PATIENT BLOG: Plenadren for Addisons

Dear Unnamed Author,

Thank you for creating this Plenadren for Addisons blog to share your experience with Plenadren. You have a new reader from Cushing's, the other side of cortisol. Your efforts to document your experience is very much appreciated.

Signed, Moxie

New Drug for Adrenal Insufficiency to Hit the Market Soon.

With each article I post, I am becoming more excited. Details details details! I love details!

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ViroPharma Announces the First EU Market to Launch Plenadren® (Hydrocortisone, Modified-Release Tablet) for Treatment of Adrenal Insufficiency, the First New Treatment Innovation for Over 50 Years

Share on facebookShare on emailShare on favoritescheckorphan.org's article here:More Sharing ServiceFriday, September 21, 2012

BRUSSELS. - ViroPharma Incorporated (Nasdaq: VPHM) today announced the launch of Plenadren® (hydrocortisone modified-release tablet) in Denmark, the first country in the EU to provide a new treatment option for adrenal insufficiency (AI) in over 50 years. Plenadren is a novel, oral, once daily hydrocortisone, modified-release tablet that is designed to better mimic the body's normal, cortisol profile, compared with standard treatment. The results of a pivotal Phase II/III trial showed Plenadren to be effective and well tolerated, compared to standard treatment, demonstrating a delivery of cortisol, which is more in line with the body's own cortisol profile, thus avoiding the afternoon/evening peaks. These peaks are thought to be associated with an increased risk of morbidity and premature mortality.

"There is a clear need in the treatment of AI for an oral hydrocortisone replacement therapy that better resembles the body's natural cortisol production, that can be given once a day and that may improve patients' metabolic status and compliance to treatment," said Dr Anna G Nilsson, Principal Investigator for all Plenadren studies and Consultant in Endocrinology at the Sahlgrenska University Hospital, Goteborg, Sweden. "A new therapeutic option for the first time in 50 years, that appears to achieve this is exciting news for AI patients. The once-daily dosing schedule will help them to manage their condition in a more simple and effective way."

AI is a rare, chronic and potentially fatal endocrine disorder characterised by a reduction or failure in the production of the hormone cortisol. AI is an orphan disease that affects less than 4.5 in 10,000 people in Europe. Standard cortisol replacement treatments aim to restore the body's natural, daily production of cortisol over 24 hours. Standard, immediate release, hydrocortisone replacement therapy, however, cannot mimic the body's natural production of cortisol, despite multiple dosing throughout the day. AI patients using standard treatment are two or three times more likely to die compared to people without AI.

Plenadren is taken orally as a single dose in the morning and has been designed to provide a dual release pattern of hydrocortisone. The outer coating provides a high concentration of cortisol in the morning, meeting the body's requirements. The extended-release core provides a smooth cortisol release throughout the day, followed by a cortisol-free interval during the night, avoiding the unphysiological afternoon/evening peaks observed with standard treatment. Furthermore, Plenadren is proven to be well tolerated with patients reporting favourable impact on quality of life compared to standard treatment, in terms of psychosocial and cognitive functioning, and psychological general well-being. In an 18-month long-term follow-up trial, 98.5% of patients rated tolerability as 'very well', 'well' or 'acceptable'.

"For people living with a cortisol deficiency, conventional treatment can have an impact on quality of life, with many patients struggling with peaks and troughs in their replacements as well as managing complicated regimes," said Pat McBride, Patient Support Manager of The Pituitary Foundation, UK. "The availability of Plenadren is an important step forward for patients and may help to improve the management of their condition, helping patients to more actively take part in their work, family and social commitments."

In November 2011, the European Commission (EC) granted European Marketing Authorisation for Plenadren, an orphan drug for treatment of adrenal insufficiency in adults. Plenadren is not approved in the United States; however, it has received orphan drug designation status in the United States and has maintained orphan status in Europe.

"We are very pleased to be able to launch Plenadren in Denmark," said Arun Mistry, Senior Director, Medical Affairs, Europe "At ViroPharma, we are committed to delivering important solutions that address critical gaps in care for patients living with few, if any, clinical treatment options. We are excited to be working closely with physicians to help and support patients through the management of adrenal insufficiency."

About Plenadren ® (hydrocortisone, modified-release tablet)

Plenadren, is a novel, once daily hydrocortisone, modified-release tablet, designed to better mimic the body's natural cortisol production compared to standard treatment. The Plenadren tablet is available in 5 and 20 mg strengths. The immediate release outer coating provides physiological cortisol concentrations within 20 minutes of intake; the extended release core provides a smooth serum cortisol level with reduced exposure in the late afternoon and over the 24 hour period. In addition there is no dose accumulation.

