- First marketing authorization submission for
sepiapterin with additional global submissions to follow in 2024
-
SOUTH
PLAINFIELD, N.J., March 28,
2024 /PRNewswire/ -- PTC Therapeutics, Inc.
(NASDAQ: PTCT) announced today the submission of the sepiapterin
MAA to the European Medicines Agency. The MAA submission is for the
treatment of pediatric and adult patients with PKU, including the
full spectrum of disease subtypes.
"This MAA submission is the first in a series of global
submissions for sepiapterin as we move closer to bringing this
therapy to children and adults with PKU worldwide," said
Matthew B. Klein, M.D., Chief
Executive Officer of PTC Therapeutics. "The strength of the
sepiapterin data generated in clinical trials substantiate
how differentiated sepiapterin is from currently available
therapies and support how sepiapterin could address the significant
unmet medical need for PKU patients."
The sepiapterin MAA includes the results of the phase 3 APHENITY
trial in which sepiapterin had a statistically significant and
clinically meaningful reduction in blood phenylalanine levels in
pediatric and adult PKU patients. Sepiapterin treatment resulted in
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, and 22% of subjects had
normalization of Phe levels. Data from the ongoing APHENITY
open-label extension study demonstrates that these sepiapterin
treatment effects are durable. In addition, data from the Phe
tolerance sub study demonstrate that subjects are liberalizing
their diet, beyond the recommended daily allowance of protein, and
maintaining control of Phe within target levels from guidelines.
The ability to enable patients to liberalize their highly
restrictive diets and still maintain Phe control, is a very
meaningful sepiapterin treatment benefit for physicians, payers and
patients.
PTC expects to submit the sepiapterin NDA to the FDA no later
than the third quarter of 2024. Submissions in 2024 are planned in
a number of additional key countries where PTC has existing rare
disease commercial infrastructure including Brazil and Japan.
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.1 It is caused
by a defect in the gene that helps create the enzyme needed to
break down phenylalanine.1 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.1 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.2,3
Diagnosis of phenylketonuria usually takes place during newborn
screening programs.4 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 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
PKU: Phenylketonuria
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SOURCE PTC Therapeutics, Inc.