- Review of European marketing application for
PKU now initiated -
WARREN,
N.J., May 28, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the
sepiapterin MAA for PKU has been validated and accepted for review
by the EMA.
"The validation of the MAA is an important step towards making
sepiapterin available to children and adults affected by PKU in
Europe," said Matthew B. Klein, M.D., Chief Executive Officer
of PTC Therapeutics. "The European submission is the first of
several global submissions planned in 2024."
PTC expects to submit the sepiapterin NDA to the FDA no later
than the third quarter of 2024. In addition, submissions are
planned in a number of additional countries in 2024 including
Brazil and Japan.
The sepiapterin MAA includes data from the phase 3 APHENITY
trial which demonstrated a mean reduction in Phe levels of 63% in
the overall treated population and 69% in the subgroup of subjects
with classical PKU. The vast majority of subjects (84%) achieved
Phe control in accordance with treatment guidelines of <360
µmol/L, and 22% of subjects had normalization of Phe levels. The
application also includes data from the APHENITY open-label
extension study which provides evidence of sepiapterin durability
of effect as well as data from the Phe tolerance sub-study. Phe
tolerance sub-study results collected to date demonstrate that
subjects are liberalizing their diet, beyond the recommended daily
allowance of protein, and maintaining control of Phe within
guideline target levels of <360 μmol/L. The ability to enable
patients to liberalize their highly restrictive diets and still
maintain Phe control is a very meaningful sepiapterin treatment
benefit for patients as well as for physicians and payers.
About Sepiapterin
Sepiapterin (formerly PTC923) is an
oral formulation of synthetic sepiapterin, a precursor to
intracellular tetrahydrobiopterin, which is a critical enzymatic
cofactor involved in the metabolism and synthesis of numerous
metabolic products. Sepiapterin is a more bioavailable precursor
than exogenously administered synthetic BH4 and has the potential
to treat the broad range of PKU patients.
About Phenylketonuria
Phenylketonuria (PKU) is a rare,
inherited metabolic disease, which affects the brain. It is caused
by a defect in the gene that helps create the enzyme needed to
break down phenylalanine. If left untreated or poorly managed,
phenylalanine – an essential amino acid found in all proteins and
most foods – can build up to harmful levels in the body. This
causes severe and irreversible disabilities, such as permanent
intellectual disability, seizures, delayed development, memory
loss, and behavioral and emotional problems. Newborns with
phenylketonuria initially don't have any symptoms, but symptoms are
usually progressive, and damage caused by toxic levels of
phenylalanine in the first few years of life is irreversible.
Diagnosis of phenylketonuria usually takes place during newborn
screening programs. There are an estimated 58,000 people with
phenylketonuria globally.
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; expectations with respect to
sepiapterin, including any regulatory submissions and potential
approvals, commercialization, 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 Quarterly Report on Form 10-Q and 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
sepiapterin.
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:
EMA: European Medicines Agency
FDA: U.S. Food and Drug Administration
MAA: Marketing Authorization Application
NDA: New Drug Application
Phe: Phenylalanine
PKU: Phenylketonuria
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SOURCE PTC Therapeutics, Inc.