Plenadren is proven in a pivotal Phase II/III trial to be effective and well tolerated compared to standard therapy, demonstrating a delivery of cortisol, which is more in line with the body's own cortisol profile thus avoiding the unphysiological cortisol peaks seen with standard glucocorticoid therapy. These peaks are thought to be associated with an increased risk of morbidity and premature mortality. Sustained and consistent results across all patients have been seen for up to 27 months.

Plenadren's once-daily dose simplifies treatment management, with patients reporting favourable impact on quality of life compared to standard treatment in terms of psychosocial and cognitive functioning, and psychological general well-being. In a 12-week trial, 85% of patients were found to prefer once-daily Plenadren to standard treatment. Furthermore, in an 18-month, long-term follow-up trial, 98.5% of patients rated tolerability as 'very well, 'well' or 'acceptable'.

The safety profile was similar to standard hydrocortisone tablets given three times daily. The most common adverse event reported in clinical trials associated with Plenadren was fatigue. There were also some common adverse reactions in the form of gastroenteritis, upper respiratory tract infection, viral infection, sedation, vertigo, dry eye, oesophagitis, nausea, upper abdominal pain, tooth erosion, pruritic rash, Joint swelling, HDL decrease and weight increase. There were also some adverse reactions observed in the eight week period after first changing from standard hydrocortisone tablets three times daily to Plenadren. These adverse reactions were abdominal pain, diarrhoea, nausea and fatigue and were mild or moderate, transient, of short duration.

About Adrenal Insufficiency

Adrenal insufficiency (AI) is a rare, chronic and potentially fatal endocrine disorder characterised by a reduction or failure in the production of the hormone cortisol. AI affects less than 4.5 in 10,000 people in Europe.

AI can lead to serious, life-threatening conditions such as cardiovascular, malignant or infectious diseases, as well as disorders which impact on health and quality of life. The many symptoms of AI include fatigue, anorexia, weight-loss, fever, muscle weakness, abdominal pains, dizziness and headaches. Because these symptoms can be attributed to other disorders, diagnosis can be difficult and delayed, leading to unnecessary morbidity and mortality. To survive, AI patients need replacement therapy with glucocorticoids (usually hydrocortisone) and because it is a chronic condition, they require this life-saving therapy throughout their lives.

There are two main types of AI:

1. Primary (Addison's disease) AI occurs when there is gradual destruction of the adrenal cortex usually by the body's own immune system and occurs when at least 90 per cent of the adrenal cortex has been destroyed.
2. Secondary AI occurs when the pituitary gland fails to produce enough adrenocorticotropin (ACTH), a hormone that stimulates the adrenal glands to produce cortisol. Often, the cause is damage to the pituitary gland following a pituitary tumour or surgery. Secondary adrenal insufficiency is more common than primary. NOTE: THIS IS US, cyclical Cushies, post op Cushies, and those with bilateraladrenalectomies.

About ViroPharma Incorporated

ViroPharma Incorporated is an international biopharmaceutical company committed to developing and commercialising novel solutions for physician specialists to address unmet medical needs of patients living with diseases that have few, if any, clinical therapeutic options, including C1 esterase inhibitor deficiency, treatment of seizures in children and adolescents, adrenal insufficiency, and C. difficile infection (CDI). Our goal is to provide rewarding careers to employees, to create new standards of care in the way serious diseases are treated, and to build international partnerships with the patients, advocates, and healthcare professionals we serve.

ViroPharma routinely posts information, including press releases, which may be important to investors in the investor relations and media sections of our company's website, http://www.viropharma.com/. The company encourages investors to consult these sections for more information on ViroPharma and our business.

Denmark first to launch Plenadren

Crossing fingers that its new American company will deliver Plenadren to the USA soon. 

Visit Goteborg Bio page in Sweden for more detailas on the new Plenadren drug to treat adrenal insufficiency.

Denmark first to launch Plenadren

Published: 2012-09-25 03:37

Denmark first to launch Plenadren

Duocort sold its drug candidate Plenadren to American Viropharma last year for approximately SEK 1 billion and the medicine will now enter its first market. Denmark will be the first country to sell Plenadren and will also constitute the first nation to supply a drug against Addison's disease in over 50 years.

Plenadren was developed by the virtual company Duocort with headquarters in West Sweden. The drug is based on hydrocortisone and has been developed for the treatment of adrenal insufficiency, also referred to as Addison's disease.

