To restrict access to therapy with KYNAMRO to patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia (HoFH).

FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia

The vote was close, with 9 for approval and 6 against, and only for treatment in a rare genetic disease called homozygous familial hypercholesterolemia (HoFH).

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The U.S. Food and Drug Administration today approved Kynamro (mipomersen sodium) injection as an addition to lipid-lowering medications and diet to treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).

FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia

Last night, the Food and Drug Administration announced the approval of Kynamro, a new drug to lower cholesterol in patients with homozygous familial hypercholesterolemia (HoFH), a rare genetic disease that causes sky-high cholesterol levels and heart attacks at a young age.

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The Committee noted that a high proportion of patients stopped taking the medicine within two years, even in the restricted group of patients with homozygous familial hypercholesterolemia, mainly due to side effects such as flu-like symptoms, injections site reactions and liver toxicity.

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KYNAMRO, given as a 200 mg weekly subcutaneous injection, has been approved as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (Apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia