Sephience Approval and Launch
Sephience received EU approval in late June and FDA approval shortly after, with a broad label for PKU patients aged 1 month and above. The U.S. revenue opportunity is expected to exceed $1 billion. European launch began in Germany, with early access mechanisms used in other countries.
Strong Financial Position
PTC closed the quarter with approximately $1.99 billion in cash, supporting all planned commercial and R&D initiatives. This financial strength allows for strategic business development and achieving cash flow breakeven without additional capital.
DMD Franchise Revenue
Achieved $96 million in revenue from the global DMD franchise, including continued Translarna revenue despite nonrenewal of EU conditional license, leveraging Article 117 in Europe.
Evrysdi Royalty Revenue
Roche's second quarter global revenue of approximately $559 million for Evrysdi resulted in $58 million in royalty revenue for PTC.