PTC THERAPEUTICS, INC.

Overview

PTC Therapeutics, Inc. is a global biopharmaceutical company that discovers, develops and commercializes medicines for children and adults with rare disorders, with a focus that includes rare neurological and metabolic diseases. Its stated mission is to provide access to best-in-class treatments for patients with unmet medical needs.[1]

The company’s portfolio has included treatments for conditions such as phenylketonuria, Duchenne muscular dystrophy and AADC deficiency, including Sephience, Emflaza and Upstaza/Kebilidi.[2][3] PTC reports an international presence spanning more than 50 geographies.[4] It reported FY2025 revenue of $1.731 billion and had 991 employees at December 31, 2025.[5][6] The company dates its inception to 1998.[7] [1]+6

Products & services

PTC Therapeutics’ commercial products treat rare genetic disorders. Sephience (sepiapterin) is used for phenylketonuria (PKU); it acts as a precursor to tetrahydrobiopterin (BH4), helping activate phenylalanine hydroxylase and increase phenylalanine breakdown [8][9]. Emflaza (deflazacort) is a corticosteroid approved in the United States for Duchenne muscular dystrophy (DMD) in patients aged two years and older [10]. Translarna (ataluren) is intended for DMD caused by a nonsense mutation, with the aim of enabling protein production despite the mutation [10][11].

Upstaza (eladocagene exuparvovec), marketed as Kebilidi in the United States, is a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, a central nervous system disorder associated with mutations in the DDC gene [12][13]. The company’s Q2 2026 filing separately reports Sephience, Translarna, Emflaza, Upstaza/Kebilidi, and other products as net product revenue. For the quarter, the reported product-revenue mix was Sephience $151.310 million (63.36%), Translarna $42.217 million (17.68%), Emflaza $24.634 million (10.31%), Upstaza/Kebilidi $11.163 million (4.67%), and all other products $9.495 million (3.98%), out of $238.819 million total net product revenue; percentages are calculated from reported figures [14]. [8]+11

Geographic presence

PTC Therapeutics’ principal facility is in Warren, New Jersey, with additional laboratory and office space in Bridgewater, New Jersey. The company also lists offices in Dublin, Ireland, and São Paulo, Brazil; it added leased office and laboratory space in Mountain View, California, beginning in 2025. It maintains office space in various countries for international employees, primarily through workspace providers.[15][16] Its principal executive office is in Warren, New Jersey.[17][18]

The company sells products in the United States and internationally, but its filings generally report geographic sales as U.S. and international rather than by individual country. Named markets include the EEA and UK for Upstaza and the U.S. for Kebilidi; PTC also holds commercialization rights for Tegsedi and Waylivra in Latin America and the Caribbean and reports sales of both in Brazil.[19]+3

In FY2025, net product revenue was $252.2 million in the U.S. and $334.5 million internationally—approximately 43% and 57%, respectively.[23] For the six months ended June 30, 2026, net product revenue was $288.7 million in the U.S. and $175.7 million internationally, approximately 62% and 38%.[24] On a broader total-revenue basis, FY2025 revenue was $1,494.8 million in the U.S. and $235.9 million outside the U.S.; these figures are distinct from net product revenue.[25] [15]+15

Business segments

PTC Therapeutics reports one operating and reportable segment, “life science,” encompassing the discovery, development and commercialization of medicines for rare disorders. Its therapeutic areas and product categories are not separately reported as formal business segments.[26][27]

For the six months ended June 30, 2026, net product revenue was $464.4 million. Sephience, for phenylketonuria (PKU), contributed $275.9 million (59.4% of product revenue); Translarna, for nonsense-mutation Duchenne muscular dystrophy (DMD), $101.2 million (21.8%); Emflaza, for DMD, $46.1 million (9.9%); Upstaza/Kebilidi, for aromatic L-amino acid decarboxylase (AADC) deficiency, $20.8 million (4.5%); and other products, $20.5 million (4.4%). Percentages are calculated as shares of net product revenue.[24]+3

For full-year 2025, net product revenue was $586.7 million, about 33.9% of consolidated revenue of $1,730.7 million. By product, Translarna contributed $235.3 million (13.6% of consolidated revenue), Emflaza $146.4 million (8.5%), Sephience $111.2 million (6.4%), Upstaza/Kebilidi $56.6 million (3.3%), and other products $37.3 million (2.2%). Collaboration and license revenue was $998.4 million (57.7% of consolidated revenue), while royalty revenue was $244.2 million (14.1%). Product-category percentages are calculated using consolidated revenue as the denominator.[23][31] [26]+22

Company history

PTC Therapeutics was founded in 1998 by Stuart W. Peltz, Ph.D., and completed its initial public offering in June 2013, listing on Nasdaq as PTCT [32][33]. In 2014, the European Commission granted conditional marketing authorization to Translarna for nonsense-mutation Duchenne muscular dystrophy. In 2017, the FDA approved Emflaza for Duchenne muscular dystrophy; PTC acquired its rights from Marathon Pharmaceuticals and launched it in the United States [34]+3.

PTC expanded into gene therapy by completing its acquisition of Agilis Biotherapeutics in 2018, adding a program for AADC deficiency [38]. It added further development programs through acquisitions of BioElectron assets in 2019 and Censa Pharmaceuticals in 2020 [39][40]. In 2022, Upstaza, a gene therapy for AADC deficiency, received European Commission authorization [30]. Founder Peltz stepped down as CEO in March 2023, and Matthew B. Klein succeeded him [32]. In 2025, the FDA approved Sephience for phenylketonuria, while the European Commission decided not to renew Translarna’s conditional marketing authorization [41][2]. [36]+11

Sources

• PTC THERAPEUTICS, INC. — Full financial report (2025 Y)
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