Cushing's Patient Assistance Program

WASHINGTON, April 6, 2012

In partnership with Corcept Therapeutics Inc., the National Organization for Rare Disorders (NORD) has established a Cushing's Syndrome Patient Assistance Program for patients with endogenous Cushing's syndrome who need financial assistance with certain costs related to their medical diagnosis and/or care.

Endogenous Cushing's syndrome is a serious and debilitating rare disorder caused by over-production of cortisol, a steroid hormone that increases blood sugar levels, by the adrenal glands.  The syndrome most commonly affects adults between the ages of 25 and 40.

"It is especially appropriate that we are making this announcement just before Cushing's Awareness Day, which will be observed on Sunday," noted NORD President and CEO Peter L. Saltonstall.  "The Cushing's Support and Research Foundation (CSRF), a non-profit organization for patients and families affected by Cushing's syndrome, is a member of NORD.  We are pleased to join CSRF and the entire Cushing's community in promoting awareness of Cushing's syndrome at this time." 

The new program, which launches on April 11, will provide assistance with deductible and/or co-pay expenses to ensure patient access to Korlym™, a drug developed by Corcept and approved by the Food and Drug Administration earlier this year.  

The program will also provide financial assistance to eligible uninsured and underinsured patients for reasonable expenses required for diagnosis, direct treatment, and care that are not covered by insurance.  These would include incidental medical expenses such as lab and diagnostic tests, as well as related expenses such as travel for a medical consultation.

Patients seeking information about the program, or to apply, may contact NORD through a toll free phone number (800 999-6673 Ext. 326) or by email (cushings@rarediseases.org).

Prior to FDA's approval of Korlym™ on Feb. 17, 2012, there were no approved medical therapies for the treatment of endogenous Cushing's syndrome.  Korlym™ is 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 have not responded to prior surgery.

NORD is a nonprofit organization, established in 1983, that represents the 30 million Americans with rare diseases.  In addition to patient assistance programs, NORD provides programs of advocacy, education, awareness, and research.

SOURCE National Organization for Rare Disorder (NORD)

http://www.sacbee.com/2012/04/06/4396105/nord-announces-new-cushings-syndrome.html

Day 9 of Cushing's Awareness Challenge: What to Expect

I have been really struggling mentally and physically with this blogging challenge. The stress has been overwhelming. I just can't manage to pull it all together.
Today, I had help to clean my house (hi Lil), and I cleaned for a portion of the day, too. We started at 8 am. After two hours, I took pain meds. After 6 hours, I was relegated to the couch. By 5 pm, I made a walk-in appointment with my chiropractor. My back had seized up, pulling at my neck and shoulders, with arthritis pain in my ankles and wrists. Yes, today, I had done too much. I fell asleep in bed by 8:30 pm.
So yet again, tonight I write this brief blog on my iPhone in my bed, wishing I had planned ahead for these blog entries yet thankful for the relief of lying instead of sitting.

Day 8: Happy Birthday Harvey

"Harvey Cushing, M.D."

Dr. Harvey Cushing is known as the "father of neurosurgery," given credit for laying the foundation upon which the whole field of brain surgery is built. At the time he began to concentrate on improving the results of brain surgery in the early 20th century, such surgery was nearly 100 percent fatal. As a result of removing a tumor from Gen. Leonard Wood, his study of the pituitary gland led to identification of what is known as Cushing's syndrome. Cushing's Law states that an increase in intracranial tension causes an increase in blood pressure. The blood pressure remains higher, however, than the intracranial pressure, thus preventing the brain from being shut off from its blood supply. His work by 1932 led to a reduction of the mortality rate in brain surgery to 10 percent.

Click to buy a stamp of our favorite neurosurgeon.

Day 7: Out & Living a Little

I have shared as many things as I could think to here on this blog.  However, I believe I forgot one of the most important lessons of all.

If you feel Cushing's grant you a reprieve, go out and live your life.  

You may have a few good hours. 

You may be lucky to get a few good days.

Whatever it is, don't waste it.

Be sure to grab it with both fists, and don't turn loose of it.

~ * ~ * ~ * ~ * ~ * ~ * ~ * ~ * ~ * 

To that end, I plan to spend the rest of easter weekend out with my daughter / favorite miracle bunny.

Happy Easter, y'all.

Day 6: A Good Friday

Today, I took my daughter to her first Easter egg hunt. She is 3.5 years old.

Wait. What?  Is that right? How can this be?

