FOR INFORMATION ON COVID-19, GO TO COVID-19 INFORMATION HERE.
NADF has a part-time POSITION open: PROGRAMS AND PUBLICATIONS COORDINATOR
INFORMATION ON firST CAH PATIENT TO RECEIVE INVESTIGATIONAL GENE THERAPY
MARCH 3, 2022
Letter from BridgeBio regarding the Adrenas program which provides information about the first patient to receive Adrenas' investigational gene therapy. See letter here.
Letter from BridgeBio regarding the Adrenas program which provides information about the first patient to receive Adrenas' investigational gene therapy. See letter here.
bridgebio pharma press release for CAH clinical trial
JANUARY 26, 2022
BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH) - If successful, BridgeBio’s investigational gene therapy BBP-631 would be the first therapy for CAH to restore the body’s hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone. Read the full press release here.
BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH) - If successful, BridgeBio’s investigational gene therapy BBP-631 would be the first therapy for CAH to restore the body’s hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone. Read the full press release here.
antares pharma announces positive result from phase i study for auto-injector
JANUARY 12, 2022
Antares Pharma, a specialty pharmaceutical company, announced positive results from a Phase I study for an auto-injector for adrenal crisis. See Antares Pharma's full press release here.
Antares Pharma, a specialty pharmaceutical company, announced positive results from a Phase I study for an auto-injector for adrenal crisis. See Antares Pharma's full press release here.
FDA approves antiviral pills to treat covid-19 at home
The Food and Drug Administration announced an emergency use authorization (EUA) of antiviral treatments for mild-to-moderate coronavirus disease (Covid-19) on December 22 and 23rd. This wonderful news provides a useful new course of action for covid-19 infections, including breakthrough cases of omicron as well as the other variants.
These antiviral treatments will eventually be for everyone, but initially will be indicated especially for those who are at high risk of complications, including those with adrenal insufficiency. The remedies will be very similar to the use of Tamiflu which is used for acute cases of influenza.
A provider will need to verify a positive PCR, symptoms of covid, and then start the treatments within 5 days of onset. However, currently these therapies are not available but are expected to be distributed gradually in the next month, with supplies increasing over the next few months.
In the meantime, those who do have breakthrough symptomatic illness should try to get the one monoclonal antibody infusion that works for omicron, though it is difficult to get because of shortages. Regeneron and Eli Lilly both announced their antibody treatments didn’t work as effectively in diffusing omicron.
Before any treatments, patients must review their current condition with their physician, including the list of medications they are currently taking. Some drugs cannot be taken with these new antiviral treatments.
More information can be found in FDA’s announcements:
Coronavirus (COVID-19) Update: FDA Authorizes First Oral Antiviral for Treatment of COVID-19 | FDA
Coronavirus (COVID-19) Update: FDA Authorizes Additional Oral Antiviral for Treatment of COVID-19 in Certain Adults | FDA
These antiviral treatments will eventually be for everyone, but initially will be indicated especially for those who are at high risk of complications, including those with adrenal insufficiency. The remedies will be very similar to the use of Tamiflu which is used for acute cases of influenza.
A provider will need to verify a positive PCR, symptoms of covid, and then start the treatments within 5 days of onset. However, currently these therapies are not available but are expected to be distributed gradually in the next month, with supplies increasing over the next few months.
In the meantime, those who do have breakthrough symptomatic illness should try to get the one monoclonal antibody infusion that works for omicron, though it is difficult to get because of shortages. Regeneron and Eli Lilly both announced their antibody treatments didn’t work as effectively in diffusing omicron.
Before any treatments, patients must review their current condition with their physician, including the list of medications they are currently taking. Some drugs cannot be taken with these new antiviral treatments.
More information can be found in FDA’s announcements:
Coronavirus (COVID-19) Update: FDA Authorizes First Oral Antiviral for Treatment of COVID-19 | FDA
Coronavirus (COVID-19) Update: FDA Authorizes Additional Oral Antiviral for Treatment of COVID-19 in Certain Adults | FDA
NADF HAS A NEW ADDRESS!
We would like to take this opportunity to inform you of a change in our mailing address. The new address for the National Adrenal Diseases Foundation is:
PO Box 95149, Newton, MA 02495
Please mail all communications / donations to the new address or donate online here.
Q: If I mailed a letter or donation recently to the old address will NADF still get it?
A: Yes, absolutely, and thank you! Your correspondence will be safely forwarded to the new address by the United States Postal Service (USPS).
