FDA rejects Alnylam’s Onpattro label expansion after adcomm voted in favor
The FDA has rejected Alnylam Pharmaceuticals’ expansion plans for its siRNA injection Onpattro, serving a complete response letter for the treatment of ATTR cardiomyopathy.
The Monday morning surprise no-go comes weeks after the agency’s panel of outside advisors had voted 9 to 3 in favor of the drug’s benefits outweighing its risks. The FDA raised questions at the adcomm on the effect size in this indication, and independent experts had agreed that Onpattro’s benefit was small, but risks were smaller for the patients who experience malformed protein buildup in the heart.
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