Year 1: no walking, no hunt 

Year 2: hunted eggs in a park, Mama supplied eggs, thus no competition
Year 3: surgery at cancer center to remove pesky tumor from Mama's pituitary for the second time.

Oh yes yes yes. Now I remember. OK. OK. So that's how we got here. That's how my daughter is 3.5 years old and never engaged in a proper Easter egg hunt.

In 2011, I returned home from my four-day hospital stay on Saturday. Easter was the next day. I had not made that Right-Turn-Clyde onto Recovery Road yet. I had been so sick in the hospital with normal post-op cortisol-finally-dropping misery coupled with diabetes insipidus. To survive, I was adamant about wanting the room silent and dark. The slightest sound overwhelmed my body. A beam of light was too intrusive for me to process. When I returned home, I tried to protect myself by replicating that environment, except with hopes of real control over ingress and egress. Thankfully, no one obsessed over my vitals at home. With no sound, no light, I found sleep, but then, the tinnitus set in. Don't know what that nifty symptom is?  Be thankful. It's the omnipresent ringing in the ears. Nearly one year later, I still hear it. I hear it right now as I type in my new state of silence. Grasshopper-rubbing-legs-together silence. Great. 

What's that? An Easter egg hunt? Oh yes. 

So, I tried to plan ahead and all (always the best intentions without all that feel-good execution).  I had purchased the Easter egg decorating stuff before I went into the hospital. After the surgery, I just didn't feel up to it. My daughter stayed with my parents while I was in the hospital, and she remained with them that Saturday night so I could sleep.

I remember my mama calling me Sunday morning about Easter plans at my sister's.

No, I don't think I can go. 

Say what?

You want to take my child over there and let her hunt eggs with her young cousin, without me to see her or share in her joy? 

No. Yes, sure, you can take her. 

She will have fun. 

No, no, I don't want her to miss Easter just because of me. 

No, I'm sure. 

I'll be fine. 

Just bring her home to me after all y'all finish up.

OK. Bye.

I buried my head into my husband's chest as I sobbed.

Looking for reassurance I knew no one could give, I asked, Next year will be different, right?  All my lovey could do is nod.

* ~ * ~ * ~ * ~ * ~ * ~ * ~ * ~ * ~ * ~ * ~ * ~

I took my daughter to her first Easter Egg hunt today.  She had so much fun with the other kids, finding treats inside the eggs, and really marveling in the glory of it all.  I feel pretty confident in saying that it made both of us very happy. I thank all the new friends I've made in California for hosting such a fun Easter celebration. It means more to me than I could ever tell you.  I can only hope my little one will always be as delighted and delightful as she was today.

Today is a good Friday because I didn't think about Cushing's too much. That's really a good Anyday. I'll take this any day of the week and any day of the year. 

So go ahead. Ask someone with Cushing's how this disease has altered the trajectory of their lives and those of their family. I bet they will answer with a thousand stories of unfulfilled promises. What I know for sure is that as long as I am alive, I will try to make up for all the times I couldn't do like I had planned or promised. I just hope this body lets me.

Day 5: Today is Your Day

Last summer, still recovering from my April 20, 2011 surgery, I start watching the Why Not with Shania Twain series on the Oprah Winfrey Network.

As a Texan and in-my-blood country music lover, I had always enjoyed Shania's music.  Seriously, who wasn't glued to their television sets when they first saw her videos "Dance with the One that Brought You" and "Whose Bed Have Your Boots Been Under?"

I admit to only knowing a little about her personal life (I blame studying in college). I knew that her parents died when she was just out of high school, and she took charge singing to earn a living to support her younger orphaned siblings.  She had it rough, worked very hard and succeeded. That always inspired me.
Plus, she wore mid drift shirts in her videos and that shocked Nashville. Apparently, that had never been done in country music. That always made me laugh.

Well, the Why Not with Shania Twain followed Shania's breakdown and recovery after finding out her husband/ producer was having an affair with her best friend.  She, understandably, was devastated.  She lost her voice and had to find the courage to be herself again.

Here is a clip about the show in her own words:

Why Not? with Shania Twain is the intimate portrait of country/pop music sensation Shania Twain on her emotional journey from heartbreak back to the top. This highly anticipated OWN original series is filled with revelation and unexpected adventure. Follow Shania as she heals, shares and rediscovers her voice.

During the show, Shania visited with others who had experienced immense loss. She listened and they talked about grief and grieving for the life you once had but lost.  Throughout the trip, Shania worked through the melodies and lyrics on her song, Today is Your Day. The song truly unfolds in front of our eyes.