Q: Did the NADF phone # change too?
A: No, NADF’s phone number has not changed. In fact, you can reach NADF several ways:
Q: If I mailed a letter or donation recently to the old address will NADF still get it?
A: Yes, absolutely, and thank you! Your correspondence will be safely forwarded to the new address by the United States Postal Service (USPS).
Q: Did the NADF phone # change too?
A: No, NADF’s phone number has not changed. In fact, you can reach NADF several ways:
- Phone: (847) 726-9010
- Email: nadfsupport@NADF.us
- Mail: PO Box 95149, Newton, MA 02495
Pediatric Adrenal Insufficiency Action Plan with Pictograms
Health information related to adrenal insufficiency can seem unfamiliar and complicated. Instructions can seem overwhelming to caregivers and are often presented in ways that make it difficult to understand and act on. That’s why NADF was excited to support the work of Dr. Patrick Reeves at the Walter Reed National Military Medical Center. Dr. Reeves desire was to improve pediatric adrenal insufficiency literacy using pictograms and he enlisted NADF to help the effort.
The objective of the tool is to improve the ability for parents and clinicians to fully comprehend and take appropriate medical action for children with adrenal insufficiency. The new Action Plan tool and video instructions can be viewed here the full article below.
The objective of the tool is to improve the ability for parents and clinicians to fully comprehend and take appropriate medical action for children with adrenal insufficiency. The new Action Plan tool and video instructions can be viewed here the full article below.
nadf meeting with fda
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July 12, 2021
We are an adrenal disease community bonded by our shared experiences, and finally, the US Food and Drug Administration (FDA) had a chance to hear about it. Organized by Lori Engler, NADF Executive Director, the session described topics that are most important to the community. The presentation was conducted by several NADF leaders to educate and bring awareness to these topics, clearly explain how they affect our community and to suggest solutions.
Patient Listening Sessions help the FDA understand what is important to patients, caregivers, and advocates when medical products are being developed. The format was a one-hour discussion designed to help those involved in the drug approval process understand the obstacles and unmet needs of patients with adrenal insufficiency.
Here's a summary of topics discussed during NADF’s meeting with the FDA. For each topic, NADF discussed possible solutions, including immediate needs: Modified release hydrocortisone options, Autoinjector for hydrocortisone, and better protection of life-preserving medications to prevent shortages.
According to the FDA, “Patient Listening Sessions are a resource for the medical product Centers to quickly engage with patients for their advocates and are one of many ways the patient community can share their experience with a disease or condition by talking directly with FDA staff.” The FDA hopes to better understand the burdens that diseases and treatments have on our daily lives and to learn what is important for our community.
This was a strong starting point and a very important new direction for NADF. Just a couple of days after the meeting the FDA shortage database was updated…a positive sign that they really were listening.
We are an adrenal disease community bonded by our shared experiences, and finally, the US Food and Drug Administration (FDA) had a chance to hear about it. Organized by Lori Engler, NADF Executive Director, the session described topics that are most important to the community. The presentation was conducted by several NADF leaders to educate and bring awareness to these topics, clearly explain how they affect our community and to suggest solutions.
Patient Listening Sessions help the FDA understand what is important to patients, caregivers, and advocates when medical products are being developed. The format was a one-hour discussion designed to help those involved in the drug approval process understand the obstacles and unmet needs of patients with adrenal insufficiency.
Here's a summary of topics discussed during NADF’s meeting with the FDA. For each topic, NADF discussed possible solutions, including immediate needs: Modified release hydrocortisone options, Autoinjector for hydrocortisone, and better protection of life-preserving medications to prevent shortages.
- Topic I: How can the FDA help create significant drug innovation in the management of adrenal insufficiency? How can we place a higher priority on new adrenal insufficiency treatments given patients’ lower quality of life and higher mortality rates?
Novel modified-release hydrocortisone preparations are available in Europe and have been shown to improve quality of life, improve innate immunity responses, lead to weight reduction, and reduce hemoglobin. NADF leaders also emphasized the need for Solu-Cortef Emergency Administration Technology and explained adrenal crisis is recognizable by its typical symptoms of diarrhea, vomiting and nausea. Unfortunately, the delay between the start of these symptoms and the administration of emergency treatment is the principal cause of death. - Topic II: Drug shortages have been a significant problem especially given the lack of options for treatment of life-preserving medications. Drug shortages can be a matter of life and death.
