Stabilizer-first treatment gains ground in ATTR-CM
The failure of a major silencer trial has strengthened the case for stabilizers as the starting point of ATTR-CM treatment, according to analysts and company executives.
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The failure of a major silencer trial has strengthened the case for stabilizers as the starting point of ATTR-CM treatment, according to analysts and company executives.
Stabilizers bind transthyretin in the bloodstream and stop it breaking apart, a process that leads to amyloid deposits in the heart. Silencers cut production of the protein in the liver. After CARDIO-TTRansform, STAT reported that analysts saw the result as a point in favor of oral stabilizers over injectable silencers.
BridgeBio management told investors the failure could reinforce stabilization as a first-line standard, while saying it would wait for fuller data before updating its market-share outlook. Attruby's prescribing information describes near-complete stabilization of the protein in lab measurements from day 28 through a 30-month study.
Route of administration is also in play. Attruby and Vyndamax are taken by mouth at home. Amvuttra's label says it should be given by a healthcare professional every three months.
Current clinical guidance does not name a single first-line drug, and researchers say future trials will need to be designed around treatment on top of stabilizers.