WARREN, N.J. , Aug. 21, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its executives will speak at the following conferences: Cantor Global Healthcare Conference 2025 Wednesday, Sept. 3 at 8:35 a.m. ET Wells Fargo 2025 Healthcare Conference Thursday, Sept. 4 at 11:00 a.m. ET Morgan Stanley 23 rd Annual Global Healthcare Conference Monday, Sept. 8 at 9:15 a.m. ET The presentations will be... Read More

WARREN, N.J. , Aug. 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc . (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia. "We are of course disappointed by the FDA's decision to not approve vatiquinone," said Matthew B. Klein... Read More

European and FDA approval of Sephience™ (sepiapterin) with broad labeling for PKU Global launch underway in Europe and U.S. Total Q2 Revenue of $179M WARREN, N.J. , Aug. 7, 2025 /PRNewswire/ -- PTC Therapeutics, Inc ., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30 , 2025. "We had another strong quarter highlighted by the first approvals of Sephience for the... Read More

Broad labeling inclusive of all disease subtypes for individuals 1 month of age and older PTC will host a conference call on Monday, July 28 th at 5:00 pm ET WARREN, N.J. , July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The... Read More

WARREN, N.J. , July 21, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the company will host a webcast conference call to report its second quarter 2025 financial results and provide an update on the company's business and outlook on Thursday, August 7 , at 4:30 p.m. ET . To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays,... Read More

Broad label inclusive of all ages and disease severities Launch to be initiated in Germany WARREN, N.J. , June 23, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Sephience™ (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and... Read More

WARREN, N.J. , June 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc . (NASDAQ: PTCT) today announced that on June 16, 2025 , the company approved non-statutory stock options to purchase an aggregate of 12,725 shares of its common stock and 27,790 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 34 new employees. The awards were made pursuant to the Nasdaq... Read More

Strong revenue performance of $190 million Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025 , NDA review remains on track for July 29, 2025 PDUFA date Global Sephience launch activities progressing well Strong cash position of over $2.0 billion as of March 31, 2025 WARREN, N.J. , May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. , (NASDAQ: PTCT) today announced a corporate update and financial... Read More

Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 Signals of dose-dependent clinical benefit relative to matched natural history cohort as well as dose-dependent lowering of NfL in Stage 2 patients at Month 24 Continued favorable safety and tolerability profile with no treatment-related NfL spikes PTC... Read More

WARREN, N.J. , May 1, 2025 /PRNewswire/ -- PTC Therapeutics, Inc . (NASDAQ: PTCT) announced today that on April 25, 2025 , the company approved non-statutory stock options to purchase an aggregate of 7,000 shares of its common stock and 10,780 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 12 new employees. The awards were made pursuant to the Nasdaq... Read More

Label includes full spectrum of PKU patients European launch preparations underway WARREN, N.J. , April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults... Read More

WARREN, N.J. , April 24, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its executives will speak at the following conferences: Bank of America Securities 2025 Health Care Conference Tuesday, May 13 at 10:00 a.m. PT / 1:00 p.m. ET RBC Capital Markets Global Healthcare Conference 2025 Tuesday, May 20 at 2:05 p.m. ET William Blair 45 th Annual Growth Stock Conference 2025 Tuesday, June 3 at... Read More

WARREN, N.J. , April 17, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the company will host a webcast conference call to report its first quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, May 6 , at 4:30 p.m. ET . To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we... Read More

WARREN, N.J. , March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on March 24, 2025 , the company approved non-statutory stock options to purchase an aggregate of 12,000 shares of its common stock and 28,220 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 35 new employees. The awards were made pursuant to the Nasdaq... Read More

WARREN, N.J. , March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the... Read More

Results show 97% of phenylalanine (Phe) tolerance study participants were able to increase their dietary Phe intake with a mean increase of 126% Genetic variant analysis of Phase 3 APHENITY trial demonstrates meaningful effect in classical subjects with non-BH4 responsive genotypes WARREN, N.J. , March 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3... Read More

