PTC Therapeutics, Inc.
ptcbio.comWhat Do They Do?
Develops and sells medicines for rare disorders, including treatments for neurological and metabolic diseases, for children and adults.
10-K for FY2025, filed Feb 19, 2026
How Do They Make Money?
Collaboration and license arrangements are the largest revenue stream: partners pay upfront fees, milestones and research funding, and PTC can earn royalties on partner product sales. PTC also sells medicines to distributors, hospitals and pharmacies; customers are invoiced after delivery and generally pay within 30 to 90 days.
10-K for FY2025, filed Feb 19, 2026The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license…View ↗
In addition to research and development and sales milestones, the Company is eligible to recognize royalties on worldwide annual net sales…View ↗
The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD…View ↗
$1.7B
-
10-K for FY2025, filed Feb 19, 2026
$1.7B ↗
| Segment | Share | Revenue | How money arrives |
|---|---|---|---|
|
Net product revenue
Sold outright
10-K for FY2025, filed Feb 19, 2026 |
34%
FY2022: 77%
|
Net product revenue, FY2025
$586.7M Sources
|
Sold outright |
|
Collaboration and license revenue
Licensed
10-K for FY2025, filed Feb 19, 2026 |
58% |
Collaboration and license revenue, FY2025
$998.4M Sources
|
Licensed |
|
Royalty revenue
Licensed
10-K for FY2025, filed Feb 19, 2026 |
14%
FY2022: 16%
|
Royalty revenue, FY2025
$244.2M Sources
|
Licensed |
|
Manufacturing revenue
|
0.0%
FY2022: 0.0%
|
Manufacturing revenue, FY2025
$0 Sources
|
Who Are Their Customers?
Medicine buyers include distributors, hospitals, specialty pharmacies and retail pharmacies, which order products; collaboration partners such as Roche license programs and fund research and development.
10-K for FY2025, filed Feb 19, 2026The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD…View ↗
The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license…View ↗
In November 2011, the Company and the SMA Foundation entered into a licensing and collaboration agreement with Roche. Under the terms of…View ↗
Do Revenues Turn Into Profits?
Yes. It kept 39 cents of every revenue dollar as net income in FY2025 and turned 41 cents into free cash flow.
10-K for FY2025, filed Feb 19, 2026
$866.9MView ↗
10-K for FY2025, filed Feb 19, 2026
$682.6MView ↗
10-K for FY2025, filed Feb 19, 2026
$702.3MView ↗
How Are They Changing?
10-Q for Q2 FY2026, filed Jul 30, 2026
Open tableView ↗
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Aug 28, 2026
Agreed to acquire Sangamo's ST-920 Fabry disease assets
The deal includes an upfront payment and contingent approval milestones and remains subject to bankruptcy court approval and other conditions.
Sources
8-K, filed Aug 28, 2026
On August 25, 2026, PTC Therapeutics, Inc. (the “Company”) entered into an asset purchase agreement (the “Asset Purchase Agreement”) with…View ↗
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Jul 30, 2026
Raised 2026 revenue and product guidance
Total revenue guidance rose to $1.18 to $1.28 billion and product guidance to $850 to $950 million.
Sources
8-K, filed Jul 30, 2026
– Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950…View ↗
Growing. Revenue grew 3.2x in 4 years, from $538.6M in FY2021 to $1.7B in FY2025, about 34% a year.
Sephience commercialization
Sepiapterin was described as a clinical-stage PKU program; it is now marketed as Sephience, an approved medicine for PKU.
10-K for FY2022, filed Feb 21, 2023 10-K for FY2025, filed Feb 19, 2026Metabolic Platform – The most advanced molecule in our metabolic platform is sepiapterin. We initiated a registration-directed Phase 3…View ↗
In June 2025, Sephience was granted marketing authorization by the EC for the treatment of children and adults living with PKU within the…View ↗
Votoplam partner role
Novartis has taken over further development of the Huntington's disease program, while PTC continues to collaborate with Novartis.
10-K for FY2022, filed Feb 21, 2023 10-K for FY2025, filed Feb 19, 2026Splicing Platform – In addition to our SMA program, our splicing platform also includes PTC518, which is being developed for the treatment…View ↗
Splicing Platform – In addition to our SMA program, our splicing platform also includes votoplam, which is being developed for the…View ↗
Stated disease focus
The older report listed oncology alongside neurology and metabolism among its rare-disease areas; the latest overview names neurology and metabolism.
10-K for FY2022, filed Feb 21, 2023 10-K for FY2025, filed Feb 19, 2026We have a portfolio pipeline that includes several commercial products and product candidates in various stages of development, including…View ↗
We have a diversified therapeutic portfolio that includes several commercial products and product candidates in various stages of…View ↗
Who Do They Compete With?
Competes with Otsuka Pharmaceutical, SOM Biotech, Maze Therapeutics, Agios Pharmaceuticals, Santhera Pharmaceuticals, Sarepta Therapeutics, NS Pharma, Dyne Therapeutics, Wave Life Sciences, Daiichi Sankyo, Nippon Shinyaku, Solid Biosciences, REGENXBIO, Ionis, Cheisi Farmaceutica, Alnylam Pharmaceuticals, Pfizer, Scholar Rock, Biohaven, and Roche Pharmaceuticals
10-K for FY2025, filed Feb 19, 2026Sephience for PKU. Sephience faces competition from Kuvan (sapropterin dihydrochloride), including generic versions, and Palynziq…View ↗
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