NADF discussed the issues created by shortages including treatment interruption, transitions to less-effective treatments, and rationing of life-saving treatments.
According to the FDA, “Patient Listening Sessions are a resource for the medical product Centers to quickly engage with patients for their advocates and are one of many ways the patient community can share their experience with a disease or condition by talking directly with FDA staff.” The FDA hopes to better understand the burdens that diseases and treatments have on our daily lives and to learn what is important for our community.
This was a strong starting point and a very important new direction for NADF. Just a couple of days after the meeting the FDA shortage database was updated…a positive sign that they really were listening.
NEWS ON ETON'S ACQUISITION RIGHTS TO US CROSSJECT'S ZENEO® HYDROCORTISONE NEEDLELESS AUTOINJECTOR
June 15, 2021
Adrenal crisis is recognizable by its typical symptoms of diarrhea, vomiting and nausea. Unfortunately, the delay between the start of these symptoms and the administration of emergency treatment is the principal cause of death.
On June 15, 2021 Eton announced the acquisition of US rights to Crossject’s ZENEO® hydrocortisone needleless autoinjector. The link to the press release below.
https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-acquires-us-and-canadian-rights-zeneor
Please see Zeneo® Hydrocortisone Autoinjector FAQs.
Adrenal crisis is recognizable by its typical symptoms of diarrhea, vomiting and nausea. Unfortunately, the delay between the start of these symptoms and the administration of emergency treatment is the principal cause of death.
On June 15, 2021 Eton announced the acquisition of US rights to Crossject’s ZENEO® hydrocortisone needleless autoinjector. The link to the press release below.
https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-acquires-us-and-canadian-rights-zeneor
Please see Zeneo® Hydrocortisone Autoinjector FAQs.
STATEMENT FROM NADF'S MEDICAL DIRECTOR ABOUT THE JOHNSON & JOHNSON COVID-19 VACCINE
March 5, 2021
Everyone with adrenal insufficiency should get vaccinated with whichever of these vaccines is available to them. I am not concerned about the reported lower efficacy of the J&J vaccine in reducing the incidence of mild to moderate disease. The real benefit of the J&J vaccine is the astounding 100% prevention of severe disease leading to death. Also, the fact that it is one injection that does not require freezing will make it a lot easier for people in rural areas to be vaccinated. People with adrenal insufficiency in NY State are now eligible. Hopefully, this will be extended to all other states soon.
Everyone with adrenal insufficiency should get vaccinated with whichever of these vaccines is available to them. I am not concerned about the reported lower efficacy of the J&J vaccine in reducing the incidence of mild to moderate disease. The real benefit of the J&J vaccine is the astounding 100% prevention of severe disease leading to death. Also, the fact that it is one injection that does not require freezing will make it a lot easier for people in rural areas to be vaccinated. People with adrenal insufficiency in NY State are now eligible. Hopefully, this will be extended to all other states soon.
New international Adrenal disease Organization Consortium
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February 2021
We are excited to announce the launch of an Adrenal Patient Organization Consortium, created to work collaboratively with an international network of adrenal disease organizations who share a common bond. See the full article here: https://www.nadf.us/adrenal-disease-consortium.html |
CORTISONE ACETATE SHORTAGE
December 30, 2020
West-Ward Pharmaceuticals stated that 25 mg cortisone acetate tablets are on a backorder currently due to production delays. The ETA for distributors to receive inventory is the week of January 4th and to allow a few days for availability.
See the Corticosteroid Comparison Chart here
West-Ward Pharmaceuticals stated that 25 mg cortisone acetate tablets are on a backorder currently due to production delays. The ETA for distributors to receive inventory is the week of January 4th and to allow a few days for availability.
See the Corticosteroid Comparison Chart here
statement from NADF's medical advisor about the covid-19 vaccine
December 17, 2020
Many are questioning whether the COVID-19 vaccine is safe for those with adrenal insufficiency. After a review, NADF’s Medical Advisor Dr. Margulies provided this statement:
“At this time, I am satisfied that the FDA has done the appropriate review of the Pfizer vaccine, and I am enthusiastic about recommending it to every adult. I plan to take it myself. There is no reason for anyone with adrenal insufficiency, whether primary or secondary, to avoid this vaccine. It is as safe as the influenza vaccine. It has mild side effects, such as sore arm, mild fever and fatigue that last about one day. The Moderna vaccine will be reviewed soon, but so far it looks very similar to the Pfizer product. Unless something surprising is found, I expect it will also be suitable for every adult.”
