PTC THERAPEUTICS, INC.
PTC Therapeutics, Inc. (NASDAQ: PTCT) is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare disorders. The company maintains its website at www.ptcbio.com. From the company’s description.
Margins and return on equity change in ; everything else in percent. Fewer shares and less debt are shown plainly, not as bad news — it depends what you make of buybacks. Dotted labels explain each measure. is shown separately from the annual figures.
What PTC THERAPEUTICS, INC. does
In the company’s own words
PTC Therapeutics, Inc. (NASDAQ: PTCT) is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare disorders. The company maintains its website at www.ptcbio.com.
PTC operates a diversified therapeutic portfolio focused on rare diseases in neurology and metabolism. The company's commercial products include Sephience for phenylketonuria treatment, approved in the United States, European Economic Area, Japan, and Brazil; Emflaza for Duchenne muscular dystrophy in the United States; Upstaza and Kebilidi for Aromatic L-Amino Decarboxylase deficiency in the European Economic Area and United States respectively; Tegsedi and Waylivra for rare diseases in Latin America and the Caribbean; and Evrysdi for spinal muscular atrophy, developed through collaboration with Roche and the Spinal Muscular Atrophy Foundation.
The company's development pipeline includes votoplam for Huntington's disease, being developed in collaboration with Novartis Pharmaceuticals Corporation following a Phase 2 study that met its primary endpoints of blood huntingtin protein lowering and safety. The inflammation and ferroptosis platform includes vatiquinone for Friedreich's ataxia, for which the company submitted a new drug application to the FDA in December 2024 and received a complete response letter in August 2025 requesting additional clinical data.
PTC's scientific approach leverages two primary platforms: splicing modulation and inflammation and ferroptosis targeting. The splicing platform addresses diseases amenable to regulation of pre-mRNA splicing, while the inflammation and ferroptosis platform targets oxidative stress and inflammatory pathways central to central nervous system and non-central nervous system diseases.
The company maintains a global commercial footprint with operations across multiple geographies. PTC has historically incurred significant operating losses and continues to invest substantially in research and development. The company relies on collaborations with strategic partners including Roche, Novartis, and Ionis Pharmaceuticals for development and commercialization of certain products, generating revenues through milestone payments, royalties, and profit-sharing arrangements in addition to direct product sales.
Company details
- Sector
- Healthcare
- Industry
- Pharmaceutical Preparations
- Headquarters
- WARREN, NJ
- Fiscal year end
- December
- CIK
- 1070081
- SIC
- 2834
Financial performance, year by year
Fiscal 2013–2025 · USDThe full income statement, balance sheet and cash flow for each of these years — every figure traceable to its filing — is on the Financials tab with a free account.
Who owns PTC THERAPEUTICS, INC. — and what changed
Funds as of 2026Q2 · superinvestors as of 2026-06-30
Superinvestors in PTC THERAPEUTICS, INC. · what they did last quarter
Share of each manager’s reported 13F portfolio. Change is in shares held, since the previous quarter.
Largest fund holders · PTC THERAPEUTICS, INC.’s weight in each
Fund ownership is a floor, not a census — pensions, insurers and people holding shares directly are not visible here.
PTC THERAPEUTICS, INC.’s bonds, as funds hold them
Funds report holding 2 issues of PTC THERAPEUTICS, INC.’s debt, $87.61M at market value — what funds hold, not the company’s total debt. Weighted-average coupon 1.50%, maturing 2026 to 2031.
Fund ownership counts N-PORT filers only, so it is a floor rather than a census — pensions, insurers and direct holders are not visible here, and the remainder is not “held by individuals”.
Fund ownership is a subset of institutional 13F ownership — the two describe overlapping populations and must not be added together.
Every fund and institutional holder, quarter by quarter, is on the Ownership tab with a free account.
Recent filings
All filings →Each filing opens into its own statements and disclosures here.
Put this business in context.
Compare this company with peers, find it in your funds and follow its financial history with your connected AI:
- “How do PTC THERAPEUTICS, INC. and its peers compare on margins and cash generation over ten years?”
- “Where did five years of PTC THERAPEUTICS, INC.'s earnings go — cash, debt, buybacks, reinvestment?”
- “What changed in PTC THERAPEUTICS, INC.'s last four quarterly filings that the numbers alone won't show?”