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FDA NEWS RELEASE

For Immediate Release: Feb. 17, 2012
Media Inquiries: Morgan Liscinsky, 301-796-0397; morgan.liscinsky@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA approves Korlym for patients with endogenous Cushing’s syndrome

Today, Korlym (mifepristone) was approved by the U.S. Food and Drug Administration to control high blood sugar levels (hyperglycemia) in adults with endogenous Cushing’s syndrome. This drug was approved for use in patients with endogenous Cushing’s syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or who have not responded to prior surgery. Korlym should never be used (contraindicated) by pregnant women.

Prior to FDA’s approval of Korlym, there were no approved medical therapies for the treatment of endogenous Cushing’s syndrome.

Endogenous Cushing’s syndrome is a serious, debilitating and rare multisystem disorder. It is caused by the overproduction of cortisol (a steroid hormone that increases blood sugar levels) by the adrenal glands. This syndrome most commonly affects adults between the ages of 25 and 40. About 5,000 patients will be eligible for Korlym treatment, which received an orphan drug designation by the FDA in 2007.

Korlym blocks the binding of cortisol to its receptor. It does not decrease cortisol production but reduces the effects of excess cortisol, such as high blood sugar levels.

The safety and efficacy of Korlym in patients with endogenous Cushing’s syndrome was evaluated in a clinical trial with 50 patients. A separate open-label extension of this trial is ongoing. Additional evidence supporting the agency’s approval included several safety pharmacology studies, drug-drug interaction studies and published scientific literature. Patients experienced significant improvement in blood sugar control during Korlym treatment, including some patients who had marked reductions in their insulin requirements. Improvements in clinical signs and symptoms were reported by some patients.

The most common side effects experienced by endogenous Cushing’s syndrome patients treated with Korlym in clinical trials were nausea, fatigue, headache, arthralgia, vomiting, swelling of the extremities, dizziness and decreased appetite. Other side effects of Korlym include adrenal insufficiency, low potassium levels, vaginal bleeding and a potential for heart conduction abnormalities. Certain drugs used in combination with Korlym may increase its drug level. Health care professionals must be aware of the potential for drug-drug interactions and adjust dosing or avoid using certain drugs with Korlym. 

Korlym should never be used by pregnant women. Although pregnancy is an extremely rare occurrence in Cushing’s syndrome patients because of the suppressive effect of excess cortisol on female reproductive function, Korlym will carry a Boxed Warning advising health care professionals and patients that the therapy will terminate a pregnancy.

The FDA has determined that a Risk Evaluation and Mitigation Strategy (REMS) is not necessary for Korlym to ensure that the benefits outweigh the risks for patients with endogenous Cushing’s syndrome. Several factors were considered in this determination including the following:

• There are no other approved medical therapies for this debilitating form of Cushing’s syndrome and very sick patients would suffer if impediments to access were imposed.
• The number of Cushing’s syndrome patients who will require treatment with Korlym is small, with an estimated 5,000 patients being eligible for treatment.
• The number of health care professionals in the United States who would potentially prescribe Korlym is very small and highly specialized. They are familiar with the risks of Korlym treatment in the endogenous Cushing’s syndrome population and frequently monitor patient status.
• The risks of Korlym treatment in the intended population can be managed through physician and patient labeling. The risks associated with Korlym will be outlined in a medication guide for patients.

The company has voluntarily proposed distributing Korlym through a central pharmacy to ensure the timely, convenient and appropriate delivery of the drug to Cushing’s patients or to the health care institutions where this therapy may be initiated. Most retail pharmacies are unlikely to keep adequate supplies of the drug for this rare condition and central distribution will give patients with Cushing’s syndrome better access to Korlym. 

Korlym is manufactured by Corcept Therapeutics of Menlo Park, Calif.

For more information:

• Approved Drugs: Questions and Answers

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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Page Last Updated: 02/17/2012 

From http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm292462.htm

Brochures for Primary Care Physicians, to increase awareness of Cushing’s syndrome. In English and Spanish

These were sent to me by a Facebook friend who wrote:

it’s Vida A, I have writen in the facebook group that I would like to upload some information that I think it’s very useful. Most of it is in Spanish but maybe someone is able to understand it.