Additional information:
As always, you and your doctor should decide together if the vaccine is right for you and, if so, which one.
Many are questioning whether the COVID-19 vaccine is safe for those with adrenal insufficiency. After a review, NADF’s Medical Advisor Dr. Margulies provided this statement:
“At this time, I am satisfied that the FDA has done the appropriate review of the Pfizer vaccine, and I am enthusiastic about recommending it to every adult. I plan to take it myself. There is no reason for anyone with adrenal insufficiency, whether primary or secondary, to avoid this vaccine. It is as safe as the influenza vaccine. It has mild side effects, such as sore arm, mild fever and fatigue that last about one day. The Moderna vaccine will be reviewed soon, but so far it looks very similar to the Pfizer product. Unless something surprising is found, I expect it will also be suitable for every adult.”
Additional information:
- Many people in the clinical trials had some short-term side effects from taking the COVID-19 vaccine. The side effects are usually not serious -- one of the most common is a sore or achy arm. Some people develop low-grade fevers or chills and feel tired. This is because the vaccine is working and causing a response from the immune system. As always, follow NADF’s stress dosing guidelines to determine if you need additional steroids.
- Remember that the Pfizer vaccine is given in two doses three weeks apart. It is more common to have side effects from the second dose than the first. You must receive both doses to get the full benefit from the vaccine.
- The vaccine may not be available to most people until spring or summer of 2021. Because of the limited supply of the vaccines, people at very high risk, such as health care providers and people who live in nursing homes or assisted living facilities, will get a vaccine before people whose risk of illness is lower.
As always, you and your doctor should decide together if the vaccine is right for you and, if so, which one.
Eton Pharmaceuticals Announces Availability of Orphan Drug ALKINDI® SPRINKLE (hydrocortisone) in the United States
November 24, 2020
DEER PARK, Ill., (GLOBE NEWSWIRE): Eton Pharmaceuticals, Inc. (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the full availability of ALKINDI® SPRINKLE for sale and distribution in the United States.
SEE FULL ANNOUNCEMENT: https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-availability-orphan-drug-alkindir
DEER PARK, Ill., (GLOBE NEWSWIRE): Eton Pharmaceuticals, Inc. (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the full availability of ALKINDI® SPRINKLE for sale and distribution in the United States.
SEE FULL ANNOUNCEMENT: https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-availability-orphan-drug-alkindir
eton pharmaceuticals announces fda approval of orphan drug alkindi® sprinkle (hYDROCORTISONE) AS REPLACEMENT THERAPY IN PEDIATRIC PATIENTS WITH ADRENOCORTICAL INSUFFICIENCY
September 29, 2020
ALKINDI SPRINKLE is the first and only FDA-approved granular hydrocortisone formulation for adrenocortical insufficiency specifically designed for children. Eton expects ALKINDI SPRINKLE to be available in the fourth quarter of 2020.
SEE ARTICLE: https://www.globenewswire.com/news-release/2020/09/29/2101010/0/en/Eton-Pharmaceuticals-Announces-FDA-Approval-of-Orphan-Drug-ALKINDI-SPRINKLE-hydrocortisone-as-Replacement-Therapy-in-Pediatric-Patients-with-Adrenocortical-Insufficiency.html
ALKINDI SPRINKLE is the first and only FDA-approved granular hydrocortisone formulation for adrenocortical insufficiency specifically designed for children. Eton expects ALKINDI SPRINKLE to be available in the fourth quarter of 2020.
SEE ARTICLE: https://www.globenewswire.com/news-release/2020/09/29/2101010/0/en/Eton-Pharmaceuticals-Announces-FDA-Approval-of-Orphan-Drug-ALKINDI-SPRINKLE-hydrocortisone-as-Replacement-Therapy-in-Pediatric-Patients-with-Adrenocortical-Insufficiency.html
Acella Pharmaceuticals, LLC Issues Voluntary Nationwide Recall of Two Lots of NP Thyroid®, Thyroid Tablets, USP Due to Sub Potency
September 22, 2020
Acella Pharmaceuticals, LLC is voluntarily recalling one lot of 15-mg and one lot of 120-mg NP Thyroid®, Thyroid Tablets, USP [levothyroxine (T4) and liothyronine (T3)] to the consumer level. The products are being recalled because testing has found these lots to be sub potent. The product may have as low as 87% of the labeled amount of levothyroxine (T4). More information can be found at https://npthyroid.com/product-updates/.
