Cholesterol lowering is linked to lower risk of cardiac episodes and is very well managed by treatment with a class of drugs called statins that include Lipitor, Zocor, etc. Lipitor and other statins are now available as cheap generic drugs and have a very well-established safety and efficacy profile. So, there was obvious skepticism to a new class of injectable cholesterol lowering drugs developed by drug giants Sanofi and Amgen. Both Amgen and Sanofi have each developed monoclonal antibody products for control of bad cholesterol in patients who do not respond well to statins alone. Both biological drugs work by blocking PCSK9 that interferes with liver’s ability to clear LDL cholesterol (the bad cholesterol). The drugs are considered as the first major breakthrough in research since the launch of popular statin drugs in the 80s. Both drugs dropped bad cholesterol level significantly without any safety concerns. FDA scientists, however, opined that the approval should be withheld till further safety studies on these drugs have not been completed. Previously such drugs have been linked to higher rates of heart problems and death in patients in the studies. The FDA Advisory panel agreed that the additional safety of these products should be completed but recommended their immediate approval for those patients whose bad cholesterol is not managed with statins alone due to genetic diseases. The limited approval will provide earlier access of these new treatments to patients who may benefit the most, while limiting their use in general population. FDA is expected to make a final decision in the next month or two for both. Although FDA is not bound by law to accept the recommendations of the Advisory Committee, traditionally it does agree with the recommendations, and most analyst believe that these drugs will be approved for treatment of patients with genetic diseases making them resistant to statins. From a practical point of view, these drugs may be used off-label by other patients. We should see insurance battles for availability of these new very expensive therapies in the age of abundant generic statins. We will keep you posted.
Source: The Washington Post