Initially, a listener may only dismiss this song as one dripping in positivity.  To a viewer of the show, it means much more. It was a Song of Self-Soothing. She was singing it until it stuck, singing it until her words rang true for her life, singing it until that song's shoe fit.  I understood it instantly, and it quickly became a favorite of mine.

As I putter around in the waiting room or the patient room at a doctor's visit, I somehow manage to think of her.  I open up iTunes, and I play this song. It soothes me instantly. Humming it in my mind while dealing with yet another medical professional is just the right medicine.  It keeps me calm and focused, alert and content.  It is the perfect mix of "This-totally-applies-to-me-as-a-sick-person" and "See-everyone-struggles-with-something" that makes me feel like I can fight my way out, too... AND WIN.  After all this time struggling to do it other ways, I am happy to have found peace some where. While she wrote it to rebuild her courage to sing, Shania's song is a gift to me.

I plan to see Shania as she opens a long engagement singing at Caesars Palace, starting December 2012. I really look forward to being well enough to do that.

Thank you, Shania Twain. I wish you all the happiness.

xxoo Melissa

P.S. You can download the song Today is your Day on iTunes here.

Today is your Day
You got what it takes you can win…You got what it takes you can winToday is your day to begin.Don't give up here, don't you quit.The moment is now, this is itKnow that you can then you willGet to the top of the hillPart of the fun is the climbYou just gotta make up your mind
That today is your dayAnd nothing can stand in your wayToday is your dayEverything's goin' your wayToday (you can do it)Today (c'mon c'mon)Today (c'mon do it)Today
When somebody throws sticks and stonesAll they can break are your bonesAnd life's gonna kick you aroundThen kick you again when you're down
But today is your dayAnd nothing can stand in your wayToday is your dayEverything's goin' your wayToday (c'mon do it now)TodayTodayToday
mmmm life's gonna kick you, its gonna kick you around, its gonna kick you down
Brush yourself off no regretsThis is as good as it getsDon't expect more or lessJust go out and give it your best (give it your best)
Today is your dayAnd nothing can stand in your wayToday is your dayEverything's goin' your way.

Day 4: Not Tonight, I Have a Headache

 

When I was wondering if I could blog every day, I worried about days like this.  

"Mama told me there'd be days like this."

And this blog post almost didn't happen.

-@-@-@-@-@-@-

I woke up today with my back and neck out of whack.  I called a new chiropractor and asked if I could come right over. Points for her, she said yes.

Nice chiropractor asked, "So what brings you in today?"

I replied, "My back -- upper and lower -- are locked up. I have a headache. It feels like a sword is stabbing me, from this fleshy left side of my neck to this lower left quadrant of the back of my skull. Then, it feels like the tip is coming out of this northwest quadrant up here at the front ... and then the sword u-turns to finish the job and stabs me straight down into my left shoulder.  It bypassed my heart today out of pity." 

I looked up after stretching and distorting my body for effect. Greeted by blank stare.

Undeterred, she ventured on, detailing her treatment protocols and what might help. She was able to make spinal adjustments to unlock things to get my back moving again -- goodbye slab of concrete -- but my headache has remained. My body is aching.

I did try to find a fun headache photo, but 99% were of people holding the foreheads or temples in pain. Nope, not accurate for me. My pain shoots upwards along the neck muscles into the occipital bone, which forms the base of the skull. Mine feels like there is so much upwards pressure on this bone that I want to pull my own head off and throw it across the room. 

So this is me with Cushing's, swords and all.

-@-@-@-@-@-@-

The root of my headaches seem to be muscle contractions.

See where those lines come together behind his ear? That's the sword's entry point.

This is how I feel. Wish I could pull it together for a better blog post, but hey folks, this is Cushing's.  Have fun with it: Say it like James Earl Jones says "This is CNN."

Day 3 of Cushing's Awareness Challenge: Cushing's Patient Groups Get Fundraising Boost

I'm happy to report today's news from Corcept Therapeutics, the manufacturer of Korlym. This medication is the first FDA approved medication for the treatment of persistent Cushing's in the United States.

Corcept has shown great respect for the Cushing's community, including patient groups, bloggers, and yes, even Cushing's with Moxie.  They are responsive to ideas and feedback.  I am so happy to have a caring company on our side.  I was ecstatic to get the phone call about their project for Cushing's Awareness Day.

Support Cushing's ...with a Click.

