First PCSK9 Inhibitor in the world is approved in Europe

First PCSK9 Inhibitor in the world is approved in Europe

The European Commission (agency analogous to the FDA in the US) approved evolocumab (Repatha) July 21, 2015 in Europe. This new family of medicines offers great hope for treating abnormal cholesterol. Prescribers in Europe may soon be able to be use Repatha for:

1)      Treating adult patients who have a kind of cholesterol problem known as primary hypercholesterolemia (Heterozygous familial and non-familial HeFH or mixed dyslipidemia in addition to dietary steps. In these patients, Repatha can be used:

Alone or in combination with other lipid-lowering treatments when they don’t tolerate an earlier approved family of medicines known as statins (HMG-CoA reductase inhibitors such as Lipitor [atorvastatin] or rosuvastatin [Crestor]. This is a novel approach for such statin-intolerant people. Repatha will also be able to be used in patients who should not be given a statin (those for whom a statin is contraindicated).
In combination with a statin or statin combined with other lipid-lowering therapies in people who do not reach their LDL cholesterol goals with the maximum tolerated dose of a statin, OR

2)      Treating of adults or adolescents who are 12 years old or older who have homozygous familial hypercholesterolemia (HoFH) in combination with other lipid-lowering therapies.

While this discussion is somewhat complicated, several Repatha Essentials emerge in this exciting new topic:

In Europe, current therapies (statins and other approved lipid-lowering medicines) fail to adequately lower bad cholesterol (LDL-C) over 60% of the time in high risk patients.
In very high risk patients, previously available treatments failed more than 80% of the time to lower bad cholesterol enough.
Repatha is the first PCSK9 inhibitor to be approved anywhere in the world.
Problems from Repatha (adverse event or AE profile) was similar overall to the placebo (control) groups of patients. The most common Adverse Reactions (ARs) more than or equal to 2% were nausea, flu (influenza)joint or back pain, upper respiratory tract infection or nose or throat soreness (nasopharyngitis). Most new medicines are studied after approval have results based on approval studies and reports are submitted which show more about potential problems as time goes on. The FOURIER study below will undoubtedly tell more about additional benefits from Repatha treatment and while not expected, will reveal any problems not seen in the approval studies.
The cost of cardiovascular disease (CVD) in the European Union (EU) is immense and totals roughly 106 billion Euros each year.
While Repatha may be reasonably expected (based on the ability to lower LDL cholesterol and thinking toward parallel results from the statin family of medicines) to decrease problems from and death from heart disease (cardiovascular morbidity and mortality), this is still unknown. There is a large study (clinical trial) called FOURIER- Further Cardiovascular Outcomes Research with PCSK9 Inhibition in Subjects with Elevated Risk (FOURIER) which should let us know that answer. This is a 22 500-patient trial comparing evolocumab to statin treatment to see how it compares in:

  • Lowering
    • cardiovascular death
    • heart attack (MI)
  • Lowering hospitalization for unstable angina, stroke, or coronary revascularization.

The full results from FOURIER won't be available until 2018 at the earliest.

Because evolocumab (Repatha) is the first proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor in the world, I will most likely create a profile for the new Essential Guide to Prescription Drugs, using Repatha as the representatve member of this impressive new family. Once the results of the FOURIER study are known, I may opt to include it in the True Breakthroughs in Medicines section.

The original European press release is available at :  

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