Welcome to our dedicated page for Ptc Therapeutics news (Ticker: PTCT), a resource for investors and traders seeking the latest updates and insights on Ptc Therapeutics stock.
PTC Therapeutics develops and commercializes medicines for children and adults with rare disorders, with a life-science business focused on clinically differentiated therapies in areas including neurology and metabolism. Company news commonly covers product revenue, commercial launch activity, financial guidance, investor presentations, and updates from its rare-disease development portfolio.
Recurring developments include Sephience™ (sepiapterin) launch and revenue updates, clinical and regulatory disclosures involving votoplam, vatiquinone and Translarna™ (ataluren), and partnership-related program responsibilities. PTC also reports governance changes and Nasdaq inducement equity grants tied to employee compensation.
Phenylketonuria market (includes BioMarin - BMRN) is forecast to expand through 2034 driven by rising diagnosis rates, expanded newborn screening, and multiple emerging therapies.
Key datapoints: US had 18,800 diagnosed PKU cases in 2024; sepiapterin received regulatory approvals in 2025 and pegvaliase sBLA entered FDA review.
PTC Therapeutics (NASDAQ: PTCT) announced on February 12, 2026 that it has withdrawn the New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (DMD) following feedback from the U.S. Food and Drug Administration.
According to the company, FDA reviewers indicated the NDA data are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval, and PTC therefore withdrew the submission; leadership expressed disappointment after more than two decades of development.
PTC Therapeutics (NASDAQ: PTCT) will report fourth quarter and full year 2025 financial results on Thursday, Feb. 19, 2026 at 4:30 p.m. ET. The company will host a webcast conference call to discuss results, business updates, and outlook.
Investors can register for phone access or join the webcast on the company's Investors site. A replay will be archived on the company website for 30 days.
PTC Therapeutics (NASDAQ: PTCT) reported unaudited 2025 product and royalty revenue of approximately $823.4M, with unaudited product revenue of about $587.8M. Sephience generated unaudited Q4 2025 net revenue of approximately $92.5M and total 2025 net revenue since launch of $112.1M. PTC ended 2025 with approximately $1.94B in cash, cash equivalents and marketable securities. In December 2025 PTC sold remaining Evrysdi royalty rights for $240M upfront plus up to $60M in milestones while retaining a separate $150M milestone right. For 2026 PTC guides total product revenue of $700–$800M (ex-Evrysdi royalties), GAAP R&D and SG&A of $775–$815M, and non-GAAP R&D and SG&A of $680–$720M. Clinical updates include Phase 3 alignment and Accelerated Approval openness for votoplam HD and FDA follow-up requests for vatiquinone MOVE-FA.
PTC Therapeutics (NASDAQ: PTCT) approved an inducement equity award on Jan. 2, 2026 granting 300 restricted stock units (RSUs) to a new non-executive employee. Each RSU represents the right to one share of common stock upon vesting. The award was approved by the company's Compensation Committee and made under the Nasdaq inducement grant exception (Listing Rule 5635(c)(4)) as part of the employee's hire compensation. The RSUs vest over four years, with 25% vesting on each annual anniversary of the hire date, subject to continued service through each vesting date.
PTC Therapeutics (NASDAQ: PTCT) announced MHLW approval of Sephience (sepiapterin) in Japan for the treatment of children and adults living with phenylketonuria (PKU).
The Japanese label covers all ages and the full spectrum of disease severity. This is PTC's first product approval in Japan and follows approvals in the EU and US within less than six months, enabling a coordinated global launch. PTC said pricing discussions in Japan are expected to conclude in Q1 2026, with commercial launch occurring shortly thereafter.
PTC Therapeutics (NASDAQ: PTCT) announced inducement equity grants approved Dec. 16, 2025 for five new employees under Nasdaq Listing Rule 5635(c)(4).
The company granted non-statutory stock options to purchase an aggregate of 3,825 shares and 4,385 restricted stock units (RSUs). Options have an exercise price of $75.42 (closing price on Dec. 16, 2025), a 10-year term, and vest over four years (25% after one year, then 6.25% quarterly). RSUs vest over four years with 25% vesting on each annual anniversary, subject to continued service.
PTC Therapeutics (NASDAQ: PTCT) announced that CEO Matthew B. Klein, M.D., will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 12, 2026 at 9:00 a.m. PST / 12:00 p.m. EST.
The presentation will be webcast live on the company’s Events and Presentations investor page at https://ir.ptcbio.com/events-presentations and will be archived for 30 days following the presentation.
PTC Therapeutics (NASDAQ: PTCT) announced inducement equity grants approved Nov. 24, 2025, to 15 new employees under Nasdaq Listing Rule 5635(c)(4).
The company approved non-statutory stock options to purchase an aggregate of 2,000 shares and 8,200 restricted stock units (RSUs). Options have an exercise price of $79.83 (closing price on Nov. 24, 2025), a 10-year term, and vest over four years: 25% at first anniversary then 6.25% each subsequent three months. RSUs vest over four years with 25% on each annual anniversary. Grants were approved by the Compensation Committee and issued as inducements material to each hire's acceptance of employment.
Gildan (NYSE: GIL) and several companies will move between S&P indices effective late November–early December 2025. Sandisk (SNDK) is being added to the S&P 500 on November 28, 2025, with PTC Therapeutics (PTCT) joining the S&P SmallCap 600 the same day. Upwork (UPWK) and First Interstate BancSystem (FIBK) also join the SmallCap 600 on November 28 and December 2, 2025, respectively; Interpublic, Premier and Hanesbrands will be removed.