Full year 2024 revenue of $807 million , exceeding guidance All 2024 clinical and regulatory milestones were achieved on schedule, including four NDA submissions, all of which were accepted for filing License and collaboration agreement with Novartis for PTC518 Huntington's disease program closed in January 2025 Cash of over $2.0 billion as of January 2025 WARREN, N.J. , Feb. 27, 2025 /PRNewswire/ -- PTC Therapeutics, Inc .,... Read More

WARREN, N.J. , Feb. 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its executives will participate in fireside chats at the following investor conferences: TD Cowen 45 th Annual Health Care 2025 Conference Tuesday, March 4 at 11:10 a.m. ET Barclays 27 th Annual Global Healthcare Conference Tuesday, March 11 at 12:30 p.m. ET The presentations will be webcast live on the Events and... Read More

PDUFA target action date of Aug. 19, 2025 WARREN, N.J. , Feb. 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia (FA). The application has been granted Priority Review and assigned a Prescription Drug User... Read More

WARREN, N.J. , Feb. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its fourth quarter and full year 2024 financial results and provide an update on the company's business and outlook on Thursday, Feb. 27 , at 4:30 p.m. EST . To access the call by phone, please click here to register and you will be provided with dial-in details. To... Read More

WARREN, N.J. , Jan. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Jan. 3, 2025 , the company approved 1,200 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to two new employees. The awards were made pursuant to the Nasdaq inducement grant exception as a component of the new hires' employment compensation. The... Read More

Unaudited 2024 total revenue of approximately $814 million , exceeding guidance Four approval applications submitted to FDA in 2024 Global launch preparations underway for sepiapterin for PKU, a $1 billion market opportunity; CHMP opinion expected Q2 2025, U.S. approval decision in July 2025 License and collaboration agreement with Novartis for PTC518 program closed PIVOT-HD data readout for PTC518 expected Q2 2025 WARREN,... Read More

WARREN, N.J. , Dec. 23, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that CEO Matthew B. Klein , M.D., will present at the 43 rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 , at 11:15 a.m. PST / 2:15 p.m. EST . The presentation will be webcast live on the Events and Presentations page under the Investors section of PTC Therapeutics' website at... Read More

If approved, vatiquinone would be the first and only authorized therapy for children with FA PTC's fourth approval application submitted to FDA in 2024 WARREN, N.J. , Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration... Read More

WARREN, N.J. , Dec. 17, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Dec. 13, 2024 , the company approved non-statutory stock options to purchase an aggregate of 1,725 shares of its common stock and 5,060 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to eight new employees. The awards were made pursuant to the Nasdaq... Read More

PTC to receive $1.0B in cash at closing PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones PTC to share profits in the U.S. and tiered double-digit royalties on ex-U.S. net sales Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of ongoing PIVOT-HD study PTC will host a conference call on Dec. 2, 2024 ,... Read More

WARREN, N.J. , Nov. 27, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. PTC was granted the PRV on November 13, 2024 along with the U.S. Food and Drug Administration (FDA) approval of KEBILIDI™ (eladocagene exuparvovec-tneq) for the treatment... Read More

Study failed to meet primary and secondary efficacy endpoints WARREN, N.J. , Nov. 26, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the global Phase 2 placebo-controlled CardinALS study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis. While there was modest numerical benefit recorded on the primary endpoint and correlation of... Read More

WARREN, N.J. , Nov. 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Nov. 20, 2024 , the company approved non-statutory stock options to purchase an aggregate of 18,815 shares of its common stock and 17,995 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to ten new employees. The awards were made pursuant to the Nasdaq... Read More

First-ever FDA approval for gene therapy directly administered to the brain Priority review voucher granted Broad label including children and adults PTC pioneers new approach for CNS drug delivery WARREN, N.J. , Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the... Read More

Strong revenue performance, supporting increase in full-year revenue guidance to $750 -800 million Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone WARREN, N.J. , Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc ., (NASDAQ: PTCT) today announced a corporate update and... Read More

