Most of the cancers observed in the clinical trials occurred in elderly patients who did not have homozygous FH.
FORBES: FDA Panel Recommends Approval Of Mipomersen For Familial Hypercholesterolemia
In the pivotal trial with homozygous FH patients mipomersen reduced LDL by 24.7%.
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 CHMP noted that Kynamro was effective at reducing LDL cholesterol levels in patients with homozygous and severe heterozygous familial hypercholesterolaemia.
FORBES: Read The European Medicines Agency's Smackdown Of Kynamro
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).
Several panelists thought that the cancer signal may have reflected an ascertainment bias, and, further, that a young homozygous FH population would be less susceptible to an elevated cancer risk.
FORBES: FDA Panel Recommends Approval Of Mipomersen For Familial Hypercholesterolemia
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.
FORBES: CHMP Recommends Against Approval For Mipomersen In Europe
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 hypercholesterolaemia, mainly due to side effects such as flu-like symptoms, injections site reactions and liver toxicity.
FORBES: Read The European Medicines Agency's Smackdown Of Kynamro
Given the severity and rarity of homozygous FH it is difficult to predict how the committee will vote, but the safety concerns raised by the FDA make it extremely unlikely the drug could receive an expanded indication for heterozygous FH anytime in the near future.
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
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