Plenadren has orphan drug status in the EU, Switzerland and the USA and was granted market authorisation by the European Medicines Agency at the start of November last year.

http://www.goteborgbio.se/nyhetsarkiv/nyhetsarkiv2012/denmarkfirsttolaunchplenadren.5.30d0092e1399c72e2e1237a.html

Wow! New Drug for Adrenal Insufficiency

Plenadren. Plenadren. I hope this word becomes so familiar to me that it easily rolls off my tongue.

Plenadren is the new, once-a-day, hydrocortisone modified-release tablets that researchers in Denmark believe will work better for patients who face adrenal insufficiency. The patients fight Addison's, cyclical Cushing's, as well as Cushing's post-op. 

This is very big news.

http://cushingsmoxie.blogspot.com

• ViroPharma launches Plenadren for rare endocrine disorder

• September 21, 2012
 
• | Kevin Grogan, Pharma Times

ViroPharma has begun the roll-out in Europe of Plenadren, which it says is "the first true innovation in over 50 years in the treatment of adrenal insufficiency".

The firm has launched Plenadren (hydrocortisone modified-release tablets) in Denmark, after it was approved by the European Commission in November 2011. The once-daily pill is designed to better mimic the body's normal, cortisol profile, compared with standard glucocorticoid treatment.

Plenadren proved to be effective and well-tolerated in a Phase II/III trial compared to standard therapy, also avoiding the unphysiological cortisol peaks seen with the latter. ViroPharma noted that these peaks are thought to be associated with an increased risk of morbidity and premature mortality.

Anna Nilsson of the Sahlgrenska University Hospital in Gothenburg, Sweden and principal investigator for all Plenadren studies, said "there is a clear need in the treatment of AI for an oral hydrocortisone replacement therapy that better resembles the body's natural cortisol production, that can be given once a day and that may improve patients' metabolic status and compliance to treatment". She added that "a new therapeutic option for the first time in 50 years, that appears to achieve this is exciting news".

AI is a rare, chronic and potentially fatal endocrine disorder that affects less than 4.5 in 10,000 people in Europe.

Click here for PharmaTimes article.

Fiddling with Nature Causes Tumors

Genetically-engineered food:  another environmental cause of pituitary tumors.And doctors still have the nerve to call us fat, tell us to exercise, and dismiss our symptoms systematically?!

GE Foods Shorten Life, Cause Tumors, Says First-Ever Long-Term Study

"The researchers found that rats fed GE corn and those whose water contained environmentally relevant levels of RoundUp faced the following:

·  Two to three times more large tumors than the control group;

·  The females developed mammary and pituitary gland tumors, and suffered pituitary gland abnormalities and hormone disruption;

·  Male rats also developed tumors, but in particular experienced serious kidney and liver damage;

·  50 percent of males and 70 percent of females died prematurely, compared to 30 percent and 20 percent, respectively, in the control groups."You can read about the study here.

Many other countries have banned substances that our own FDA still allows.  I wrote about throwing out all our cookware here and buying stuff we could barely afford here:  Why Cushing's May Not Be As Be As Rare As They Thought: Check your Cookware and Your Chemical Neighbors

Courageous Cushies

For Lori and all of us Cushies, who wake up every morning and try to live a full life.

(Thanks to my friend Meagan for sharing.)

My friend Lori faces 5th pituitary surgery

Please keep Lori in your thoughts. Her cortisol levels dramatically improved/ dropped following Monday's operation in which the neurosurgeon removed two separate tumors. However, the lab work indicates there is still tumor tissue present. She will be having another pituitary surgery -- her 5th in an area the size of a pea-- tomorrow at 7 am central time. The neurosurgeon will remove her entire pituitary gland and the bone surrounding it. This is a much more invasive surgery.

Lori has been fighting for too long to give up now. This surgery is her only option. She first went to the National Institute of Health, long recognized as the most knowledgeable about this disease, when she was only 13. Twenty years later, she is still fighting Cushing's. For many of us, Cushing's won't turn loose. This time, it will.

http://cushingsmoxie.blogspot.com/2012/09/a-case-of-ectopic-pituitary-adenoma.html?m=1

My Friend Lori, a Cushie Warrior, Faces 4th Pituitary Surgery

A friend with persistent Cushing's had her fourth pituitary surgery this past Monday. The neurosurgeon found an 8-9 mm tumor in her sinus cavity that was definitely a Cushing's tumor. This trouble spot had a tail of tissue trailing back to her pituitary to a small spot of abnormal cells.