WARREN, N.J. , Nov. 4, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: UBS Global Healthcare Conference Wednesday, Nov. 13 at 2:45 p.m. PST / 5:45 p.m. EST Jefferies London Healthcare Conference Tuesday, Nov. 19 at 11:30 a.m. GMT / 6:30 a.m. EST Citi's 2024 Global Healthcare Conference Tuesday, Dec. 3 at 2:30 p.m. EST... Read More

WARREN, N.J. , Oct. 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc . (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). "The NDA acceptance for review is a significant milestone that brings us one step closer to providing this... Read More

WARREN, N.J. , Oct. 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Oct. 23, 2024 , the company approved non-statutory stock options to purchase an aggregate of 13,690 shares of its common stock and 20,250 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to nineteen new employees. The awards were made pursuant to the... Read More

WARREN, N.J. , Oct. 24, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its third quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, Nov. 7 , at 4:30 p.m. EST . To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays,... Read More

Opinion to be reviewed by European Commission WARREN, N.J. , Oct. 18, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has maintained its negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne Muscular Dystrophy... Read More

WARREN, N.J. , Oct. 14, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has set a target regulatory action date of July 29 , 2025, for the review of the New Drug Application (NDA) for sepiapterin, consistent with a standard review timeline. The NDA was submitted with an indication for the treatment of pediatric and adult patients with phenylketonuria (PKU), including all age groups and... Read More

Statistically significant results on primary endpoint of long-term study analyses Following FDA pre-NDA feedback, submission on track for December 2024 WARREN, N.J. , Oct. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) provided today several positive updates on the vatiquinone Friedreich ataxia (FA) program. The pre-specified endpoint for two different FA long-term extension studies was met, with highly... Read More

WARREN, N.J. , Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter. "The FDA filing acceptance for sepiapterin is a critical... Read More

WARREN, N.J. , Sept. 26, 2024 /PRNewswire/ -- PTC Therapeutics (NASDAQ: PTCT) announced today that the FDA has granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. Fast Track designation is awarded to promising therapies for diseases of high unmet need. "The granting of Fast Track designation to the PTC518 program further supports the potential of PTC518 to provide a disease... Read More

WARREN, N.J. , Sept. 12, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Sept. 8, 2024 , the company approved non-statutory stock options to purchase an aggregate of 16,180 shares of its common stock and 23,725 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to nine new employees. The awards were made pursuant to the Nasdaq... Read More

WARREN, N.J. , Aug. 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: Morgan Stanley 22 nd Annual Global Healthcare Conference Thursday, Sept. 5 at 9:15 am ET 2024 Wells Fargo Healthcare Conference Friday, Sept. 6 at 12:00 pm ET Cantor 2024 Global Healthcare Conference Tuesday, Sept. 17 at 2:30 pm ET The presentations... Read More

Continued strong revenue performance NDAs for sepiapterin and Translarna™ submitted to FDA Positive interim data readout from PTC518 PIVOT-HD study On track to achieve remaining 2024 clinical and regulatory milestones WARREN, N.J. , Aug. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. , (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ending June 30 , 2024. "I am proud of our... Read More

WARREN, N.J. , July 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin NDA to the U.S. FDA. The NDA submission is for the treatment of pediatric and adult patients with phenylketonuria (PKU), including the full spectrum of ages and disease subtypes. "This NDA submission is an important step in bringing this therapy to children and adults living with PKU in the... Read More

WARREN, N.J. , July 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its second quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, Aug. 8 , at 4:30 p.m. EDT . To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays,... Read More

PTC to submit a request for re-examination WARREN, N.J. , June 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the CHMP has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nmDMD. This opinion follows the return of the previously issued negative opinion by the European Commission (EC) for re-review. PTC plans... Read More

WARREN, N.J. , June 21, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on June 17, 2024 , the company approved non-statutory stock options to purchase an aggregate of 1,380 shares of its common stock and 6,260 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to nine new employees. The awards were made pursuant to the Nasdaq... Read More

FDA lifts PTC518 partial clinical hold based on PIVOT-HD data Conference call and webcast to be held June 20 th at 8:00 am EDT WARREN, N.J. , June 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. At Month 12, PTC518 treatment resulted in dose-dependent lowering of mutant huntingtin (mHTT) protein in the... Read More

