PTC TherapeuticsPTC Therapeutics Announces Validation of Sepiapterin European MAA
28 May 2024
Clinical ResultNDAPhase 3
- Review of European marketing application for PKU now initiated -
WARREN, N.J., May 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc.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 TherapeuticsPTC 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 www.ptcbio.com and follow us on Facebook, Instagram, LinkedIn and Twitter at @PTCBio.
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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:
MAA: Marketing Authorization Application
NDA: New Drug Application
Phe: Phenylalanine
PKU: Phenylketonuria
SOURCE PTC Therapeutics, Inc.
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