The neurosurgeon believes that just a few microscopic cells fell during her first pituitary surgery and started a growth of abnormal ACTH cells that caused her Cushing's. The patient had this abnormality in her sinus MRI and no doctor related it to her prior history of transsphenoidal pituitary resection. In fact, pervious pituitary surgeries, while done transsphenoidally (across the sphenoid sinus), were done sublabial (under the lip). This surgery was done endoscopically or through the nose, and this offered the surgeon visual access to the tumor that may have been hiding there since her first pituitary surgery 20 years prior.

Several other Cushies with persistent Cushing's  have commented since Monday that they too have a "cyst" in their sinus cavity. Many will now ask their neurosurgeons to examine these suspicious areas more closely as possible source of abnormal ACTH cells which cause Cushing's.

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A case of ectopic pituitary adenoma occurring in the sphenoid sinus

Neurological surgery (2011)

Volume: 39, Issue: 6, Pages: 601-605
Click for full article full article.

Abstract

Ectopic pituitary adenomas are relatively rare tumors. We present a case of ectopic pituitary adenoma occurring in the sphenoid sinus. A 63-year-old woman was referred to our hospital complaining of headache. She had no endocrinological abnormalities. Magnetic resonance imaging showed a tumor in the sphenoid sinus, adjacent to the sellar floor and appearing as a low-signal on T1-weighted image and a high signal on T2-weighted image. No connection between the normal pituitary gland and tumor was observed. Using an endonasal-transsphenoidal approach assisted with neuro-endoscopy, we performed total removal of the tumor. No connection between the normal pituitary gland and the tumor was found. Histopathological analysis showed a pituitary adenoma. As demonstrated by our case report, differential diagnosis of a tumor occurring in the sphenoid sinus must include consideration of the existence of an ectopic pituitary adenoma.

PATIENT STORY: Jennifer S. - My Story

Thank you to the Pituitary Network Association (www.pituitary.org) for sharing patient stories.

https://www.pituitary.org/library/library_detail.aspx?page_id=2566

Jennifer S. - My Story, a Patient Perspective

8/2/2011    Categories:  Women's Health | Inspirational | Patient Perspectives |Featured Articles

It was initially discovered that I had a pituitary tumor in 1992 after a year of strange periods, emotional strife and infections . They did an MRI after my prolactin levels were slightly elevated, I was lactating out of both breasts. Despite the full lactation, they decided that the lab levels indicated nothing much at all, and so my lesion was declared an incidentaloma.  From 1992 until 2000, I had to beg for MRIs or testing as I was told that any problem I had, if any, was thyroid, PCOS, endometriosis or that I had no issue at all - but it was certainly not pituitary.

I went from endocrinologist to endocrinologist. I waited months to see the doctors noted in magazines only to be told that I had no problem at all. I saw all sorts of specialists as I kept getting sick. I saw infectious disease specialists, dermatologist, oncologists, hematologists. My docs were so mystified they kept giving me HIV testing (all negative) and even sent me to a specialist who could not figure it out either. I was not typical in any way - I did not have diabetes, high glucose or high cholesterol or even high blood pressure. But I gained weight fast, had a bleeding disorder, would not heal, got sick very easily, had acne,  had striae (who thought stretch marks were symtptoms!), and a red sweaty face. I was called a liar many times about my diet, exercise and life.

At one point, like many of us pituitary patients, I gave up and stopped seeing doctors. I got worse. The next doctor I saw was a turning point. I gave my list of symptoms and he was the first to say the word "Cushing's". However, he gave up after one urine test but just saying the word was enough that I could do research and realize Cushing's, sadly, fit me perfectly.

At that point I did all I could to educate myself on Cushing's syndrome and testing. I would test barely high at times and then normal. I knew I had the cyclical or episodic form and that most doctors do not acknowledge or treat that form. Finally in 2004, I found a doctor that would treat me and I had surgery a few months later after having some elevated testing. They found that I had two lesions - a 3mm prolactinoma and acth secreting corticotroph hyperplasia.

I had a short period where I was ok, but I never lost my Cushing's, In fact, I got worse. So after 2 years, it was decided to remove my adrenals. They were enlarged and showed bilateral cortical hyperplasia.

Since the removal of my adrenals, I have had both shoulders frozen. I have had significant myopathy, proximal weakness and loss of stamina. I was not able to return to work.  My ACTH which was always normal, rose to over 1000 within a few months of surgery. It is currently near 4000. Since my pathology and my diagnosis is a matter of *dispute* as some doctors cannot accept cyclical patients and/or acth secreting hyperplasia, getting aftercare has been a challenge. Aftercare is not being addressed well by doctors. My next treatment option is radiation with no defined target.