WARREN, N.J. , May 29, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conference: William Blair's 44th Annual Growth Stock Conference Tuesday, June 4 , at 9:00 am ET / 8:00 am CT The presentation will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website at... Read More

Review of European marketing application for PKU now initiated WARREN, N.J. , May 28, 2024 /PRNewswire/ -- 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... Read More

European Commission decides against adoption of negative opinion Translarna authorization remains active in Europe 2024 revenue guidance paused WARREN, N.J. , May 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna™... Read More

PDUFA target action date of November 13, 2024 WARREN, N.J. , May 14, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency. The application has been granted Priority Review with a target regulatory action date of November 13, 2024 . "We... Read More

WARREN, N.J. , May 2, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: RBC Capital Markets 2024 Global Healthcare Conference Tuesday, May 14 , at 11:00 am EDT Bank of America Securities 2024 Health Care Conference Thursday, May 16 , at 11:40 am EDT / 08:40 am PDT The presentations will be webcast live on the Events and... Read More

Strong revenue performance across product portfolio Submitted MAA for sepiapterin and BLA for Upstaza On target to achieve remaining 2024 clinical and regulatory milestones, including global submissions for sepiapterin WARREN, N.J. , April 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. , (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31 , 2024. "We are off to a... Read More

WARREN, N.J. , April 23, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on April 19, 2024 , the company approved non-statutory stock options to purchase an aggregate of 8,090 shares of its common stock and 8,290 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to six new employees. The awards were made pursuant to the Nasdaq... Read More

SOUTH PLAINFIELD, N.J. , April 11, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its first quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, April 25 , at 4:30 p.m. ET . To access the call by phone, please click here to register and you will be provided with dial-in details. To... Read More

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... Read More

BLA submitted to FDA for Upstaza™ for the treatment of AADC deficiency NDA to be submitted mid-year for Translarna™ based on FDA feedback SOUTH PLAINFIELD, N.J. , March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. In... Read More

34% year-over-year growth in 2023 total revenue Global filings of sepiapterin remain on track with first submission of the EU MAA expected in March Potential NDA submission for vatiquinone for Friedreich ataxia expected in late 2024 SOUTH PLAINFIELD, N.J. , Feb. 29, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. , (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending... Read More

SOUTH PLAINFIELD, N.J. , Feb. 27, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: TD Cowen 44th Annual Health Care Conference Monday, March 4 , at 1:30 p.m. EST Leerink Partners Global Biopharma Conference 2024 Monday, March 11 , at 2:40 p.m. EDT Barclays 26th Annual Healthcare Conference Tuesday, March 12 , at 4:05 p.m.... Read More

SOUTH PLAINFIELD, N.J. , Feb. 22, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Feb. 15, 2024 , the company approved non-statutory stock options to purchase an aggregate of 1,840 shares of its common stock and 4,275 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to six new employees. The awards were made pursuant to the... Read More

SOUTH PLAINFIELD, N.J. , Feb. 15, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its fourth quarter and full year 2023 financial results and provide an update on the company's business and outlook on Thursday, Feb. 29 , at 4:30 p.m. EST . To access the call by phone, please click here to register and you will be provided with dial-in... Read More

SOUTH PLAINFIELD, N.J. , Jan. 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™ (ataluren). "We are disappointed that the CHMP has maintained its negative opinion on the... Read More

Unaudited 2023 total revenue of $946 million, representing 35% year-over-year growth Regulatory filings in the EU and US for sepiapterin in PKU, a potential $1 billion global commercial opportunity, remain on track for 2024 Multiple study readouts planned for 2024, including 12-month interim data from the PIVOT-HD study of PTC518 in HD patients SOUTH PLAINFIELD, N.J. , Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc.... Read More

SOUTH PLAINFIELD, N.J. , Jan. 3, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the 42 nd Annual J.P. Morgan Healthcare Conference on Monday, Jan. 8 , at 7:30 a.m. PST / 10:30 a.m. EST . The presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website at... Read More

