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US approval for Sanofi’s Kynamro in genetic cholesterol disorder

Comes one month after drug was turned down in Europe due to safety concerns

Sanofi building

Regulators in the US have approved Sanofi/Isis Pharmaceuticals' add-on cholesterol treatment Kynamro a month after it was turned down in Europe due to safety concerns.

The FDA said the drug should be available as an addition to lipid-lowering medications and diet to treat patients with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder that results in high cholesterol.

However, the approval contradicts the verdict from the European Medicines Agency, which determined that Kynamro (mipomersen) was effective at reducing levels of bad cholesterol, but the number of side effects, including the build-up of fat in the liver, was a concern considering the drug is intended for long-term use.

The FDA did take these concerns into consideration during its review, with an advisory committee only narrowly recommending the drug by 9 to 6 in October, 2012.

The final recommendation for Kynamro also comes with a risk evaluation and mitigation strategy (REMS), which has been designed to make sure prescriber and pharmacists ensure safe use of the drug, while the drug's label will contain a boxed warning on the risk of liver toxicity.

In addition, the approval comes with a requirement that Isis and Sanofi, which markets the drug through its Genzyme business, carry out four postmarketing studies.

These follow-on programmes include building a registry of patients with HoFH to determine the long-term safety of Kynamro, as well as a pharmacovigilance programme to monitor reports of malignancy, immune-mediated reactions and hepatic abnormalities in patients.

Despite these safety concerns, the drug, which is taken as a once-weekly injection, has been shown to reduce levels of low-density lipoprotein (LDL) cholesterol by 25 per cent in the first 26 weeks of treatment.

As HoFH is a rare condition, affecting about 6,000 people across the world, Kynamro has been granted an orphan drug license that will provide its manufacturers with tax credit and marketing incentive benefits.

HoFH can be a very serious condition from a young age, and teenagers often need coronary artery bypass grafts while deterioration of the aortic valve also occurs in sufferers.

Genzyme president and CEO, David Meeker, commented on what the approval means for people with the condition in the US.

"Today's FDA approval of Kynamro is great news for patients with HoFH who are in need of additional treatment options for this rare and often under-diagnosed disease.

“As the leader in treatments for rare diseases, we are pleased to bring our expertise to HoFH patients living with this serious condition to better help them manage their disease."

30th January 2013

From: Sales



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