PTC Therapeutics, Inc.
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- PTC Therapeutics, Inc. (PTCT) is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for rare disorders, including Duchenne muscular dystrophy (DMD), aromatic L-amino acid decarboxylase (AADC) deficiency, phenylketonuria (PKU), Friedreich's ataxia (FA), and spinal muscular atrophy (SMA). Founded in 1998 and headquartered in Warren, New Jersey, the company offers approved therapies such as Translarna (ataluren), a small molecule splicing modifier for nonsense mutation DMD; Emflaza (deflazacort), a corticosteroid for DMD; Evrysdi (risdiplam), an SMA treatment developed in collaboration with Roche and the SMA Foundation; and Kebilidi (eladocagene exuparvovec-tneq), the first gene therapy directly infused into the brain for AADC deficiency, approved by the FDA in November 2024. Its pipeline includes sepiapterin for PKU, with an FDA action date of July 29, 2025; vatiquinone for FA, with filing acceptance anticipated in Q1 2025; PTC518, an oral therapy for Huntington's disease; and other candidates targeting post-transcriptional control mechanisms in orphan diseases. PTC operates globally, with commercial activities in the U.S. and select international markets, generating net product revenues of approximately $814 million in 2024, driven by strong DMD franchise performance. Recent developments include a $1.0 billion upfront licensing deal with Novartis for PTC518 closed in late 2024, FDA approval of Kebilidi, regulatory submissions for sepiapterin, vatiquinone, and Translarna label expansion, and a cash balance of $2.1 billion as of year-end 2024 following the Novartis proceeds.
Company News
PTC Therapeutics to Participate in Upcoming Investor Conferences
PTC Therapeutics Announces New Sephience™ (sepiapterin) Data Reinforcing Broad Patient Benefits Including Sustained Metabolic Control and Diet Liberalization as well as Consistent Favorable Safety Profile
PTC Therapeutics: An Acquisition Which Could Unlock Massive Potential
PTC Therapeutics Targets Fabry Gene Therapy in $111M Bankruptcy Auction Deal
PTC Therapeutics Expands Rare Disease Portfolio with 'Unique Opportunity'
PTC Therapeutics, Inc. (PTCT) Discusses Acquisition of ST-920 Fabry Gene Therapy Asset and Clinical Data from STAAR Study Transcript
PTC to Expand Rare Disease Portfolio with Acquisition of BLA-Stage ST-920 Fabry Disease Program
PTC Therapeutics (PTCT) Loses 20.3% in 4 Weeks, Here's Why a Trend Reversal May be Around the Corner
PTC Therapeutics (PTCT) Q2 Earnings: Taking a Look at Key Metrics Versus Estimates
PTC Therapeutics, Inc. (PTCT) Q2 2026 Earnings Call Transcript
PTC Therapeutics (PTCT) Beats Q2 Earnings and Revenue Estimates
PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results
PTC Therapeutics, Inc. $PTCT Shares Purchased by Fifth Third Bancorp
Hamilton Lane Board Elects Michael Schmertzler as New Independent Director
PTC Therapeutics, Inc. $PTCT Shares Purchased by Bessemer Group Inc.
PTC Therapeutics to Report Second Quarter 2026 Financial Results on Thursday, July 30, 2026
What This PTC Therapeutics Insider Sale Means as Product Revenue Hit $226 Million
Reasons Why Investors Should Consider Betting on PTC Stock Now
Stocks Look Shaky, But This Sector is Booming (Biotech)
PTC Therapeutics (PTCT) Upgraded to Strong Buy: Here's What You Should Know