SOUTH PLAINFIELD, N.J. , Dec. 14, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Dec. 10, 2023 , the company approved non-statutory stock options to purchase an aggregate of 3,575 shares of its common stock and 3,035 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to two new employees. The awards were made pursuant to the... Read More

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... Read More

SOUTH PLAINFIELD, N.J. , Nov. 3, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Nov. 1, 2023 , the company approved non-statutory stock options to purchase an aggregate of 14,005 shares of its common stock and 19,125 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 21 new employees. The awards were made pursuant to the... Read More

Third quarter 2023 total revenues of $197 million and remain on target for 2023 revenue guidance PTC strengthened its financial position following recent restructuring initiatives and Royalty Pharma transaction PTC provides regulatory updates for pipeline programs SOUTH PLAINFIELD, N.J. , Oct. 26, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. , (NASDAQ: PTCT) today announced a corporate update and financial results for the... Read More

PTC to receive $1B in upfront cash for approximately 67% of outstanding royalties, with option for additional $500M Financing proceeds will be used to retire Blackstone debt obligations and fund planned operations SOUTH PLAINFIELD, N.J. , Oct. 19, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced an agreement with Royalty Pharma plc. to monetize up to $1.5 billion of the Evrysdi royalty stream. Under... Read More

Translarna CHMP opinion re-examination request to be submitted SOUTH PLAINFIELD, N.J. , Sept. 28, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today further strategic prioritization and associated workforce reduction. The portfolio prioritization continues the process initiated in May 2023 as the company continues to focus its resources on its differentiated, high potential R&D programs and on support... Read More

Conference call to be held at 8:45 am EDT SOUTH PLAINFIELD, N.J. , Sept. 15, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna™ (ataluren) for the treatment of... Read More

BOSTON, Sept. 12, 2023 (GLOBE NEWSWIRE) -- Sensorium Therapeutics (Sensorium), a biotechnology company translating insights from nature and human experience to deliver transformational therapeutics, today announced the appointment of veteran life sciences executive David Southwell to its Board of Directors. “We are excited to welcome David to our Board,” said Dick Simon, Chief Executive Officer of Sensorium. “He is a... Read More

2023 recipients include patient advocacy organizations in Australia and Brazil SOUTH PLAINFIELD, N.J. , Sept. 7, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the two recipients of its 2023 STRIVE Awards for Duchenne Muscular Dystrophy. Through the STRIVE program, the company awards grants to non-profit patient advocacy organizations serving the Duchenne community. This year's grants will fund... Read More

SOUTH PLAINFIELD, N.J. , Aug. 30, 2023 /PRNewswire/ -- PTC Therapeutics (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: Citi 2023 18 th Annual BioPharma Conference Wednesday, Sept. 6 , at 9:40 a.m. EDT Morgan Stanley 21 st Annual Global Healthcare Conference 2023 Monday, Sept. 11 , at 1:35 p.m. EDT 2023 Cantor Global Healthcare Conference Tuesday, Sept. 26 , at... Read More

Results from the APHENITY trial in PKU and long-term extension study including Phe tolerance to be highlighted SOUTH PLAINFIELD, N.J. , Aug. 28, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that results from the APHENITY clinical trial and long-term extension study will be presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 which is being held from... Read More

Second-quarter 2023 total revenue of $214 million , representing 29% year-over-year growth Positive APHENITY and PIVOT-HD data readouts in the second quarter Numerous regulatory milestones planned for the second half of 2023 SOUTH PLAINFIELD, N.J. , Aug. 3, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ending June 30 , 2023. "I am... Read More

Following ratification by the European Commission, Evrysdi will be available to treat all spinal muscular atrophy patients, including babies from birth SOUTH PLAINFIELD, N.J. , July 21, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi ® (risdiplam) marketing authorization to... Read More

SOUTH PLAINFIELD, N.J. , July 20, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its second quarter 2023 financial results and provide an update on the company's business and outlook on Thursday, Aug. 3 , at 4:30 p.m. ET . To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid... Read More

