UPdates

MT-7117 PHASE 3 TRIAL PARTICIPANTS WILL CONTINUE TO HAVE ACCESS TO DERSIMELAGON WITH AN INCREASED DOSE

 

Mitsubishi Tanabe Pharma America (MTPA) announced an update to the MT-7117 Open Label Extension of the Phase 3 clinical trial.

Patients who completed the Phase 3 clinical trial and are currently participating in the Open Label Extension (OLE) will now have continued access to dersimelagon.

  • Who is eligible? Patients who completed the Phase 3 clinical trial, including the double-blind extension, and are currently participating in the optional open label extension.
  • Extension time period: Participants will have access to dersimelagon for up to an additional 30 months.
  • Dose: Participants will receive 200mg dose (rather than initial 100mg open label dose).
  • Action: Contact your Study Site to transition to this dose at your next regular study visit.

Please direct questions to your study site or United Porphyrias Association at info@porphyria.org

 

PRELIMINARY FINDINGS RELEASED FOR BITOPERTIN
Disc Medicine recently shared preliminary findings from its ongoing, Phase 2 open-label BEACON trial in Australia which is evaluating bitopertin in patients with EPP and XLP. Early results found:

  • Bitopertin consistently decreases protoporphyrin IX
  • Patients had significant increases in reported sunlight tolerance and quality of life
  • Bitopertin was well-tolerated with no serious adverse events, no reported discontinuations or dose reductions, no adverse events greater than Grade 1 (mild), and no meaningful changes in observed in mean hemoglobin level

These data were presented at the European Hematology Association 2023 Congress in Frankfurt, Germany. More details about the results are available HERE.
 

INTERESTED IN PARTICIPATING IN THE BITOPERTIN TRIALS?
The phase 2 AURORA trial in the US for bitopertin is still recruiting participants! Participants must have EPP, live in the US or Canada, be over 18 and meet additional eligibility requirements. ?Please complete an INTEREST FORM. Your response is completely confidential, only for designating your interest in further information. You're also free to call our office at 800-868-1292 or email us at info@porphyria.org. 

 

UPA ON YOUTUBE – SCIENTIFIC ADVISORY BOARD VIDEO SERIES
The UPA Scientific Advisory Board contributed to a video series highlighting responses to questions regarding porphyria diagnosis, management, and treatment. Access videos HERE.

 

 

UP and Coming

LET'S CONNECT! NEW ONLINE COMMUNITY

You're invited to our new Discord server - a safe, welcoming space for the porphyria community. Join us to share your experiences and tips, learn from others and gain support from fellow porphyria patients. Discord is an intuitive platform where you can chat in topic-specific channels, use direct messages and voice call, access resources, and find solace. Let's navigate this journey together!
JOIN the Porphyria Discord
 

YOU ARE NOT ALONE!Connecting with others who understand what you're going through is a great way to build a support system and community. UPA is pleased to launch Connect Up small group meetings for patients and caregivers to connect and socialize with others in a safe and inclusive environment. Connect UP groups will be peer-led and offered regularly over Zoom. SIGN UP HERE.
 

PORPHYRIA TOGETHER ON FACEBOOKWe welcome you to join Porphyria Together, a new closed Facebook group where you will find helpful information in a safe community setting. JOIN US online!

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The United Porphyrias Association is committed to improving the quality of life of the porphyria patient community and is relentlessly focused on advancing disease awareness, research, and therapies in all the porphyrias.

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Victor Mejias

Erythropoietic Protoporphyria (EPP)

"I was diagnosed at the age of 6 with EPP after my parents noticed that every time I was outside I was experiencing anguishing discomfort. I never fully comprehended my diagnosis until I was able to connect with others. With the help of the UPA, I want to be able to help others live more comfortably with EPP and support the vision of a world where patients are free from the pain and challenges of porphyria. "

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