Corcept Therapeutics will donate $5 for every person who clicks to support people with Cushing's. Corcept has jump-started the effort to build awareness and support with a $15,000 contribution to provide support through the Cushing's Support and Research Foundation and Cushing's Help. With your support, we can aim even higher!* Take part...and spread the word.

When:    now through April 20, 2012 

What:     Corcept Therapeutics will donate $5 for every click

Where:   http://cushingsawareness.korlym.com/  

For:       two Cushing's patient advocacy groups, Cushings-Help.com and Cushing’s Support and Research Foundation, will share all money raised. Corcept has promised a minimum of $15,000. 

Please be sure to share the link, and ask your friends and family to click, too.  

* ~ * ~ * ~ * ~ * ~ * ~ *

For more information on Korlym, visit these websites to see full safety and prescribing information, including boxed warning.

DISCLAIMER:  I participated in Corcept's market research study in February 2012. Corcept Therapeutics paid for my travel expenses. I flew to Philadelphia, where I gave feedback on marketing material and told my story on camera.  For that, I was received monetary compensation of $250. In addition, I am a Corcept stockholder. 

Day 2: Getting My Mind Right

Fighting a disease like Cushing's is not as simple as keeping a positive attitude. Cushing's attacks your spirits and leaves you riddled with depression, anxiety, and suicidal thoughts. In short, it makes seeing yourself as your real self nearly impossible.  

If you were to ask a Cushie which changes were more devastating from Cushing's: the physical, psychological, or mental, I bet each would come up with a different answer with a very good reason. For me, I believe it depends on the day. While my body is clearly nothing like it was, it is the lost mental acuity and psychological disturbances that really make feel not very much like myself.

I always wonder what my old friends would say if I told them I was depressed, anxious, agoraphobic, overwhelmed, and fearful daily, and since I'm telling the truth, suicidal at least every few months. When I see that, when I address those realities as my realities, it is too overwhelming to consider. For, like my friends, they are such drastic departures from the person I once was.  The difference is, most of my old friends don't see me except through facebook, and I am imprisoned in this altered reality every day.

Endocrinologists argue about many things related to Cushing's. One point upon which all can agree is that "Major depression is a common, life-threatening complication of Cushing's:"

"Patients with Cushing's disease and depression appeared to suffer from a more severe form of illness."

* ~ * ~ * ~ * ~ * ~ * ~

So with all that said, what do I do if I know that this is happening to me?  Is there anything to do? 

All I can do is just do my best, and model the part of my outlook I can control after this sweet woman. 

Courtesy of Intelligent Redneck's facebook page.

Day 1: Cushing's on my Mind

There is no real way to explain how devastating Cushing's is to a patient unlucky enough to suffer from high cortisol. In nearly four years of blogging, I find that this word cloud I made does the best job so far. 

Click on the image to enlarge it.

Yes, it is really this bad, and isn't that so sad for me and other Cushies.

  

I can’t believe I have lived in that cloud for over 60 months now. To be a patient suffering with Cushing’s is to feel and experience all that word swirl of emotion and more. That word cloud isn’t just my disease. Cushing's is the monkey on my back that I cannot shake. Hours and days, then months and years pass without feeling better, looking better, or being better.  When the days carrying moments of relief arrive for a short surprise visit, I am pleased. Looking back, I realize the strength I have in just walking through it, never seeing any light at the end of the tunnel.  May I and the many other Cushing’s patients continue to find the strength to keep going.

Cushing's has altered who I have become, but I can't let it change who I can become anymore.

236,892 Page Views & Counting

April is my favorite month of all. I think about it all year!

It's Cushing's Awareness Month.  On April 8th, we honor Dr Harvey Cushing's, the father of modern neurosurgery and the namesake to this terrible disease I have. We don't blame you, Harvey. We like you!

I wanted to let you in on a project I've got going. In appreciation of where I am in my journey and all the close friends I've made along the way, I plan to blog every day through the month of April. Maybe, just maybe, a few patients suspecting Cushing's will see enough information to reach out and stand up to the MDs that are blocking their way to treatment, cures, and ultimately, reclaiming their lives.

Now, back to the writing and daily blogging for a sec. For someone so scatterbrained, exhausted, and unreliable as someone with Cushing's, even agreeing to a challenge strikes fear in me. Eek. That's a lot of commitment, and Cushing's makes us so flaky, I really hate that part. All I can do is try, right?!

