Quiet Riot? Gene Silencing Candidates Still Ready To Make Noise In Lipid Disorders
Despite complete response letter for Akcea's Waylivra, RNA therapeutics may have regulatory pathway to approval for lipid disorders.
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Akcea Therapeutics has lowered its asking price in England for its antisense therapy for familial chylomicronaemia syndrome. It has also struck a pricing and reimbursement deal for the drug in Germany.
The $9.7bn acquisition hinges on the small interfering RNA inclisiran, but an ongoing cardiovascular outcomes trial, manufacturing hurdles, and reimbursement challenges provide subjects for concern.
With one rare disease drug already available in Europe, and a second imminent, Akcea’s CEO outlined in a Scrip interview the European plans for this still-youthful US biotech.