I’ve found it in: www.ciberer.es

 

then you go to enfermedades raras > divulgación social > boletin social  and then it’s in Boletin Social number 3 (dedicated to Cushing)

I think it may be very useful

Thanks very much

Núria (Vida A)

GUIA_CUSHING_completa.pdf Download this file

Brochure_for_Primary_Care_physicians_-_final_version.pdf Download this file

Excerpt from: http://thechart.blogs.cnn.com/2012/02/14/
making-sense-of-your-childs-health-numbers/

“…Unfortunately, as obesity becomes more common among children, diseases once thought to be primarily a problem of adulthood are showing up in kids. For this reason, children should be screened for medical complications of obesity, such as diabetes, high blood pressure and high cholesterol.

Not everyone agrees when you should start screening your children, but the latest recommendation is to test cholesterol between 9 and 11 years old and then again between 17 and 21. Ask your pediatrician for his or her opinion based on your child’s BMI. Blood pressure can be measured with a cuff at the doctor’s office, and diabetes and high cholesterol can be checked with simple blood tests.

These tests may include blood sugar, hemoglobin A1c and insulin levels for diabetes; a lipid panel (such as cholesterol and triglycerides) which, along with blood pressure, is a marker for heart disease; and tests for liver problems that can occur with obesity.

In some situations it might be important to rule out medical causes of obesity. For example, patients who have a higher concentration of fat in the neck and head region could have Cushing’s syndrome, and those who are relatively short for their weight and age and have other symptoms might have low thyroid function…”

Read the entire article at http://thechart.blogs.cnn.com/2012/02/14/
making-sense-of-your-childs-health-numbers/

Authors: Toja, Paola M.1; Branzi, Giovanna2; Ciambellotti, Francesca2; Radaelli, Piero3; De Martin, Martina1; Lonati, Laura Maria2; Scacchi, Massimo; Parati, Gianfranco; Cavagnini, Francesco1; Giraldi, Francesca Pecori

Source: Clinical Endocrinology, Volume 76, Number 3, 1 March 2012 , pp. 332-338(7)

Publisher: Wiley-Blackwell

Abstract:

Objectives  Sustained hypercortisolism impacts cardiac function, and, indeed, cardiac disease is one of the major determinants of mortality in patients with Cushing’s syndrome. The aim of this study was to assess the clinical relevance of cardiac structure and function alterations by echocardiography in patients with active Cushing’s syndrome and after disease remission.

Study design  Seventy-one patients (61 women, 10 men) with Cushing’s syndrome and 70 age-, sex- and blood pressure-matched controls were enrolled. Echocardiography was performed in 49 patients with active disease and at several time points after remission in 44 patients (median follow-up 46·4 months), and prevalence of abnormal left ventricular mass measurements and systolic and diastolic functions indices was compared between patients with active disease, after remission and controls. Twenty-two patients were evaluated both before and after remission.

Results  Up to 70% of patients with active Cushing’s syndrome presented abnormal left ventricular mass parameters; 42% presented concentric hypertrophy and 23% concentric remodelling. Major indices of systolic and diastolic functions, i.e. ejection fraction and E/A ratio, respectively, were normal. Upon remission of hypercortisolism, left ventricular mass parameters ameliorated considerably, although abnormal values were still more frequent than in controls. Both cortisol excess and hypertension contribute to cardiac mass alterations and increase the prevalence of target organ damage.

Conclusions  Cushing’s syndrome is associated with an increased risk for abnormalities of cardiac mass, which ameliorates, but does not fully disappear after remission. Systolic and diastolic functions are largely within the normal range in these patients.

Document Type: Research article

DOI: http://dx.doi.org/10.1111/j.1365-2265.2011.04206.x

Affiliations: 1: Ospedale San Luca, Neuroendocrinology Research Lab, Istituto Auxologico Italiano IRCCS 2: Department of Cardiology, Ospedale San Luca, Istituto Auxologico Italiano IRCCS 3: Department of Medical Sciences, University of Milan

Buy this article here: http://www.ingentaconnect.com/content/bsc/cend/2012/00000076/00000003/art00004