New Medication for Treating Cholesterol Disorder

Drug developers at the Perelman School of Medicine, Pennsylvania have found a new drug to treat patients with hypercholesterolemia (HoFH).

The report was made in the latest issue of The Lancet.

The drug is called lomitapide, which reduces LDL or Bad Cholesterol in patients with HoFH.

Homozygous Familial Hypercholesterolemia (HoFH)

The disease is characterized by elevated levels of cholesterol in the blood – greater than 500 mg/dl that disables liver from removing LDL from blood. This results in heart and vascular conditions at a very early age and finally leads to death by the age of 30 years.

The regular statin drugs are not very effective in treating this condition. A procedure involving physical removal of LDL from the blood – apheresis is the only known treatment so far.  It is invasive and must be performed at least once every 2 weeks.

Lomitapide – The New Drug

The drug lomitapide works by inhibiting this MTP. Microsomal triglyceride transfer protein (MTP) is required for making LDL. As part of the study, 29 HoFH patients were enrolled. 23 of them have completed the safety and efficacy phases successfully. The new drug was administered along with the traditional treatment.

The drug dosage was increased gradually from 5 mg up to a maximum of 60 mg per day. At the end of the study duration, more than a third of them showed lowered LDL cholesterol levels – as less as 100 mg/dl. This is close to the therapeutic goals recommended. This has provided a new ray of hope for those suffering from this extraordinary and rare fatal condition.

Clinical research of the drug has reached the final stages, and once it is cleared, and certifications obtained, then patients suffering from Hypercholesterolemia can hope to be able to survive longer and lead a better lifestyle than they did.