Urgent Action Needed

  • Thursday, July 19, 2018

Request Priority Review of Scenesse


The new drug application for Scenesse has been submitted to the FDA - and it is time to advocate! We need your letters to the FDA Commisioner requesting Priority Review. Please send your letter immediately to add to our growing pile!! It is starting to look impressive!

All letters must be signed and sent to the APF ASAP.

We will submit the letters to the FDA by July 19, 2018.


Begin your letter:

Scott Gottlieb, M.D.
FDA Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993

Dear FDA Commissioner:


Mail your letter to:

American Porphyria Foundation
4915 St. Elmo Avenue, Suite 105
Bethesda, Maryland 20814

Alternatively you can scan your signed letter and email it to porphyrus@porphyriafoundation.com.

We will present all of the letters at once to the FDA!

Please send letters today or ASAP! Time is of the Essence!


Clinical Trial for EPP!

Research is the key to your cure! What is the most important element in research? YOU! You now have an opportunity to participate in a trial for a new EPP investigational drug. Contact the APF for details. YOU are our hope for the future. Contact the APF on 1-866-APF-3635 or email porphyrus@porphyriafoundation.com.

An oral investigational drug has been developed with the potential to increase sunlight duration and tolerance in individuals with EPP. This phase II clinical trial is needed to understand if the investigational drug works and to ensure that it is safe. The study duration is 24 weeks including follow-up. You must be age 18-70 to participate and have a confirmed diagnosis of EPP. All travel is included and will be arranged by a concierge service.

"Remember...Research is the key to YOUR cure!”

Each Step Toward Finding an Effective Treatment is Important!


Givosiran Trials

Thanks to YOU, our APF members and patients, the Givosiran trials are now full. We are so proud of each of you for participating in this important research. As of June 29, 2018 the trials have closed for enrollment.


Update your records....

We have moved.


The American Porphyria Foundation is excited to announce out move to Bethesda, Maryland - next door to our nation's capital. Please update your records with our new address.

American Porphyria Foundation
4915 St. Elmo Ave., Suite 105
Bethesda, MD 20814
Telephone: 301.347.7166



Rare Disease Legislative Advocates is hosting In-District Lobby Days to facilitate meetings for rare disease advocates across the country with members of Congress in their local offices during the 2018 summer in-district work period (July 31st through Sept. 4th).

Meeting with your representative and senators throughout the year is critical to building key relationships. These meetings also provide an opportunity to discuss legislation that is meaningful to the rare disease community, and to highlight the importance of the Rare Disease Congressional Caucus. Make YOUR voice heard as you advocate on behalf of the rare disease community!

Registration for In-District Lobby Days is FREE and open through July 4th. You will be provided the option to specify you availability and the distance you are willing to travel for a meeting.

All In-District Lobby Day participants will be invited to a webinar on Thursday, July 26th,at 2pm EST to go over key legislative issues and tips for success