Day 11: Drug Therapy for Cushies Finally Available

Today is the day my Cushie friends and I have been waiting for.  Finally, a drug therapy is available to all of us Cushies stuck in a persistent hypercortisolemic state.  Korlym, the first FDA-approved drug in the United States, is available to patients who need it and want it.

I have been watching the release of this drug closely, and I found what I was looking for today.  This Patient Guide for Korlym outlines all the ins and outs of taking this drug.  

Today, I am happy to share the statement that was music to my ears: 

"We are determined that every patient who could benefit from Korlym will receive it," added Steven Lo, Corcept's Vice President of Commercial Operations. 

If you are a Cushing's patient burdened by the high costs of diagnosis -- lab work, doctor visits, travel -- please be sure to check into SPARK (Support Program for Access and Reimbursement for Korlym). I like to see them making good on that promise that the Veep made. :)

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4/10/12 - Corcept Therapeutics Announces National Product Launch of Korlym(TM) for Patients With Endogenous Cushing's Syndrome

MENLO PARK, CA (Marketwire) 04/10/12 Corcept Therapeutics Incorporated (NASDAQ: CORT) announced today that Korlym™ (mifepristone) 300 mg Tablets is available by prescription nationwide. On February 17, 2012, the U.S. Food and Drug Administration (FDA) approved Korlym as a once-daily oral medicine to control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. Korlym is the first FDA-approved treatment for this illness. "After using mifepristone in the company's clinical trial, I am pleased that it will be available for the medical management of my patients with Cushing's syndrome for whom surgery failed," said James W. Findling, M.D., Professor of Medicine, Endocrinology at the Medical College of Wisconsin. "The majority of patients in the trial had very gratifying improvements in the clinical and metabolic manifestations of hypercortisolism." Korlym is a once daily oral medication. The medicine's FDA-approved labeling instructs physicians to titrate each patient's Korlym dose to clinical efficacy by assessing tolerability and degree of improvement in Cushing's syndrome manifestations. In the first six weeks, these manifestations may include changes in glucose control, anti-diabetic medication requirements, insulin levels and psychiatric symptoms. After two months, assessment may also be based on improvements in cushingoid appearance, acne, hirsutism, striae or decreased body weight, along with further changes in glucose control. "Korlym offers a new treatment strategy that has the potential to improve the lives of many patients," said Joseph K. Belanoff, M.D., the company's Chief Executive Officer. "We have worked hard to make it available as quickly as possible." "We are determined that every patient who could benefit from Korlym will receive it," added Steven Lo, Corcept's Vice President of Commercial Operations. "Our specialty pharmacy partner, Curascript, is prepared to dispense medicine. Our Support Program for Access and Reimbursement for Korlym (SPARK) is ready with case managers to help patients navigate their medical insurance coverage and identify the Corcept and charitable foundation support programs available to them." Physicians and patients seeking more information can visit http://www.korlymspark.com or call 1-855-4Korlym. About Cushing's SyndromeEndogenous Cushing's syndrome is a rare and life-threatening endocrine disorder that results from long-term exposure to excess levels of the hormone cortisol. This excess is caused by tumors that usually occur in the pituitary or adrenal glands that over-produce, or prompt the over-production of, cortisol. Although cortisol at normal levels is essential to health, in excess it causes a variety of problems, including glucose intolerance and diabetes, upper body obesity, a rounded face, stretch marks on the skin, an accumulation of fat on the back, thin and easily bruised skin, muscle weakness, bone weakness, persistent infections, high blood pressure, fatigue, irritability, anxiety, psychosis and depression. Women may have menstrual irregularities and facial hair growth, while men may have decreased fertility or erectile dysfunction. More than 70 percent of Cushing's syndrome patients suffer from glucose intolerance or diabetes. If left untreated, Cushing's syndrome has a five-year mortality rate of 50 percent. About Korlym™ (mifepristone) 300 mg TabletsKorlym is a once-daily oral medication that blocks the glucocorticoid receptor type II (GR-II) to which cortisol normally binds. By blocking this receptor, Korlym inhibits the effects of excess cortisol in Cushing's syndrome patients. The FDA-approved labeling instructs physicians to titrate each patient's Korlym dose to clinical efficacy by assessing tolerability and degree of improvement in Cushing's syndrome manifestations. The FDA has designated Korlym as an Orphan Drug, a special status designed to encourage the development of medicines for rare diseases and conditions. Because Korlym is an Orphan Drug, Corcept will have marketing