PTC Therapeutics (PTCT)의 주가는 최근 크게 흔들렸지만, 장기적 기반을 보면 주목할 만합니다. 2025년 7월 29일, 미국 FDA는 보도자료에서 이렇게 발표했습니다. "The U.S. Food and Drug Administration has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in patients 1 month of age and older." (Source: FDA News Release, 2025-07-29) 이 희귀질환 치료제는 미국과 유럽에서 모두 승인된 최초 경구약 중 하나로, PKU 환자에게 실질적 치료 옵션을 제공합니다. 재무적으로도 안정적입니다. PTC의 2025년 2분기 SEC 보고서에는 이렇게 명시되어 있습니다. "We ended the quarter with approximately $2.04 billion in cash, cash equivalents, and marketable securities." (Source: PTC Therapeutics Q2 2025 Form 10-Q, SEC) 이렇게 충분한 유동성은 상용화와 연구개발 모두를 안정적으로 지원할 수 있는 기반이 됩니다. 2025년 하반기에는 두 가지 중요한 FDA 이벤트가 예정되어 있습니다. "Vatiquinone (Friedreich’s ataxia) – FDA review expected in 2025" "PTC518 (Huntington’s disease) – Phase 3 enrollment ongoing, regulatory submission planned 2025" (Source: PTC Therapeutics Pipeline Update, 2025) 또한 2024년 12월, 노바티스와 PTC518 라이선스 계약을 체결하며 10억 달러의 선불금과 추가 마일스톤 지급 약정을 확보했습니다. (Source: Reuters, 2024-12-02) 단기적으로 시장은 불안정할 수 있지만, 승인된 제품의 상업화, 파이프라인 진전, 그리고 충분한 재무 역량은 회사의 장기적 가치 상승 가능성을 높이고 있습니다. PTCT’s Rare Disease Potential Remains Strong PTC Therapeutics (PTCT) has experienced significant volatility recently, but its long-term fundamentals remain noteworthy. On July 29, 2025, the U.S. FDA announced in a press release: "The U.S. Food and Drug Administration has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in patients 1 month of age and older." (Source: FDA News Release, 2025-07-29) This rare disease therapy is one of the first oral treatments approved in both the U.S. and the EU, providing a meaningful option for PKU patients. Financially, the company is solid. PTC Therapeutics’ Q2 2025 SEC filing states: "We ended the quarter with approximately 2.04 billion dollars in cash, cash equivalents, and marketable securities." (Source: PTC Therapeutics Q2 2025 Form 10-Q, SEC) This liquidity provides a strong foundation for both commercialization and continued R&D investment. Two key FDA events are expected in the second half of 2025: "Vatiquinone (Friedreich’s ataxia) – FDA review expected in 2025" "PTC518 (Huntington’s disease) – Phase 3 enrollment ongoing, regulatory submission planned 2025" (Source: PTC Therapeutics Pipeline Update, 2025) In addition, in December 2024, PTC signed a licensing deal with Novartis for PTC518, securing a 1 billion dollar upfront payment plus additional milestone payments. (Source: Reuters, 2024-12-02) While short-term market sentiment may remain shaky, the combination of an approved product entering the market, advancing late-stage pipeline programs, and strong financial resources suggests that the company’s long-term valuation potential remains compelling.