This blog has been an important part of my life since its inception in May 2008. It was a special place that I created where I could talk about Cushing's in any way I wanted, any time I wanted. For the past three years, I have shared as much and as often as I could of myself, my experiences, and tidbits of knowledge I accumulate along the way. Bringing more awareness to others is critical to how I have made sense of and come to accept this haunting disease.

So for me, this challenge is very personal.

As a pre-disease perfectionist, I always wish I could do more here.  I wish I could blog every day. I have so much to say Cushing's and how it changes the trajectory of my life each day.  However, I struggle to maintain the energy for myself and my family. For this one month of April, I really want to work to balance my focus on Cushing's and my focus forgetting Cushing's enough so I can be present with my family.  I hope I can hold it all together. I can only do my best.

----@----@----@----@----@----@----@----@----@

I always wish this body and mind would let me do more or not hold me back.  I feel so trapped in this body.  It is also so important to my self-identity to combat this reality of me being stuck inside the house all day.  I realize I can be sick with Cushing's and still contribute something to this world. With this blog and these numbers, I sorta kinda maybe see the impact I have already made in my little corner of the Cushing's world.  

I think of all the Cushing's patients -- fearful, lost, defeated -- that have come to me by way of this blog, and I am thankful that something I have done could help another kind soul in this world. I always wish I could spend more time with each person who writes me--four people last week!--but I am without a doubt always rooting for them. That's what I see in this counter, and it always makes me smile. 

Seeing that graph below makes me feel pretty, pretty, pretty, pretty, pretty certain that there are many more undiagnosed Cushies in this world than any doctor would ever care to admit.  That’s fine by me.  While I can’t control the doctor side, I can do my part to educate and advocate for the patient side.

Hope you stick around in April for a fun month, as we learn more about facing Cushing’s with Moxie.

Bye for now.  Melissa

Korlym Release Ahead of Schedule

A birdie in the know told me that the big release of Korlym is ahead of schedule.  Initially set to be released some time in May 2012, it now seems that Korlym is set for April 11, 2012. 

Korlym is the first FDA drug approved for Cushing's patients who have undergone unsuccessful surgery (-ies). It is expected to help patients just like me.

Rest in Peace, Liz. Cushing's Steals Another

It is with great sadness that I report that Death took another friend with Cushing's today.

Liz R was only 46 years old and leaves behind a 16 year old daughter in London, England. Liz was very active on the www.cushings-help.com message boards during my tenure there.

This marks the 3rd Cushie sister lost in 8 months. Who will be next? Me? Another of my friends? When will we get the help we deserve? When will doctors see that disease is destroying our bodies day after day?! What a devastatingly sad day, folks. 

Rest in peace, dear Liz. May your body, soul, and spirit fly high now without the burden of Cushing's.

 -m

Cushing's Help blogtalkradio program

Thank you, as always, to MaryO and RobinS for making this chat with Dr Friedman, the wonderfully knowledgeable and compassionate diagnostician, possible. This is truly for the betterment of all those suffering with Cushing's as well as any friends or family who care to take a listen. 

****

Have questions about the new Korlym? How about Korlym vs ketoconazole? About medical vs surgical treatment for Cushing's.   Ask Dr. Theodore Friedman.

Listen live at http://www.blogtalkr...es-for-cushings
Call in to ask your question at (646) 200-0162
This interview will be archived afterwards at the same link and on iTunes Cushie Podcasts.

Theodore C. Friedman, M.D., Ph.D. has opened a private practice, specializing in treating patients with adrenal, pituitary, thyroid and fatigue disorders. Dr. Friedman has privileges at Cedars-Sinai Medical Center and Martin Luther King Medical Center. His practice includes detecting and treating hormone imbalances, including hormone replacement therapy. Dr. Friedman is also an expert in diagnosing and treating pituitary disorders, including Cushing's disease and syndrome.

Dr. Friedman's career reflects his ongoing quest to better understand and treat endocrine problems. With both medical and research doctoral degrees, he has conducted studies and cared for patients at some of the country's most prestigious institutions, including the University of Michigan, the National Institutes of Health, Cedars-Sinai Medical Center, and UCLA's Charles Drew University of Medicine and Science.

*****

Catch up on all the Cushing's information Dr Friedman has shared with this community through the years. 

Read Dr. Friedman's First Guest Chat, November 11, 2003. 
Read Dr. Friedman's Second Guest Chat, March 2, 2004. 

Listen to Dr. Friedman First Live Voice Interview, January 29, 2009. 
Listen to Dr. Friedman Second Live Voice Interview, March 12, 2009. 
Listen to Dr. Friedman Third Live Voice Interview, February 13, 2011.

