PTC Therapeutics Provides Updates on Translarna™ Regulatory Activities

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PTC Therapeutics, Inc. (NASDAQ: PTCT) provides an update on the regulatory activities for Translarna™ (ataluren) in Europe and the United States. The company has submitted a briefing document for the re-examination of the initial CHMP negative opinion on the renewal of conditional marketing authorization of Translarna in Europe. In the U.S., PTC participated in a Type C meeting with the FDA to discuss a potential path to resubmitting a New Drug Application for Translarna. The company expects a follow-up meeting with the FDA in the first quarter of 2024.

SOUTH PLAINFIELD, N.J., Dec. 5, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today an update on Translarna™ (ataluren) regulatory activities in Europe and the United States.   

As planned, PTC has submitted the briefing document as part of the re-examination of the initial Committee for Medicinal Products for Human Use (CHMP) negative opinion on the renewal of the conditional marketing authorization of Translarna in Europe. The re-examination procedure will focus only on the renewal of the existing conditional marketing authorization. The procedure is expected to last until late January 2024, when an opinion will be provided by CHMP. This opinion will then be sent for adoption by the European Commission, within the following 67 days.

In the United States, PTC recently participated in a Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss a potential path to a resubmission of a New Drug Application (NDA) for Translarna. The discussion focused on the totality of evidence collected to date from the Translarna clinical trials and the STRIDE registry. Based on the discussion, FDA suggested that PTC request a follow-up meeting to align on the specific contents that could support a potential NDA filing. PTC expects this meeting will occur in the first quarter of 2024.

"PTC continues to work to secure global registrations that will enable access to Translarna for patients with nmDMD worldwide," said Matthew B. Klein, M.D., Chief Executive Officer of PTC. "The data collected to date demonstrate the short-and long-term benefits of Translarna on key functional aspects of disease, including ambulatory and neuromuscular function. In Europe, we believe we can address the concerns raised by the CHMP in its initial negative opinion on the renewal of Translarna conditional authorization. In the U.S., we look forward to continuing to work collaboratively with FDA on the contents of a potential NDA resubmission."

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 including Great Britain, Northern Ireland and the European Economic Area 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-20s 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 presentation, 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 commercialization of its products and product candidates; PTC's plans for interactions with the European Medicines Agency (EMA) and US Food and Drug Administration (FDA); the outcome of any re-examination process or meetings with regulatory authorities; the clinical utility and potential advantages of Translarna (ataluren); 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; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Medicines Agency (EMA) determines in the re-examination process that the benefit-risk balance of Translarna authorization supports renewal of such authorization; 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, which was a specific obligation to continued marketing authorization in the EEA; PTC's ability to utilize results from Study 041 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; 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 Translarna.

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.

FAQ

What is the update from PTC Therapeutics, Inc. (NASDAQ: PTCT) regarding Translarna™ (ataluren) regulatory activities?

PTC Therapeutics, Inc. (NASDAQ: PTCT) has submitted a briefing document for the re-examination of the initial CHMP negative opinion on the renewal of conditional marketing authorization of Translarna in Europe. The company also participated in a Type C meeting with the FDA to discuss a potential path to resubmitting a New Drug Application for Translarna.

What is the expected timeline for the re-examination procedure of Translarna in Europe?

The re-examination procedure is expected to last until late January 2024, when an opinion will be provided by CHMP, followed by adoption by the European Commission within the following 67 days.

What is the focus of the discussion with the FDA regarding Translarna in the U.S.?

The discussion with the FDA focused on the totality of evidence collected to date from the Translarna clinical trials and the STRIDE registry, with a suggestion from the FDA to request a follow-up meeting to align on the specific contents that could support a potential NDA filing.

Who commented on the company's efforts regarding Translarna global registrations?

Matthew B. Klein, M.D., Chief Executive Officer of PTC, commented on the company's work to secure global registrations for Translarna, emphasizing the short-and long-term benefits of the drug on key functional aspects of disease.

What are the key functional aspects of disease that Translarna has demonstrated benefits on?

Translarna has demonstrated benefits on ambulatory and neuromuscular function, as highlighted by Matthew B. Klein, M.D., Chief Executive Officer of PTC.