Is mipomersen discontinued?
As a matter of fact, mipomersen therapy is more likely discontinued and associated with increased risk of injection-site reactions, hepatic steatosis, hepatic enzyme elevation, and flu-like symptoms.
Is mipomersen available in the US?
On January 29, the U.S. FDA approved mipomersen sodium injection as an adjunct to treat LDL-C, Apo B, total cholesterol, and non HDL-C in patients with homozygous familial hypercholesterolemia (HoFH).
Why is mipomersen an orphan drug?
Mipomersen is a novel antisense oligonucleotide inhibitor of apolipoprotein B. It is approved by the FDA only as an orphan drug for use in HoFH, a relatively rare genetic condition. Ongoing clinical trials are being conducted to evaluate the safety and efficacy of mipomersen in varying populations.
What is the mechanism of action of mipomersen?
Mechanism of action Mipomersen binds to the messenger RNA coding for apolipoprotein B-100 (ApoB-100), a protein that is the main component of low-density lipoprotein (LDL) and very low-density lipoprotein (VLDL). As a consequence, the RNA is degraded by the enzyme ribonuclease H, and ApoB-100 is not translated.
Who treats homozygous familial hypercholesterolemia?
Homozygotes and difficult to treat heterozygotes should be referred to endocrinologists or lipidologists. The various cardiovascular manifestations, at one time or another, may require consultation with cardiologists, neurologists, radiologists, and cardiac or vascular surgeons.
Are high HDL levels good?
For HDL cholesterol, or “good” cholesterol, higher levels are better. High-density lipoprotein (HDL) cholesterol is known as the “good” cholesterol because it helps remove other forms of cholesterol from your bloodstream. Higher levels of HDL cholesterol are associated with a lower risk of heart disease.
When was mipomersen approved by the FDA?
In January 2013, the FDA approved mipomersen (Kynamro, Genzyme), an antisense oligonucleotide inhibitor of apo B, as an orphan drug for the management of HoFH. Mipomersen is indicated as an adjunct therapy to lipid-lowering agents and to lifestyle changes for reducing LDL-C, apo B, TC, and non–HDL-C levels.
How much does Kastle pay Ionis for Kynamro?
However, 3% of the cash payments that Ionis receives from Kastle—up to $2.85 million—plus a 3% royalty on Kynamro sales—will be earned by Genzyme.
What kind of cholesterol does Kynamro treat?
Kynamro is indicated as a treatment for a rare form of high cholesterol, homozygous familial hypercholesterolemia (HoFH). The deal marks Kastle’s first acquisition since it was formed last year.
What kind of drug is Kastle Therapeutics Kynamro?
Kastle Therapeutics has acquired global rights to develop and commercialize the marketed drug Kynamro ® (mipomersen sodium) injection from Ionis Pharmaceuticals for up to $95 million-plus. Kynamro is indicated as a treatment for a rare form of high cholesterol, homozygous familial hypercholesterolemia (HoFH).
How is Kynamro used in the treatment of HoFH?
Kynamro is indicated 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 HoFH.