FDA Panel Backs Zevra's Arimoclomol For Rare Genetic Disorder, Analyst Highlights Balanced Portfolio Of Marketed Products And Pipeline Assets Focused On Rare Diseases
Portfolio Pulse from Vandana Singh
The FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of Zevra Therapeutics' (NASDAQ:ZVRA) arimoclomol for treating Niemann-Pick disease type C. William Blair increased the probability of success for arimoclomol to 90% and highlighted Zevra's balanced portfolio. Zevra's stock rose 16.7% in premarket trading.
August 05, 2024 | 11:59 am
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The FDA’s Genetic Metabolic Diseases Advisory Committee voted in favor of Zevra Therapeutics' arimoclomol for treating Niemann-Pick disease type C. William Blair increased the probability of success for arimoclomol to 90%, and Zevra's stock rose 16.7% in premarket trading.
The positive vote from the FDA panel significantly increases the likelihood of arimoclomol's approval, which is a major milestone for Zevra. The increased probability of success from William Blair and the stock's premarket rise indicate strong investor confidence.
CONFIDENCE 95
IMPORTANCE 90
RELEVANCE 100