• Be an educated patient. Know the test protocols such as time of test, if ice is needed, medications that mess up testing, etc.
• Whenever possible, get copies of your records and read them. Many times I was told I was normal and I was not.
• Network with other patients to learn.
• Surgical recovery may be a few weeks, hormonal recovery can take years.
• Just because a doctor worked for you, they may or may not work for someone else so best to direct to doctor lists. Opposite is true as well.
• Read reliable information. You may not understand the lingo at first but it does sink in later. Save the links or save the pages to read.
• Everyone's case is different so a best case and worst case may not be you.
• Most people posting on the internet are there because they have problems, Those that do not are out living their lives - which can make you think everyone is having problems - it is not the case.

I Can't Repeat This Enough

Obesity & Cushing's

As I struggle to find sleep again tonight, I happened upon this article in the Los Angeles Times, entitled "Some anti-obesity campaigns may backfire, researchers say."

http://www.latimes.com/news/science/la-sci-obesity-20120912,0,5230176.story

I felt compelled to write the author to give a little information about what it is like to suffer from obesity.

If you are here taking a moment to read this, I thank you, Melissa. Please, please help us. ~m

** ** **

Hello Melissa.

I am obese and I appreciate the sensitivity shown in your recent article. I gained 110 pounds rapidly and unexpectedly despite all efforts to eat well and exercise for many years. I wonder what percentage of this country's obese population may actually have Cushing's. Many otherwise helpful doctors consider Cushing's "impossible to have" so much so that they refuse to test a patient especially when it is the patient who used the internet and available medical literature to research the symptoms online. Many physicians have blamed, scolded, humiliated, and ultimately dismissed my Cushie friends and me when tumors causing a disruption in our endocrine systems are ultimately proven to be the culprits behind our massive weight gain and constellation of bizarre symptoms.

This paragraph, in particular, somewhat explains the prejudice my fellow Cushing's patients face at the hand of those medical professionals we seek out to help us:  

"Even among physicians, obese patients elicit feelings of prejudice and blame. A 2003 survey, published in the journal Obesity Research, found that half considered their obese patients awkward, ugly, unattractive and unlikely to follow their advice. In addition, one-third of doctors viewed obese patients as weak-willed, sloppy and lazy."

On a personal note, I have had two pituitary brain surgeries to remove two separate tumors, yet I am still suffering from persistent Cushing's disease. I hope to have another major surgery within the next few months in another effort to cure myself of Cushing's.

If you are interested in exploring this cause of obesity in greater depth, it would be my honor to sit with you and share more information about Cushing's. An article such as this will spread awareness and undoubtedly help countless people who have suffered for many years undiagnosed from a disease that even the medical community fails to recognize. After all, we Cushies deserve compassion and love despite our illnesses, especially since friends and family fail to support or believe us. We did not do this to ourselves, and we have no control over the damage these tumors do to our bodies. 

Melissa

http://cushingsmoxie.blogspot.com

We are Strong. We are Survivors.

"I am not what happened to me --

I am what I choose to become."

~ Carl Jung

MAGIC & Giving Forward

The big announcement has been made!

The 8th Annual Convention for Adults with Pituitary Disorders will be held in Las Vegas, Nevada from Friday, April 19, 2013 to Sunday, April 21, 2013. Many will arrive on Thursday and leave Sunday or Monday.  Please mark these dates in your calendar and plan to attend, as this will be a wonderfully informative and fulfilling experience as you meet many others with your 'rare' disease.

I posted about the importance of the Magic Foundation on my blog before.

Today, I also wanted to direct you to a place where you can help folks who need a little extra help to cover the expenses to attend the MAGIC Convention. Knowing first hand how much the MAGIC Convention helped her, my dear friend Karen has spearheaded a program to help patients with pituitary disorders join fellow Cushies at next year's MAGIC Foundation's annual convention. I ask you to contribute to the Educational Program Scholarship at giveforward.com for this cause.

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Other resources:

• facebook.com/TheMAGICFoundation
• magicfoundation.org
• Cushing's page for the MAGIC Foundation

Feeling Hopeful and Grateful Today

"At times our own light goes out and is rekindled by a spark from another person. Each one of us has cause to think with deep gratitude of those who have lighted the flame within us."

~ Albert Schweitzer (1875-1965), Nobel Laureate

Here we go again...

Getting an MRI on both hips. Hope to rule out osteonecrosis after increasing pain in pelvis and hip joints. Unfortunately, this is so common with Cushing's :/