PTC Therapeutics, Inc. is a biopharmaceutical company, which focuses on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. The company was founded by Allan Steven Jacobson and Stuart Walter Peltz on March 31, 1998 and is headquartered in Warren, NJ.
Clinical Trials
231
138 active
Approvals
3
Total approvals
Agencies
1
Regulatory bodies
Founded
1998
Active, not recruiting
134
58.0%
Approved For Marketing
1
0.4%
Completed
60
26.0%
No Longer Available
1
0.4%
Not yet recruiting
4
1.7%
Recruiting
13
5.6%
Terminated
16
6.9%
Withdrawn
2
0.9%
- PTC Therapeutics has completed its previously announced agreement with Sangamo Therapeutics to acquire ST-920, a BLA-stage, one-time administered AAV gene therapy for Fabry disease. - A rolling BLA submission to the FDA seeking accelerated approval of ST-920 is expected to be completed in the fourth quarter of 2026. - In clinical studies, isaralgagene civaparvovec enabled long-term production of the deficient alpha-galactosidase A enzyme and significantly reduced globotriaosylceramide levels with durable clinical benefit. - The FDA has granted Orphan Drug, Fast Track and RMAT designations to ST-920, which also holds Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency.
- PTC Therapeutics announced 17 abstracts and presentations on Sephience (sepiapterin) at the 2026 SSIEM Annual Symposium in Helsinki, Finland, from Aug. 25-28. - New analyses from the AMPLIPHY study show Sephience produced a 100% greater blood Phe reduction versus sapropterin in participants switching therapies. - Real-world evidence demonstrates significant diet liberalization in adolescents while maintaining target blood Phe levels, including in classical/non-BH4-responsive PKU. - Sephience treatment achieved normalization of blood Phe (<120 µmol/L) rapidly across patient subgroups, with a consistent and favorable safety profile.
- PTC Therapeutics was selected as the winning bidder to acquire ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics in a competitive bankruptcy auction. - The transaction includes $111 million upfront and up to $100 million in contingent milestone payments, with a rolling BLA submission to the FDA expected to be completed in Q4 2026. - The Phase 1/2 STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks and sustained α-Gal A activity for up to 4.5 years, with no requirement for routine immunosuppressive agents. - ST-920 holds Orphan Drug, Fast Track, and RMAT designations from the FDA, with potential commercial launch anticipated in 2027.
- Catalyst Pharmaceuticals reported positive Phase I results showing vamorolone (Agamree) delivers anti-inflammatory activity without significant immunosuppression at clinical doses. - Part A demonstrated comparable cortisol suppression between vamorolone and deflazacort, supporting switching without dosing adjustments. - Part B found clinically relevant immunosuppression only at supratherapeutic doses (40 mg/kg/day), well above approved dosing levels. - Findings support potential label expansion into additional chronic inflammatory rare diseases beyond Duchenne muscular dystrophy.
- PTC Therapeutics launched Sephience (sepiapterin) for phenylketonuria, generating $125 million in first-quarter 2026 product revenues with 36% sequential growth. - As of March 31, 2026, 1,244 patients worldwide were receiving Sephience therapy, with the U.S. adding approximately 140 new patient start forms per month. - The company completed a $500 million convertible note offering to support future business development and pipeline advancement. - Votoplam, an oral splicing therapy for Huntington's disease, reported positive 24-month phase II extension data, triggering a $50 million milestone payment from Novartis.
- The Huntington's disease treatment market across the 7MM was valued at approximately USD 320 million in 2025 and is projected to grow at a 14% CAGR from 2026 to 2036. - Skyhawk Therapeutics' SKY-0515 demonstrated a 62% dose-dependent reduction in mutant huntingtin protein at the 9 mg dose in a nine-month interim analysis, with Phase II/III FALCON-HD now enrolling globally. - UniQure's AMT-130 gene therapy met its Phase I/II primary endpoint, significantly slowing symptom progression, and the company plans to file an MAA with the UK MHRA in Q3 2026. - Current treatments including AUSTEDO, INGREZZA, and generic tetrabenazine remain focused on symptomatic relief, underscoring the unmet need for disease-modifying interventions.
- Exelixis surged 5.0% to $50.99 on Wednesday amid a broad biotechnology rally, with no company-specific catalysts driving the move. - PTC Therapeutics jumped 5.1% to $89.80 on Tuesday, riding the same sector-wide momentum alongside peers posting gains of 4.0% to 6.9%. - Coordinated buying across multiple biotech names suggests institutional investors are rotating into the sector, reflecting improved risk appetite toward growth-oriented healthcare. - Trading volumes remained moderate, with Exelixis at 1.1M shares and PTC Therapeutics at 823,046 shares, indicating measured rather than explosive positioning.
- PTC Therapeutics reported positive 24-month interim results from the PIVOT-HD extension study showing dose-dependent benefits of votoplam in slowing Huntington's disease progression in Stage 2 patients. - The 10mg dose demonstrated 52% slowing of disease progression on the cUHDRS scale compared to natural history, while the 5mg dose showed 28% slowing. - Novartis has initiated the global Phase 3 INVEST-HD study enrolling approximately 770 early-stage HD patients based on these encouraging long-term extension data. - The safety profile remained favorable at 24 months with no treatment-related increases in neurofilament light chain protein levels across both dose groups.
- PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy after FDA indicated the data unlikely meets effectiveness thresholds. - The FDA shared that based on its review, the NDA submission data are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval. - This withdrawal represents a significant setback for treating boys and young men with nonsense mutation DMD in the U.S. after over two decades of development efforts. - Duchenne muscular dystrophy is a rare and fatal genetic disorder affecting primarily males, causing progressive muscle weakness and premature death in the mid-20s.
- Keros Therapeutics received FDA orphan drug designation for KER-065 in August 2025, targeting Duchenne muscular dystrophy through selective inhibition of TGF-β ligands including myostatin and activin A. - The company reported initial top-line results from a phase I study in healthy volunteers in March 2025 and plans to initiate a phase II trial in DMD patients in Q1 2026. - KER-065 aims to address limitations of current glucocorticoid therapy by promoting muscle regeneration and strength while reducing side effects like muscle catabolism and bone loss. - The competitive DMD landscape includes established players like Sarepta Therapeutics with gene therapy Elevidys and PTC Therapeutics with corticosteroid treatments.
