SPL84-23 for CF Splicing Mutation Named Orphan Drug in US, Europe

SPL84-23 for CF Splicing Mutation Named Orphan Drug in US, Europe

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SPL84-23 for CF Splicing Mutation Named Orphan Drug in US, Europe

Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug status to SPL84-23, SpliSense’s investigational therapy for cystic fibrosis (CF) patients carrying a common disease-causing splicing mutation. The designation is given to treatment candidates with the potential to be safe and effective for rare, life-threatening, or chronically debilitating conditions that have no approved treatments, or where an experimental therapy shows a potential for significant benefit over existing…

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