2 hours ago 1

PTC Therapeutics, Inc. (PTCT) Launches SUFIANCE for PKU

Thu, Sep 11, 2025, 10:15 AM 2 min read

In this article, we will be taking a look at the 11 Best Performing Biotech Stocks So Far in 2025. PTC Therapeutics, Inc. is one of them.

PTC Therapeutics, Inc. (NASDAQ:PTCT) is reinforcing its position in rare disease innovation with the launch of SUFIANCE (sepiapterin) for phenylketonuria (PKU). Recently approved by both the FDA and EU, SUFIANCE rolled out in Germany in July 2025, with a U.S. launch imminent. The drug is projected to generate over $1 billion annually in the U.S., making it a central driver of PTCT’s growth. To accelerate adoption, the company is leveraging early access programs and strategic partnerships to expand global reach.

While SUFIANCE strengthens its commercial momentum, PTC Therapeutics, Inc. (NASDAQ:PTCT) continues to navigate challenges in its pipeline. The FDA recently issued a Complete Response Letter for vatiquinone, its Friedreich’s ataxia candidate, requesting additional clinical data before resubmission. The corporation is working closely with regulators to address these concerns while keeping its broader rare disease studies on track.

PTC Therapeutics, Inc. (PTCT) Launches SUFIANCE for PKU

Financially, PTC Therapeutics, Inc. (NASDAQ:PTCT) remains well-positioned. Management emphasized at the Cantor Global Healthcare Conference that SUFIANCE’s revenue potential, combined with $1.9 billion in cash reserves, supports a path toward cash-flow breakeven. This stability allows PTCT to continue advancing its pipeline, including promising gene therapy programs such as PTC518 for Duchenne muscular dystrophy (DMD).

While we acknowledge the potential of PTCT as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.