exclusivity for the FDA-approved indication until February 2019. IMPORTANT SAFETY INFORMATION WARNING: TERMINATION OF PREGNANCY See full prescribing information for complete boxed warning. Mifepristone has potent antiprogestational effects and will result in the termination of pregnancy. Pregnancy must therefore be excluded before the initiation of treatment with Korlym, or if treatment is interrupted for more than 14 days in females of reproductive potential. Contraindications Pregnancy Use of simvastatin or lovastatin and CYP 3A substrates with narrow therapeutic range Concurrent long-term corticosteroid use Women with history of unexplained vaginal bleeding Women with endometrial hyperplasia with atypia or endometrial carcinoma Warnings and Precautions Adrenal insufficiency: Patients should be closely monitored for signs and symptoms of adrenal insufficiency. Hypokalemia: Hypokalemia should be corrected prior to treatment and monitored for during treatment. Vaginal bleeding and endometrial changes: Women may experience endometrial thickening or unexpected vaginal bleeding. Use with caution if patient also has a hemorrhagic disorder or is on anti-coagulant therapy. QT interval prolongation: Avoid use with QT interval-prolonging drugs or in patients with potassium channel variants resulting in a long QT interval. Use of Strong CYP3A Inhibitors: Concomitant use can increase mifepristone plasma levels significantly. Use only when necessary and limit mifepristone dose to 300 mg. Adverse ReactionsMost common adverse reactions in Cushing's syndrome ( ≥ 20%): nausea, fatigue, headache, decreased blood potassium, arthralgia, vomiting, peripheral edema, hypertension, dizziness, decreased appetite, endometrial hypertrophy. To report suspected adverse reactions, contact Corcept Therapeutics at 1-855-844-3270 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Drug Interactions Drugs metabolized by CYP3A: Administer drugs that are metabolized by CYP3A at the lowest dose when used with Korlym. CYP3A inhibitors: Caution should be used when Korlym is used with strong CYP3A inhibitors. Limit mifepristone dose to 300 mg per day when used with strong CYP3A inhibitors. CYP3A inducers: Do not use Korlym with CYP3A inducers. Drugs metabolized by CYP2C8/2C9: Use the lowest dose of CYP2C8/2C9 substrates when used with Korlym. Drugs metabolized by CYP2B6: Use of Korlym should be done with caution with bupropion and efavirenz. Hormonal contraceptives: Do not use with Korlym. Use in Specific Populations Nursing mothers: Discontinue drug or discontinue nursing. Please see the accompanying full Prescribing Information including boxed warning atwww.corcept.com/prescribinginfo.pdf Please see the accompanying Medication Guide at www.corcept.com/medicationguide.pdf About Corcept Therapeutics IncorporatedCorcept is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic and psychiatric disorders. Korlym, a first generation GR-II antagonist, is the company's first FDA-approved medication. The company has a portfolio of new selective GR-II antagonists that block the effects of cortisol but not progesterone. Corcept owns an extensive intellectual property portfolio covering the use of GR-II antagonists, including mifepristone, in the treatment of a wide variety of psychiatric and metabolic disorders. The company also holds composition of matter patents for its selective GR-II antagonists. Statements made in this news release, other than statements of historical fact, are forward-looking statements. Forward-looking statements are subject to a number of known and unknown risks and uncertainties that might cause actual results to differ materially from those expressed or implied by such statements. For example, there can be no assurances that clinical results will be predictive of real-world use, or regarding the pace of Korlym's acceptance by physicians and patients, the reimbursement decisions of government or private insurance payers, the effects of rapid technological change and competition, the protections afforded by Korlym's Orphan Drug Designation or by Corcept's other intellectual property rights, and the cost, pace and success of Corcept's other product development efforts. These and other risks are set forth in the Company's SEC filings, all of which are available from our website (http://www.corcept.com) or from the SEC's website (www.sec.gov). We disclaim any intention or duty to update any forward-looking statement made in this news release. Investor Contact: Charles Robb Chief Financial Officer Corcept Therapeutics Incorporated 650-688-8783 Media Contact: Alyson Kuritz Communications Strategies, Inc. 973-635-6669 Source: Corcept Therapeutics

FDA approves first medication for Cushing's patients

This is excellent news for all my fellow Cushies whose excess cortisol led to type 2 diabetes.  I remain uncured with persistent cushing's disease.
Other medications are expected in 2012, but each has great risks. Hopefully, with this many pharmaceutical companies working at it, something useful will stick. I don't know how long folks like me can wait. It has been five long years already.
-- melissa


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.

