PTC Therapeutics
Develops rare-disease medicines spanning PKU, DMD, gene therapy, and RNA-splicing programs.
Something off on this page? Send us feedback.
Profile
Sells medicines for PKU, DMD, AADC deficiency, and other rare disorders across the United States and international markets, while developing RNA-splicing and inflammation programs. Revenue combines product sales with collaboration and royalty income, with regulatory approvals, reimbursement, and partners shaping the business.
Develops and commercializes medicines for rare neurological and metabolic disorders. The marketed portfolio spans Sephience for phenylketonuria, Translarna and Emflaza for Duchenne muscular dystrophy, Upstaza/Kebilidi gene therapy for AADC deficiency, and Latin American rights to Tegsedi and Waylivra. Sales reach the United States, Europe, Japan, Latin America, and other international markets.
Builds candidates through RNA-splicing and inflammation-and-ferroptosis platforms. Votoplam for Huntington’s disease is being advanced in a Novartis-funded late-stage study, supporting collaboration economics, while earlier programs target neurological, hematologic, and inflammatory diseases.
Commercial execution pairs global launches with country-level pricing and reimbursement work. Operations are managed as a single segment and depend on product authorizations, external manufacturers and distributors, and collaborators that develop or commercialize partnered assets.
Company facts
Key statistics
Year to date −8.7% · Average volume (30d) 1.1M
Analysts
Ratings
Target range
Latest analyst notes
| Date | Firm | Action | Prior rating | Rating | Prior target | Target |
|---|---|---|---|---|---|---|
| Aug 14, 2026 | Barclays | Reiterate | Overweight | $120 | $122 | |
| Aug 13, 2026 | Wells Fargo | Reiterate | Overweight | $106 | $109 | |
| Jul 31, 2026 | Morgan Stanley | Reiterate | Overweight | $94 | $104 | |
| Jul 31, 2026 | Wells Fargo | Reiterate | Overweight | $86 | $106 | |
| Jul 16, 2026 | Cantor Fitzgerald | Reiterate | Overweight | $63 | $130 |
Earnings
EPS
Revenue
History
| Report date | EPS est | EPS | Surprise | Revenue est | Revenue | Surprise | Next session |
|---|---|---|---|---|---|---|---|
| Jul 30, 2026Q2 FY26 | $0.24 | $0.92 | +289.3% | $298.6M | $360.5M | +20.7% | −9.94% |
| May 7, 2026Q1 FY26 | −$0.45 | −$0.03 | +93.3% | $217.4M | $272.6M | +25.4% | +14.27% |
| Feb 19, 2026Q4 FY25 | −$0.21 | −$1.67 | −695.2% | $279.5M | $164.7M | −41.1% | +1.09% |
| Nov 4, 2025Q3 FY25 | −$1.19 | $0.20 | +116.8% | $281.5M | $211.0M | −25.0% | +5.98% |
Financial highlights
| 12 quarters | Q1 FY25 | Q2 FY25 | Q3 FY25 | Q4 FY25 | Q1 FY26 | Q2 FY26 | |
|---|---|---|---|---|---|---|---|
| Revenue | $1.18B | $178.9M | $211.0M | $164.7M | $272.6M | $360.5M | |
| Gross profit | $1.16B | $167.5M | $95.1M | $148.4M | $244.5M | $340.6M | |
| Operating income | $970.2M | −$35.0M | $3.0M | −$81.6M | $45.9M | $149.0M | |
| Net income | $866.6M | −$64.8M | $15.9M | −$135.0M | −$2.8M | $83.5M | |
| Diluted EPS | $10.04 | −$0.83 | $0.20 | −$1.67 | −$0.03 | $0.92 | |
| Gross margin | 98.9% | 93.6% | 45.1% | 90.1% | 89.7% | 94.5% | |
| Operating margin | 82.5% | -19.5% | 1.4% | -49.6% | 16.8% | 41.3% |
Fiscal years (4-quarter sums)FY21 $538.6M · FY22 $698.8M · FY23 $937.8M · FY24 $806.8M · FY25 $1.73B
Revenue by product · Q2 FY26
Revenue by geography · Q2 FY26
Balance sheet, Jun 30, 2026Cash & short-term investments $2.23B · Total debt $696.5M · Total assets $3.25B · Shareholders' equity −$165.4M
Dividends
No dividends on record.
Ownership
A 13F quarter can total more than the shares outstanding — sub-advisers file the same block twice, and a filer's own share count can be a quarter stale. No split is drawn from it.
Shares outstanding 83.46M · Float 73.66M · 88.3% free float
Top shareholders13F · 13D/G · Form 4
| Holder | Shares | % outstanding | Form | Filed |
|---|---|---|---|---|
| BlackRock, Inc. | 13.96M | 16.73% | 13F | Aug 7, 2026 |
| RTW Investments, LP | 7.75M | 9.28% | 13F | Aug 14, 2026 |
| Jupiter Topco LLC | 7.24M | 8.67% | 13F | Aug 14, 2026 |
| Janus Henderson Group PLC | 7.19M | 8.70% | 13G | May 15, 2026 |
| Vanguard Portfolio Management LLC | 4.93M | 5.90% | 13F | Aug 13, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Aug 27, 2026 | Michael Schmertzler | director | −12,500 | $71.58 |
| Aug 26, 2026 | Michael Schmertzler | director | −12,500 | $72.94 |
| Aug 20, 2026 | Michael Schmertzler | director | −12,500 | $71.11 |
| Aug 19, 2026 | Michael Schmertzler | director | −12,500 | $73.32 |
| Aug 17, 2026 | Mark Elliott Boulding | officer: EXEC. VP AND CLO | −2,813 | $72.57 |
Peers
- BioMarin
Direct PKU rival through PALYNZIQ and KUVAN
- Sarepta Therapeutics
Core DMD genetic-medicine competitor
- Biogen
FA and SMA overlap through SKYCLARYS and SPINRAZA
CompareAfter-hours close
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| PTC Therapeutics | $70.59 | +0.73% | $5.85B | — | 58.0 | 5.8 | +101.6% |
| BioMarin | $64.67 | +0.02% | $12.50B | 165.8 | 10.4 | 3.7 | +19.9% |
| Sarepta Therapeutics | $21.05 | +0.98% | $2.20B | — | 26.3 | 1.1 | −34.3% |
| Biogen | $218.19 | −0.14% | $32.28B | 38.7 | 16.5 | 3.2 | +3.4% |
| Median | 102.2 | 16.5 | 3.2 | +3.4% |