Rare Disease Day: I have Cushing's

Today, I am thankful for the friends I have met along the way. Cushing's has devastated our lives in unimaginable ways, but it has brought courageously delightful people into my friendsphere.  

I thank Trisha for her efforts to highlight the difficulty in diagnosing this rare disease.  Please consider taking just a few moments to watch this youtube video she put together for our whole community.

Cushing's Disease: A Numbers Game

CORT Does Well in After Hours Trading

I first saw the word Cushing's almost five years ago. Even at that time, there was talk in the Cushing's community of a far-away land where magical medicine brought cures when surgeries couldn't. We have waited a long time for this.

Many of us patients with Cushing's have been closely following Corcept's march towards FDA approval for the drug (Korlym, was Corlux).  It seems investors took the time they needed to research Corcept a little more over the long weekend, because folks are buying the stock and driving the price up. 

Could the market surge have been from all of us Cushies?  Maybe a portion, but not too much.  We spend all our money on medical bills!
  

I wanted to share the article Corcept Has Upside Following Drug Approval. I posted a comment under the article,  and I hope that it will be accepted and posted (hi Adam!!).  This is what I wrote:

I have persistent Cushing's disease, and I will be eligible to take Korlym. I am also a stockholder. Wanna know more about the devastating effects of Cushing's? Read more about it on my blog, http://cushingsmoxie.blogspot.com/.  Besides an investment, this knowledge may save the life of someone you know.

*****

Drugs

 Return to Article

Corcept Has Upside Following Drug Approval

Adam Feuerstein

02/21/12 - 12:54 PM EST

A foul serving of crow is what I deserve for not believing Corcept would get Korlym approved on its first attempt. More on what I got wrong later; first, let's look at Corcept's future with Korlym approved.
Corcept plans to launch Korlym on May 1. The company has yet to disclose pricing but Alan Leong of Biotech Stock Research estimates conservatively one year of therapy will cost $75,000. [Leong deserves kudos for correctly predicting Korlym's approval.]
Again being conservative, Leong estimates that about 2,000 Cushing's patients in the U.S. will be eligible for Korlym therapy, leading to $150 million in U.S. sales by 2016. Corcept is pursuing Korlym approval in Europe, which could ultimately generate another $75 million in sales or $225 million total.
Corcept intends to market Korlym on its own in the U.S. and will only need to hire a small sales force since most Cushing's patients are treated by a handful of specialists. This means Corcept should be able to maintain high profit margins.
The FDA stated that as many as 5,000 patients may be eligible for Korlym treatment, which implies a peak U.S. market opportunity of $375 million (assuming $75,000-per-year pricing.)
Corcept's current market cap of $370 million is not fully valuing Korlym's market potential. Why? Likely because some early investors in the company are taking profits now while others may wait to see how Korlym launches before jumping in.
Corcept is also studying Korlym as a treatment for psychotic depression and weight gain caused by antipyschotics.
Cushing's syndrome is a rare condition in which a tumor on an adrenal gland like the pituitary causes excess production of the hormone ACTH. Excess ACTH, in turn, causes the body to produce too much of the stress hormone cortisol. Patients with Cushing's suffer from severe cardiovascular and metabolic disease that can be fatal.
FDA approved Korlym to control high blood sugar levels in adults with Cushing's. The drug works by preventing cortisol from binding to its receptor thereby blunting cortisol's effects on the body. Korlym does not decrease levels of cortisol in the body.
Corcept also sought approval for Korlym to treat hypertension in Cushing's patients but FDA rejected that claim.
I previously predicted an FDA rejection for Korlym -- a call that obviously went wrong. My biggest mistake was probably under-estimating the significance of Cushing's being a rare, life-threatening disease with no current treatment options. Korlym causes significant side effects but the drug is also well understood by FDA (the active ingredient is mifepristone also known as the "abortion pill" RU-486) and the benefits in this vulnerable Cushing's patient population outweigh the risks. I didn't think through the risk-benefit equation for Korlym well enough.
I also placed too much emphasis on a change in Corcept's risk factors (disclosed in the company's SEC filings) that warned of FDA possibly requiring additional pre-approval requirements for Korlym in Cushing's. In the past, changes like this made to a company's SEC filings have proven to be reliable indicators of trouble, but not this time.
--Written by Adam Feuerstein in Boston.
>To contact the writer of this article, click here: Adam Feuerstein.