SEE ARTICLE: https://www.fda.gov/safety/recalls-market-withdrawals-safety-alerts/acella-pharmaceuticals-llc-issues-voluntary-nationwide-recall-two-lots-np-thyroidr-thyroid-tablets?utm_medium=email&utm_source=govdelivery#recall-announcement
Acella Pharmaceuticals, LLC is voluntarily recalling one lot of 15-mg and one lot of 120-mg NP Thyroid®, Thyroid Tablets, USP [levothyroxine (T4) and liothyronine (T3)] to the consumer level. The products are being recalled because testing has found these lots to be sub potent. The product may have as low as 87% of the labeled amount of levothyroxine (T4). More information can be found at https://npthyroid.com/product-updates/.
SEE ARTICLE: https://www.fda.gov/safety/recalls-market-withdrawals-safety-alerts/acella-pharmaceuticals-llc-issues-voluntary-nationwide-recall-two-lots-np-thyroidr-thyroid-tablets?utm_medium=email&utm_source=govdelivery#recall-announcement
adrenal insufficiency fund opens at the healthwell foundation
Copayment and Premium Assistance Now Available
September 14, 2020
GERMANTOWN, MD — The HealthWell Foundation®, an independent non-profit that provides a financial lifeline for inadequately insured Americans, has launched a new fund to provide copayment and premium assistance to patients living with adrenal insufficiency (AI).
GERMANTOWN, MD — The HealthWell Foundation®, an independent non-profit that provides a financial lifeline for inadequately insured Americans, has launched a new fund to provide copayment and premium assistance to patients living with adrenal insufficiency (AI).
PREVENTION OF ADRENAL CRISIS: CORTISOL RESPONSES TO MAJOR STRESS COMPARED TO STRESS DOSE HYDROCORTISONE DELIVERY
July 2020
A recent paper published by The Journal of Clinical Endocrinology & Metabolism* provided updated information on the most appropriate mode of hydrocortisone delivery in patients with adrenal insufficiency that are hospitalized with major surgery or major stress from severe infection. That paper can be found on our website here. https://www.nadf.us/medical-publications.html
The conclusion they came to is that continuous intravenous hydrocortisone infusion should be favored over intermittent bolus administration in the prevention and treatment of adrenal crisis during major stress. This keeps the circulating cortisol in target range over the period of time needed to treat an adrenal crisis.
*The Journal of Clinical Endocrinology & Metabolism, Volume 105, Issue 7, July 2020, Pages 2262–2274, https://doi.org/10.1210/clinem/dgaa133
A recent paper published by The Journal of Clinical Endocrinology & Metabolism* provided updated information on the most appropriate mode of hydrocortisone delivery in patients with adrenal insufficiency that are hospitalized with major surgery or major stress from severe infection. That paper can be found on our website here. https://www.nadf.us/medical-publications.html
The conclusion they came to is that continuous intravenous hydrocortisone infusion should be favored over intermittent bolus administration in the prevention and treatment of adrenal crisis during major stress. This keeps the circulating cortisol in target range over the period of time needed to treat an adrenal crisis.
*The Journal of Clinical Endocrinology & Metabolism, Volume 105, Issue 7, July 2020, Pages 2262–2274, https://doi.org/10.1210/clinem/dgaa133
cAH Clinical Program - Spruce biosciences
Spruce Biosciences, a late-stage clinical development company focused on developing and commercializing novel therapies for rare endocrine disorders, has announced the launch of CAHstudy.com a new website and patient resource to enable adults with Congenital Adrenal Hyperplasia (CAH) to register their interest in the CAHmelia clinical program, which is expected to commence in the second half of 2020.
exciting news from nadf
April 25, 2020
We would like to take this opportunity to make some exciting and important announcements. We have seen rapid growth of our foundation over the past decade or so, with the trajectory of growth in the responsibilities required to run NADF accelerating equally as quickly.
We would like to take this opportunity to make some exciting and important announcements. We have seen rapid growth of our foundation over the past decade or so, with the trajectory of growth in the responsibilities required to run NADF accelerating equally as quickly.
For those with adrenal insufficiency and confirmed or suspected Covid-19, increase the dose of hydrocortisone to 20 mg every 6 hours until the fever subsides, and then go back to the usual double dose until completely better.