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

FDA NEWS RELEASE

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

Consumer Inquiries: 888-INFO-FDA

Korlym: New Medication in Clinical Trials to treat Cushing's

Wouldn't this be interesting if this could really help some people. ~m


Corcept Therapeutics Nears Completion of Enrollment in Phase 3 Cushing's Syndrome Study -- Data Announcement Anticipated by Year End

MENLO PARK, CA, Jun 25, 2010
(MARKETWIRE via COMTEX) --

Corcept Therapeutics Incorporated ("Corcept"), a pharmaceutical company engaged in the discovery and development of drugs for the treatment of severe metabolic and psychiatric disorders, today announced that it had enrolled 49 of the planned 50 patients in its Phase 3 trial of CORLUX(R) for the treatment of Cushing's Syndrome and expects the fiftieth patient to begin dosing next week.

"We are near our target of dosing 50 patients in our Phase 3 study of CORLUX for the treatment of Cushing's Syndrome. We remain on track to announce top line results from the trial by the end of this year," said Joseph Belanoff, M.D., Chief Executive Officer of Corcept.

Cushing's Syndrome Phase 3 Trial Nears Completion of Planned Enrollment of 50 Patients

We have dosed 49 of the planned 50 patients in our open-label Phase 3 trial of CORLUX in patients with endogenous Cushing's Syndrome, which is being conducted at 20 leading medical facilities throughout the United States. The final patient is expected to be dosed next week.

The FDA has indicated that this single 50-patient open-label Phase 3 study of CORLUX may provide a reasonable basis for the submission of a New Drug Application ("NDA") for Cushing's Syndrome. In the study, each patient's dose is titrated to clinical benefit by their study investigator and the primary endpoints (either an improvement in glucose tolerance or blood pressure) are measured at the end of 24 weeks.

We expect to announce the top-line results of this study by the end of 2010 and to submit our NDA to the FDA in the first quarter of 2011.

About Cushing's Syndrome

Endogenous Cushing's Syndrome is caused by prolonged exposure of the body's tissues to high levels of the hormone cortisol due to either cortisol or adrenocorticotropic hormone (ACTH) production by tumors. Cushing's Syndrome is an orphan indication which most commonly affects adults aged 20 to 50. An estimated 10 to 15 of every one million people are newly diagnosed with this syndrome each year, resulting in over 3,000 new patients in the United States. An estimated 20,000 patients in the United States have Cushing's Syndrome. Symptoms vary, but most people have one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Cushing's Syndrome can affect every organ system in the body and can be lethal if not treated effectively.

About CORLUX

Corcept's first-generation compound, CORLUX, also known as mifepristone, directly blocks the cortisol (GR-II) receptor and the progesterone (PR) receptor. Intellectual property protection is in place to protect important methods of use for CORLUX. Corcept retains worldwide rights to its intellectual property related to CORLUX.

About Corcept Therapeutics Incorporated

Corcept is a pharmaceutical company engaged in the discovery and development of drugs for the treatment of severe metabolic and psychiatric disorders. The company has two ongoing Phase 3 programs: CORLUX for the treatment of Cushing's Syndrome, and CORLUX for the treatment of the psychotic features of psychotic depression. Corcept also has a Phase 1 program for CORT 108297, a selective cortisol receptor antagonist. Corcept has developed an extensive intellectual property portfolio that covers the use of GR-II antagonists in the treatment of a wide variety of psychiatric and metabolic disorders, including the prevention of weight gain caused by the use of antipsychotic medication, as well as composition of matter patents for our selective GR-II antagonists.

CONTACT:
Caroline Loewy
Chief Financial Officer
Corcept Therapeutics
650-688-8783

SOURCE: Corcept Therapeutics

Original Marketwire article