Duchenne Hope Fades: Rare Disease Biotech Loses 190 Billion Won in Market Value in a Day

PTC Therapeutics, Inc. (NASDAQ: PTCT) closed at $71.70 on the Nasdaq on the 16th, a 2.54% decline. Its market capitalization stands at approximately $5.9 billion (around KRW 7.7 trillion), with about $147 million (roughly KRW 190 billion) wiped out in a single day and trading volume reaching 1.04 million shares.

In February, after the U.S. FDA indicated that Translarna (ataluren), its treatment for Duchenne muscular dystrophy, was unlikely to meet approval requirements, PTC voluntarily withdrew the related New Drug Application. On March 31, the company informed the Duchenne community that it would continue supplying Translarna to existing U.S. patients for approximately six months. (drugs.com)

PTC Therapeutics is a U.S. biotech company specializing in rare diseases and gene expression modulation. Its rare-disease portfolio includes Evrysdi for spinal muscular atrophy and Sepians for phenylketonuria. In 2025, the company reported total revenues of $1.731 billion—including $831 million in product and royalty income (about KRW 2.2 trillion)—but pipeline setbacks like Translarna continue to influence investor sentiment. (en.wikipedia.org)