FDA acts to approve a new cholesterol lowering medicine called evolocumab (Repatha)

king The FDA approved evolocumab (Repatha)

August 27, 2015

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kingThe FDA approved evolocumab (Repatha)

August 27, 2015

The FDA approved a new cholesterol lowering medicine called Repatha for people who are not able to get their low-density lipoprotein (LDL) under control with diet and currently available medicines in the statin (HMGCoA reductase inhibitors) family. This follows the European Medicines Agency (EMA) approval of Repatha as the first-in-class PCSK9 inhibitor treatment May 22, 2015. The FDA previously approved a PCSK9 inhibitor called alirocumab (Praluent) for heterozygous familial hypercholesterolemia.

Repatha belongs to this new family (class) of medicines known as PCSK9 inhibitors. The medicine is actually a monoclonal antibody that inhibits PCSK9 and that inhibition leads to lower cholesterol. It is approved “for some patients who are unable to get their low-density lipoprotein (LDL) cholesterol under control with current treatment options.”

Repatha is approved (indicated for):

1)      Use in addition to diet and maximally-tolerated therapy with statin type (such as Lipitor-atorvastatin or Crestor-rosuvastatin) therapy in adults with heterozygous familial hypercholesterolemia  (HeFH) or

2)      Use in homozygous familial hypercholesterolemia (HoFH) or

3)      Use in clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol.

More information on the uses and terms in this release is available at:

Statin therapy:


Atherosclerotic cardiovascular disease:

A long-term study called FOURIER is currently underway. Fourier stands for Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk. The purpose of this study is to see if evolocumab (Repatha) is well-tolerated and decreases the risk of cardiovascular death, heart attack (myocardial infarction), hospitalization for unstable angina, stroke or coronary revascularization in people (subjects) with clinically evident cardiovascular disease. Visit and search NCT01764633 for more information.

Every medicine has a profile of benefits and risks as you've seen in the Essential Guide To Prescription Drugs. Most common side effects of Repatha include inflammation of the nasopharynx (nasopharyngitis), upper respiratory tract infection, flu, back pain, and reactions such as redness, pain, or bruising where the shot (injection) is given. There have been allergic reactions reported (such as rash and hives). People should stop taking Repatha and get medical help if they start to have these symptoms. The FDA press release is at

PDUFA day for evolocumab (Repatha) at the US FDA

surfer The first PCSK9 inhibitor approved in the world stands before the FDA today for possible US approval. Evolocumab (brand name is Repatha) was approved by the European Medicines Agency (EMA) May 22, 2015.

Repatha (evolucomab)- First PCSK9 Inhibitor in the world is approved in Europe

 building at DawnFirst PCSK9 Inhibitor in the world is approved in Europe


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 building at DawnFirst PCSK9 Inhibitor in the world is approved in Europe


We've seen the dawn of a new family of medicines to treat cholesterol. The European Commission (agency analogous to the FDA in the US) approved evolocumab (Repatha) July 21, 2015 in Europe. This new class (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:

  1. 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).
  2. 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:

1)      Lowering

  • 1) cardiovascular death
  • 2) heart attack (MI)


2)      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 : 

This website is not intended as medical advice, and you should consult your doctor before changing or adding any medicines or vitamins to those you may now be taking and about applying any strategies BEFORE you adopt any approach in this report. While diligent care has been taken to ensure the accuracy of the information provided during the preparation of this edition, no claim is made that all known actions, uses or side effects, strategies for cost containment, targets or cholesterol pathways are included in this report. The accuracy and currentness of information are ever subject to change relative to new guidelines, new information derived from drug research, development and general usage.