Dr. Jacobson recognized for his pioneering work in nonsense-mediated mRNA decay SOUTH PLAINFIELD, N.J. , July 19, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Allan Jacobson , Ph.D., co-founder of PTC and member of the Board of Directors, was presented the 2023 Gruber Genetics Prize for his work in identifying and describing the mechanism of nonsense-mediated mRNA decay today in a ceremony... Read More

SOUTH PLAINFIELD, N.J. , July 18, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on July 13, 2023 , the company approved non-statutory stock options to purchase an aggregate of 66,380 shares of its common stock and 30,685 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 11 new employees. The awards were made pursuant to the... Read More

SOUTH PLAINFIELD, N.J. , July 17, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the appointment of Pierre Gravier to the role of Chief Financial Officer (CFO). Mr. Gravier brings more than 17 years of experience as an investment banker, venture capitalist and scientist to PTC. Most recently, Pierre was a managing director in the healthcare group of Perella Weinberg Partners, a leading independent... Read More

SOUTH PLAINFIELD, N.J. , July 11, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), announced today that the company will provide a virtual presentation on PKU including discussion of the current therapeutic and commercial landscape with an expert key opinion leader. In addition, the company will discuss the sepiapterin clinical program followed by a Q&A session. The event will occur on Wednesday, July 19 , from 12... Read More

SOUTH PLAINFIELD, N.J. , June 29, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint of reduction in observable motor seizures. The study showed evidence of treatment effect in reducing seizure frequency in the overall study population and in the largest... Read More

Dose-dependent lowering of blood Huntingtin (HTT) protein levels at 12 weeks Favorable tolerability profile with no treatment-related serious adverse events or NfL spikes Conference call and webcast to be held June 21 st at 8:00 am EDT SOUTH PLAINFIELD, N.J. , June 21, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared interim data from the 12-week portion of the PIVOT-HD Phase 2 study of PTC518 in... Read More

SOUTH PLAINFIELD, N.J. , June 15, 2023 /PRNewswire/ -- PTC Therapeutics, Inc . (NASDAQ: PTCT) today announced that on June 12, 2023 , the company approved non-statutory stock options to purchase an aggregate of 11,260 shares of its common stock and 17,660 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 23 new employees. The awards were made pursuant to... Read More

SOUTH PLAINFIELD, N.J. , June 2, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: Jefferies Healthcare Conference Wednesday, June 7 at 12:30 p.m. EDT Goldman Sachs 44 th Annual Global Healthcare Conference Wednesday, June 14 at 1:40 p.m. EDT / 10:40 a.m. PDT The presentations will be webcast live on the Events and... Read More

Conference call and webcast to be held at 5:00 pm EDT SOUTH PLAINFIELD, N.J. , May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia. The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population. However, vatiquinone treatment did... Read More

Preclinical and early research gene therapy programs discontinued Expected reductions of approximately 15% in residual 2023 OPEX SOUTH PLAINFIELD, N.J. , May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the discontinuation of preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization. PTC will continue its development and global commercialization... Read More

Highly statistically significant and clinically meaningful results 63% mean blood Phe reduction in primary analysis population (p<0.0001) Conference call and webcast to be held at 8:00 AM EDT SOUTH PLAINFIELD, N.J. , May 17, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the primary endpoint was achieved in the APHENITY, Phase 3 registration-directed clinical trial of sepiapterin in adult and... Read More

SOUTH PLAINFIELD, N.J. , April 28, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on April 24, 2023 , the company approved non-statutory stock options to purchase an aggregate of 10,770 shares of its common stock and 18,110 restricted stock units (RSUs), each representing the right to receive one share of its common stock upon vesting, to 29 new employees. The awards were made pursuant to the... Read More

First-quarter 2023 total revenue of $220 million, representing 48% year-over-year growth Readouts from four clinical studies expected in second quarter of 2023, three of which are registration-directed SOUTH PLAINFIELD, N.J. , April 27, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. ( NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31 , 2023. "We are pleased to report... Read More

