Thursday, Isis announced trials that could get mipomersen approved in the rare genetic disease familial hypercholesterolemia (FH).
This is the very worst form of a genetic disorder called familial hypercholesterolemia.
Familial hypercholesterolemia emerged as a way to get it on the market fast.
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
It shows promise for all forms of high cholesterol, but the prospects are best in a rare disease called familial hypercholesterolemia (FH).
The ENHANCE study was conducted in 720 patients with heterozygous familial hypercholesterolemia, a genetic disease that can boost untreated cholesterol levels above 300.
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).
The novel antisense oligonucleotide works by inhibiting the synthesis of apo-B and is under development in the United States and Europe for the treatment of familial hypercholesterolemia.
FORBES: CHMP Recommends Against Approval For Mipomersen In Europe
In another study of patients with heterozygous familial hypercholesterolemia, a genetic disorder that caused high cholesterol, patients who took REGN727 saw their cholesterol decline between 30% and 65% depending on how the medicine was dosed.
FORBES: New Drug Leads To Dramatic Reductions In Bad Cholesterol
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
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
He will continue to prescribe the drug to his patients, who all suffer from the genetic illness familial hypercholesterolemia and desperately need their cholesterol lowered, but he wants more evidence about how Zetia works before a big study of the drugs benefits concludes in 2012, at least a year later than originally expected.
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