
expenditures, as well as those risks more fully discussed in the
section entitled "Risk Factors" in Rocket’s Annual Report on Form
10-K for the year ended December 31, 2022, filed February 28, 2023
with the SEC and subsequent filings with the SEC including our
Quarterly Reports on Form 10-Q. Accordingly, you should not place
undue reliance on these forward-looking statements. All such
statements speak only as of the date made, and Rocket undertakes no
obligation to update or revise publicly any forward-looking
statements, whether as a result of new information, future events
or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20230608005337/en/
Media Kevin Giordano kgiordano@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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