SOUTH PLAINFIELD, N.J. , April 13, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its first quarter 2023 financial results and provide an update on the company's business and outlook on Thursday, April 27 , at 4:30 p.m. ET . To access the call by phone, please click here to register and you will be provided with dial-in details. To... Read More

SOUTH PLAINFIELD, N.J. , March 28, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on March 22, 2023 , the company approved non-statutory stock options to purchase an aggregate of 58,465 shares of its common stock and 29,710 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 58 new employees. The awards were made pursuant to... Read More

Chief Operating Officer Matthew Klein , M.D., M.S., F.A.C.S. to become Chief Executive Officer and join the Board of Directors Conference call to be held today, Fri., March 24 th at 8:30 am ET SOUTH PLAINFIELD, N.J. , March 24, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT ). Stuart Peltz , Ph.D., founding Chief... Read More

2022 total revenue of $699 million , representing 30% year-over-year growth, or $740 million and 37% growth at CER* Guidance for full-year 2023 total revenue of $940 million to $1 billion reaffirmed Four important study readouts expected in first half of 2023, three of them registration-directed SOUTH PLAINFIELD, N.J. , Feb. 21, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and... Read More

SOUTH PLAINFIELD, N.J. , Feb. 21, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: Raymond James 44 th Annual Institutional Investor Conference Monday, March 6 , at 8:05 a.m. EST Cowen 43 rd Annual Health Care Conference Tuesday, March 7 , at 12:50 p.m. EST Barclays Global Healthcare Conference Tuesday, March 14 , at 8:00... Read More

SOUTH PLAINFIELD, N.J. , Feb. 7, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its fourth quarter and full year 2022 financial results and provide an update on the company's business and outlook on Tuesday, Feb. 21 , at 4:30 p.m. ET . To access the call by phone, please click here to register and you will be provided with dial-in... Read More

This award recognizes PTC's 25-year legacy in discovering and delivering novel therapies that address the underlying cause of rare diseases SOUTH PLAINFIELD, N.J. , Feb. 3, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company has been named the winner of 2023 EURORDIS Black Pearl Company Award for Innovation. The Black Pearl Awards recognize and celebrate companies who have undertaken... Read More

SOUTH PLAINFIELD, N.J. , Feb. 2, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conference: SVB Securities Global Biopharma Conference Tuesday, Feb. 14 at 9:20 a.m. EST The presentation will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website at... Read More

Only approved treatment for the underlying cause of nonsense mutation Duchenne muscular dystrophy SOUTH PLAINFIELD, N.J. , Jan. 19, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the National Institute for Health and Care Excellence (NICE) has issued a Final Evaluation Document recommending Translarna™ (ataluren) for reimbursement and use across the National Health Service (NHS) in England... Read More

2023 Program to Focus on Innovation and Transition to Adulthood Initiatives Submissions Due March 17, 2023 SOUTH PLAINFIELD, N.J. , Jan. 17, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the launch of the ninth annual STRIVE™ awards program. Through this program, PTC awards grants to patient advocacy organizations who are committed to developing initiatives that address the needs of the Duchenne... Read More

SOUTH PLAINFIELD, N.J. , Jan. 11, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on Jan. 5, 2023 , the company approved non-statutory stock options to purchase an aggregate of 39,585 shares of its common stock and 13,045 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 33 new employees. The awards were made pursuant to the... Read More

~$710 million unaudited 2022 total revenue, representing an impressive over 30% year-over-year growth $940 million - $1.0 billion 2023 total revenue guidance Results from three registration-directed clinical trials expected in 2023 One additional registration-directed clinical trial expected to initiate in 2023 SOUTH PLAINFIELD, N.J. , Jan. 9, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update... Read More

SOUTH PLAINFIELD, N.J. , Jan. 4, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the 41 st Annual J.P. Morgan Healthcare Conference on Monday, Jan. 9 , at 7:30 a.m. PST / 10:30 a.m. EST . The presentation will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website at... Read More

First global regulatory approval for treatment of this rare genetic disease SOUTH PLAINFIELD, N.J. , Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil . This is the first approval... Read More