- Label includes full spectrum of PKU patients
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- European launch preparations underway
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WARREN,
N.J., April 25, 2025 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) has issued a positive opinion on
the marketing authorization application for Sephience™
(sepiapterin) for the treatment of children and adults living with
phenylketonuria (PKU). The opinion includes a broad label inclusive
of all ages and disease severities.
"This positive opinion is great news for the PKU patient
community in Europe for whom there
remains a significant unmet medical need," said Matthew B.
Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We look
forward to initiating the European launch immediately following the
adoption of this positive opinion by the European Commission. The
data collected to date continue to support the potential for
Sephience to provide a safe and effective treatment that not only
lowers phenylalanine levels but also enables diet liberalization
even for those with the most severe disease subtypes."
Launch planning in Europe is
well underway with priorities in Germany and other key European markets where
named patient access will be immediately available.
PTC expects the European Commission to ratify the marketing
authorization for Sephience in approximately two months. The
decision will be applicable to all 27 European Union member states,
as well as Iceland, Norway and Liechtenstein.
The New Drug Application (NDA) for sepiapterin is under review
by the U.S. Food and Drug Administration (FDA) with a Prescription
Drug User Fee Act (PDUFA) target action date of July 29, 2025. Review of approval applications is
ongoing in several other countries including Japan and Brazil.
About Sephience™ (sepiapterin)
Sephience, an oral
formulation of synthetic sepiapterin, has a dual mechanism of
action to increase activity of the phenylalanine hydroxylase (PAH)
enzyme. First, Sephience is a precursor compound that is rapidly
absorbed and converted intracellularly to tetrahydrobiopterin
(BH4), a critical cofactor of PAH. Sephience also has an
independent pharmacological chaperone effect, correcting PAH
misfolding to enhance the enzyme function. Through this dual
mechanism of action, Sephience effectively reduces blood
phenylalanine (Phe) levels and has the potential to treat a 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 (Phe). If left untreated or poorly
managed, Phe – 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 PKU
initially don't have any symptoms, but symptoms are usually
progressive, and damage caused by toxic levels of Phe in the first
few years of life is irreversible. Diagnosis of PKU usually takes
place during newborn screening programs. There are an estimated
58,000 people living with PKU globally.
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company that discovers, develops and
commercializes clinically differentiated medicines that provide
benefits to children and adults living with rare disorders. PTC's
ability to innovate new therapies and to globally commercialize
products is the foundation that drives investment in a robust and
diversified pipeline of transformative medicines. To learn more
about PTC, please visit us at www.ptcbio.com and follow
us on Facebook, X, and LinkedIn.
For More Information:
Investors:
Ellen
Cavaleri
+1 (615) 618-6228
ecavaleri@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 clinical trials and studies,
availability of data, regulatory submissions and responses,
commercialization and other matters with respect to its products
and product candidates; expectations with respect to Sephience,
including any regulatory decision made by the European Commission;
PTC's strategy, future operations, future financial position,
future revenues, projected costs; 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
Sephience, including any regulatory submissions and potential
approvals, commercialization and the potential achievement of
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
Sephience.
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.
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SOURCE PTC Therapeutics, Inc.
