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Akcea Faces Tricky Risk/Benefit Scenario In Orphan Disease FCS

 
• By Joseph Haas

The Ionis subsidiary's volanesorsen shows robust efficacy in a pivotal Phase III trial for familial chylomicronemia syndrome, but with high dropout levels for platelet reduction. Firm says platelet reductions are manageable and stem from the drug working as expected in the rare disease.

test tube

Ionis Pharmaceuticals Inc. subsidiary Akcea Therapeutics Inc. now has Phase III efficacy data in hand for volanesorsen in familial chylomicronemia syndrome (FCS), but market analysts are mixed on the implications of safety data showing frequent instances of reduced platelet counts, potentially leading to thrombocytopenia.

FCS, a rare disease affecting an estimated 3,000-5,000 patients worldwide, results in extremely high triglyceride levels and can lead to...

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