PTC Therapeutics (PTCT) withdraws U.S. Translarna NDA after FDA feedback

Rhea-AI Filing Summary

PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) as a treatment for nonsense mutation Duchenne muscular dystrophy after U.S. FDA feedback on the review. FDA indicated the data are unlikely to meet its standard of substantial evidence of effectiveness needed for approval.

CEO Matthew B. Klein said the company is disappointed that FDA approval for Translarna in the U.S. “cannot be achieved” despite more than two decades of development work. The update underscores the challenges of securing U.S. approval for this rare disease therapy, even as PTC continues broader work on treatments for genetic disorders.

Positive

• None.

Negative

• Translarna U.S. regulatory setback: PTC withdrew its NDA resubmission for Translarna in nonsense mutation Duchenne muscular dystrophy after FDA said the data are unlikely to meet its effectiveness threshold for approval.

Insights

Biopharma regulatory analyst

FDA feedback led PTC to withdraw its Translarna NDA resubmission, removing a potential U.S. approval path for this DMD therapy.

PTC reports that FDA, based on its review to date, considers the Translarna data unlikely to meet the required “substantial evidence of effectiveness” standard. In response, the company has chosen to withdraw the U.S. NDA resubmission for nonsense mutation Duchenne muscular dystrophy.

This is a material setback for Translarna’s U.S. prospects, as it halts the current approval effort rather than extending review. The press release also references broader regulatory and reimbursement risks for PTC’s products, highlighting continued uncertainty that investors will need to evaluate through future company disclosures.

0001070081false00010700812026-02-122026-02-12

​

​

UNITED STATES

​

SECURITIES AND EXCHANGE COMMISSION

​

WASHINGTON, D.C. 20549

​

FORM 8-K

​

CURRENT REPORT

​

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

​

Date of Report (Date of earliest event reported): February 12, 2026

​

PTC THERAPEUTICS, INC.

​

(Exact Name of Company as Specified in Charter)

​

​	​	​	​	​
Delaware	​ ​ ​	001-35969	​ ​ ​	04-3416587
(State or Other Jurisdiction	​	(Commission	​	(IRS Employer
of Incorporation)	​	File Number)	​	Identification No.)

​

​

​	​	​	​
500 Warren Corporate Center Drive	​	​ ​ ​	​
Warren, NJ	​	​	07059
(Address of Principal Executive Offices)	​	​	(Zip Code)

​

Registrant’s telephone number, including area code: (908) 222-7000

​

Not applicable

(Former Name or Former Address, if Changed Since Last Report)

​

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

​

☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

​

☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

​

☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

​

☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

​

Securities registered pursuant to Section 12(b) of the Act:

​

​	​	​	​	​
Title of each class	​ ​ ​	Trading Symbol(s)	​ ​ ​	Name of each exchange on which registered
Common Stock, $0.001 par value per share	​	PTCT	​	Nasdaq Global Select Market

​

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

​

Emerging growth company ☐

​

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

​

​

​

​

Item 7.01. Regulation FD Disclosure.

On February 12, 2026, PTC Therapeutics, Inc. issued a press release announcing that it has withdrawn the New Drug Application resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy following U.S. Food and Drug Administration feedback on the application review. The press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K (this “Report”) and is incorporated by reference into this Item 7.01.

The information in this Item 7.01 of this Report, including Exhibit 99.1 shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

​	​	​
Exhibit No.	​ ​ ​	Description
99.1	​	Press Release, dated February 12, 2026 issued by PTC Therapeutics, Inc.
104	​	The cover page from this Current Report on Form 8-K, formatted in Inline XBRL

​

​

​

​

​

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.

​	​	​
​	PTC Therapeutics, Inc.
​	​
Date: February 12, 2026	By:	/s/ Pierre Gravier
​	Name:	Pierre Gravier
​	Title:	Chief Financial Officer

​

​

Exhibit 99.1

PTC Therapeutics Provides Regulatory Update on Translarna™

​

​

WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S. Food and Drug Administration (FDA) feedback on the application review.

​

“FDA shared that based on its review to date, the data in the NDA submission are unlikely to meet the Agency’s threshold of substantial evidence of effectiveness to support approval of Translarna. We have therefore made the decision to withdraw the NDA submission,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “We have worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense mutation DMD in the U.S. and are disappointed that FDA approval cannot be achieved.”

​
​​

​

About Translarna™ (ataluren)
Translarna (ataluren), 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.

​

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 dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit www.ptcbio.com and follow 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; 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 geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the U.S. Translarna NDA on other regulatory bodies; 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 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.

FAQ

What did PTC Therapeutics (PTCT) announce about Translarna in this 8-K?

PTC Therapeutics announced it has withdrawn the New Drug Application resubmission for Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy after U.S. FDA feedback indicated the data are unlikely to meet the Agency’s effectiveness standard for approval.

Why did PTC Therapeutics withdraw the Translarna NDA resubmission?

PTC withdrew the Translarna NDA resubmission because the FDA shared that, based on its review to date, the data are unlikely to meet the threshold of substantial evidence of effectiveness required to support U.S. approval for nonsense mutation Duchenne muscular dystrophy.

What condition was Translarna intended to treat according to PTC Therapeutics?

Translarna was intended to treat nonsense mutation Duchenne muscular dystrophy, a rare, progressive, and fatal genetic disorder that causes muscle weakness from early childhood and typically leads to premature death in the mid-20s due to heart and respiratory failure.

How does PTC Therapeutics describe Translarna’s mechanism of action?

PTC describes Translarna (ataluren) as a protein restoration therapy designed to enable production of a functioning protein in genetic disorders caused by a nonsense mutation, such as restoring dystrophin in Duchenne muscular dystrophy, where a premature stop in the genetic code prevents full protein synthesis.

What did PTC’s CEO say about the FDA decision on Translarna?

CEO Matthew B. Klein said FDA indicated the data are unlikely to meet its effectiveness threshold, and PTC therefore chose to withdraw the NDA. He noted the company has worked for over two decades and is disappointed that U.S. approval for Translarna cannot be achieved.

What broader risks does PTC Therapeutics highlight in the Translarna update?

PTC highlights risks related to maintaining marketing authorizations, pricing, coverage and reimbursement, and the potential impact of European and U.S. regulatory outcomes for Translarna on other regulators, alongside general uncertainties in its development, regulatory, and commercialization activities.

Filing Exhibits & Attachments

5 documents

Press Releases

• EX-99.1 EX-99.1 12.9 KB

Other Documents

• EX-101 EX-101.SCH 3.3 KB
• EX-101 EX-101.DEF 2.7 KB
• EX-101 EX-101.LAB 16.2 KB
• EX-101 EX-101.PRE 9.4 KB