Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application to the U.S. Food and Drug Administration
27 Dezember 2023 - 1:30PM
Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) a rare disease
therapeutics company, today announced it resubmitted its New Drug
Application (NDA) for arimoclomol, an investigational therapeutic
candidate for the treatment of Niemann-Pick disease type C (NPC) to
the U.S. Food and Drug Administration (FDA) on December 22, 2023.
Based on standard NDA resubmission review timelines, an
acknowledgment letter from the FDA that the resubmission is
complete and setting the PDUFA date is expected within 30 days.
Zevra expects the NDA to be classified as a Class II submission
which would be subject to a review period by the FDA within six
months from the date of submission.
“The Zevra team has worked diligently to deliver
a high quality and thorough resubmission of the NDA for arimoclomol
following multiple interactions with the FDA and after
incorporating direction from the agency,” said Neil McFarlane,
President and Chief Executive Officer of Zevra. “We continue to
accelerate our launch preparations in anticipation of FDA approval,
and believe we are one step closer to getting arimoclomol into the
hands of patients who are seeking a treatment.”
“Zevra has engaged with the advocacy community,
elevating the patient voice throughout arimoclomol’s development
process,” said Daniel Gallo, Ph.D., Zevra’s Senior Vice President
of Medical Affairs and Advocacy. “The advocacy community’s input
has been instrumental in building awareness of the need for
approved treatments that address the unmet needs of individuals and
their caregivers living with this debilitating condition.”
Zevra believes it has addressed the issues
previously raised by the FDA in the complete response letter by
providing additional evidence to support the use of the
Niemann-Pick type C Clinical Severity Scale (NPCCSS) and,
conducting additional studies used to support the potential
mechanism of action. Additionally, new data included in the
resubmission comes from multiple non-clinical studies, natural
history comparisons, real-world data generated from the ongoing
early access programs in the United States and the European Union,
as well as data from the four-year open-label extension of the
Phase 2/3 clinical trial (NCT02612129). Results from this
open-label trial suggest that arimoclomol reduces the long-term
progression of NPC.
Arimoclomol has been evaluated in a total of 21
studies across a range of Phase 1, 2 or 3 clinical trials
evaluating its safety and efficacy across more than 600 subjects in
NPC, other disease or healthy subjects. The primary efficacy trial
evaluating arimoclomol for the treatment of NPC was a Phase 2/3
double-blind, placebo-controlled trial (CT-ORZY-NPC-002) of
arimoclomol in 50 patients with NPC.
About Niemann-Pick disease type
C:Niemann-Pick disease type C (NPC) is an ultra-rare,
genetic, progressive and fatal neurological disease caused by
mutations in the NPC1 or NPC2 genes. Both genes encode proteins,
located in intracellular compartments called lysosomes, which are
essential in the transport and metabolism of lipids. Mutations in
either of these NPC genes result in a reduced amount of NPC1 or
NPC2 protein causing lysosomal dysfunction due to accumulation of
intracellular lipids and ultimately, if unchecked, cell death.
Evidence suggests that arimoclomol improves lysosomal function
within the cells, reducing the accumulation of lipids to delay and
prevent cell death. NPC is an inherited disorder that affects both
children and adults with varying ages of onset, rate of progression
and presents differently in each person. NPC is characterized by
visceral (internal organs) manifestations, including enlarged liver
and spleen, neurological and psychiatric manifestations. Those
living with NPC lose independence due to physical and cognitive
limitations. Key neurological impairments are in speech, cognition,
swallowing, ambulation, and fine motor skills. Disease progression
is irreversible and can be fatal within months or take years to
diagnose and advance.
About Arimoclomol:Arimoclomol,
Zevra’s orally-delivered, first-in-class investigational product
candidate for the treatment of Niemann-Pick disease type C (NPC),
has been granted orphan drug designation, Fast Track designation,
Breakthrough Therapy designation and rare pediatric disease
designation for NPC by the U.S. Food and Drug Administration (FDA),
and orphan medicinal product designation for the treatment of NPC
by the European Medicines Agency (EMA). Zevra resubmitted
the NDA to the FDA in December 2023.
About Zevra Therapeutics:Zevra
Therapeutics is a rare disease company melding science, data, and
patient needs to create transformational therapies for diseases
with limited or no treatment options. With unique, data-driven
clinical, regulatory, and commercialization strategies, the Company
is overcoming complex drug development challenges to bring
much-needed therapies to patients. With both regulatory and
clinical stage product candidates, the Company is building its
commercial capability to make new therapies available to the rare
disease community.
Caution Concerning Forward-Looking
Statements:This press release may contain forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Forward-looking statements include all
statements that do not relate solely to historical or current
facts, including without limitation statements regarding the
promise and potential impact of our preclinical or clinical trial
data, including without limitation the initiation, timing and
results of any clinical trials or readouts, the content,
information used for, timing or results of any NDA submissions or
resubmissions for arimoclomol or any other product candidates for
any specific disease indication or at any dosage, the potential
launch or commercialization of any of product candidates or
products, and our strategic and product development objectives,
including with respect to becoming a leading, commercially focused
rare disease company. Forward-looking statements are based on
information currently available to Zevra and its current plans or
expectations. They are subject to several known and unknown
uncertainties, risks, and other important factors that may cause
our actual results, performance, or achievements to be materially
different from any future results, performance, or achievements
expressed or implied by the forward-looking statements. These and
other important factors are described in detail in the “Risk
Factors” section of Zevra’s Annual Report on Form 10-K for the year
ended December 31, 2022, as updated in Zevra’s Quarterly Report on
Form 10-Q for the quarter ended September 30, 2023, and Zevra’s
other filings with the Securities and Exchange Commission. While we
may elect to update such forward-looking statements at some point
in the future, except as required by law, we disclaim any
obligation to do so, even if subsequent events cause our views to
change. Although we believe the expectations reflected in such
forward-looking statements are reasonable, we cannot assure that
such expectations will prove correct. These forward-looking
statements should not be relied upon as representing our views as
of any date after the date of this press release.
Investor Contact:
Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com
Media Contacts:
David Schull or Ignacio Guerrero-Ros, Ph.D.Russo
Partners+1 (858) 717-2310David@russopr.comIgnacio@russopr.com
Zevra Therapeutics (NASDAQ:ZVRA)
Historical Stock Chart
Von Mai 2024 bis Jun 2024
Zevra Therapeutics (NASDAQ:ZVRA)
Historical Stock Chart
Von Jun 2023 bis Jun 2024