- BLA submitted to FDA for Upstaza™ for
the treatment of AADC deficiency -
- NDA to be submitted mid-year for
Translarna™ based on FDA feedback -
SOUTH PLAINFIELD,
N.J., March 19, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of
a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a
gene therapy for the treatment of aromatic L-amino acid
decarboxylase (AADC) deficiency. In addition, based on recent
feedback from the FDA, PTC plans to re-submit an NDA for
Translarna™ (ataluren) for the treatment of nonsense mutation
Duchenne muscular dystrophy (nmDMD). PTC expects to make the NDA
resubmission by mid-year.
"We are excited to bring Upstaza one step closer to children in
the United States with the highly
mortal and fatal disease of AADC deficiency. The data collected to
date continue to demonstrate the transformative clinical benefits
the gene therapy provides to patients," said Matthew B. Klein, M.D., Chief Executive Officer
of PTC Therapeutics. "In addition, we look forward to the
Translarna NDA resubmission this summer. We know U.S. patients and
families have been waiting for an approved therapy that specially
targets nmDMD."
In addition, the sepiapterin MAA for PKU remains on schedule for
submission to the EMA by the end of this month.
About Upstaza™ (eladocagene
exuparvovec)
Upstaza is a one-time gene replacement therapy
indicated for the treatment of patients aged 18 months and older
with a clinical, molecular, and genetically confirmed diagnosis of
aromatic L–amino acid decarboxylase (AADC) deficiency with a severe
phenotype. It is a recombinant adeno-associated virus serotype 2
(AAV2)-based gene therapy, containing the
human DDC gene.1 It is designed to
correct the underlying genetic defect, by delivering a
functioning DDC gene directly into the putamen,
increasing the AADC enzyme and restoring dopamine
production.2,3
The efficacy and safety profile of Upstaza has been demonstrated
across clinical trials and compassionate use
programs.1 The first patient was dosed in 2010. In
clinical trials, Upstaza demonstrated transformational neurological
improvements. The most common side effects were initial insomnia,
irritability and dyskinesia.
Administration of Upstaza occurs through a stereotactic
surgical procedure, a minimally invasive neurosurgical procedure
used for the treatment of a number of pediatric and adult
neurological disorders. The Upstaza administration procedure is
performed by a qualified neurosurgeon in centers specialized in
stereotactic neurosurgery.
Upstaza has received marketing authorization in Europe, Great
Britain and Israel.
About aromatic L-amino acid decarboxylase (AADC)
deficiency
AADC deficiency is a fatal, rare genetic disorder
that typically causes severe disability and suffering from the
first months of life, affecting every aspect of life – physical,
mental and behavioral. The suffering of children with AADC
deficiency may be exacerbated by: episodes of distressing
seizure-like oculogyric crises causing the eyes to roll up in the
head, frequent vomiting, behavioral problems, and difficulty
sleeping.
The lives of affected children are severely impacted and
shortened. Ongoing physical, occupational and speech therapy, and
interventions, including surgery, also are often required to manage
potentially life-threatening complications such as infections,
severe feeding and breathing problems.
About Translarna™ (ataluren)
Translarna (ataluren),
discovered and developed by PTC Therapeutics, is a protein
restoration therapy designed to enable the formation of a
functioning protein in patients with genetic disorders caused by a
nonsense mutation. A nonsense mutation is an alteration in the
genetic code that prematurely halts the synthesis of an essential
protein. The resulting disorder is determined by which protein
cannot be expressed in its entirety and is no longer functional,
such as dystrophin in Duchenne. Translarna, the tradename of
ataluren, is licensed in multiple countries for the treatment of
nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory
patients aged 2 years and older. Ataluren is an investigational new
drug in the United States.
About Duchenne Muscular Dystrophy (Duchenne)
Primarily
affecting males, Duchenne is a rare and fatal genetic disorder that
results in progressive muscle weakness from early childhood and
leads to premature death in the mid-20's due to heart and
respiratory failure. It is a progressive muscle disorder caused by
the lack of functional dystrophin protein. Dystrophin is critical
to the structural stability of all muscles, including skeletal,
diaphragm, and heart muscles. Patients with Duchenne can lose the
ability to walk (loss of ambulation) as early as 10 years old,
followed by loss of the use of their arms. Duchenne patients
subsequently experience life-threatening lung complications,
requiring the need for ventilation support, and heart complications
in their late teens and 20s.
