PTC launches Sephience with $125M Q1 sales; Translarna/Emflaza decline, vatiquinone CRL delays approval

Filed May 7, 2026 · Period ending March 31, 2026 · Compared to 10-Q May 6, 2025 · ~2 min read

Key Changes

• high

  Sephience (sepiapterin) for PKU launched in U.S., EU, Japan, and Brazil in 2025, generating $124.6M in Q1 2026 net sales—a major new revenue stream offsetting legacy product declines.

  MD&A: Sephience commercial launch verify on EDGAR →
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  Translarna net sales fell 32% to $59.0M (from $86.2M) after EU conditional marketing authorization was not renewed in March 2025; U.S. NDA withdrawn in February 2026 with no further development planned.

  MD&A: Translarna EU/U.S. regulatory setbacks verify on EDGAR →
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  Emflaza net sales dropped 55% to $21.5M (from $47.8M) due to generic competition; remaining exclusivity for patients 2-5 years expires June 2026, signaling further erosion.

  MD&A: Emflaza net sales decline verify on EDGAR →