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically differentiated medicines that
provide benefits to patients with rare disorders. PTC's ability to
innovate to identify new therapies and to globally commercialize
products is the foundation that drives investment in a robust and
diversified pipeline of transformative medicines. PTC's mission is
to provide access to best-in-class treatments for patients who have
little to no treatment options. PTC's strategy is to leverage its
strong scientific and clinical expertise and global commercial
infrastructure to bring therapies to patients. PTC believes this
allows it to maximize value for all its stakeholders. To learn more
about PTC, please visit us at www.ptcbio.com and
follow us on Facebook, Instagram, LinkedIn and Twitter at
@PTCBio.
For More Information:
Investors:
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
Jeanine
Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Forward-Looking Statement
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. All statements contained in this release, other than
statements of historic fact, are forward-looking statements,
including statements regarding: the future expectations, plans and
prospects for PTC, including with respect to the expected timing of
regulatory submissions and responses, commercialization and other
matters with respect to its products and product candidates; PTC's
strategy, future operations, future financial position, future
revenues, projected costs; the extent, timing and financial aspects
of our strategic pipeline prioritization and reductions in
workforce; and the objectives of management. Other forward-looking
statements may be identified by the words, "guidance", "plan,"
"anticipate," "believe," "estimate," "expect," "intend," "may,"
"target," "potential," "will," "would," "could," "should,"
"continue," and similar expressions.
PTC's actual results, performance or achievements could differ
materially from those expressed or implied by forward-looking
statements it makes as a result of a variety of risks and
uncertainties, including those related to: the outcome of pricing,
coverage and reimbursement negotiations with third party payors for
PTC's products or product candidates that PTC commercializes or may
commercialize in the future; PTC's ability to use the results of
Study 041, a randomized, 18-month, placebo-controlled clinical
trial of Translarna for the treatment of nmDMD followed by an
18-month open-label extension, and from its international drug
registry study to support a marketing approval for Translarna for
the treatment of nmDMD in the United
States; whether investigators agree with PTC's
interpretation of the results of clinical trials and the totality
of clinical data from our trials in Translarna; expectations with
respect to Upstaza, including any regulatory submissions and
potential approvals, commercialization, manufacturing capabilities,
the potential achievement of development, regulatory and sales
milestones and contingent payments that PTC may be obligated to
make; significant business effects, including the effects of
industry, market, economic, political or regulatory conditions;
changes in tax and other laws, regulations, rates and policies; the
eligible patient base and commercial potential of PTC's products
and product candidates; PTC's scientific approach and general
development progress; and the factors discussed in the "Risk
Factors" section of PTC's most recent Annual Report on Form 10-K,
as well as any updates to these risk factors filed from time to
time in PTC's other filings with the SEC. You are urged to
carefully consider all such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Translarna and Upstaza.
The forward-looking statements contained herein represent PTC's
views only as of the date of this press release and PTC does not
undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
Acronyms:
BLA: Biologics License Application
DMD: Duchenne Muscular Dystrophy
EMA: European Medicines Agency
FDA: U.S. Food and Drug Administration
MAA: Marketing Authorization Application
NDA: New Drug Application
PKU: Phenylketonuria
References:
- Tai CH, et al. Long-term efficacy and safety
of eladocagene exuparvovec in patients with AADC
deficiency. Mol Ther. 2022;30(2):509-518.
- Chien et al. AGIL-AADC gene therapy results in
sustained improvements in motor and developmental milestones
through 5 years in children with AADC deficiency. Poster presented
at the 48th Annual Meeting of the Child
Neurology Society, Charlotte, NC, USA, Oct 23-26,
2019.
- Chien YH, et al. Efficacy and safety of AAV2
gene therapy in children with aromatic L-amino acid decarboxylase
deficiency: an open-label, phase 1/2 trial. Lancet Child
Adolesc Health. 2017;1(4):265-273.
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SOURCE PTC Therapeutics, Inc.