about porphyria

Porphyria Treatments

FDA Approved Treatments

  • PANHEMATIN® - hemin for injection prescription medication used to relieve repeated attacks of AIP related to the menstrual cycle in affected women, after initial carbohydrate therapy is known or suspected to be inadequate - Contact The Recordati Rare Diseases Patient Support Program for personalized support for PANHEMATIN.
  • GIVLAARI® (givosiran) - prescription medicine used to treat acute hepatic porphyria (AHP) in adults - Alnylam Assist™ provides patients with assistance accessing GIVLAARI.
  • SCENESSE® (afamelanotide) implant - prescription medication indicated to increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP) - CLINUVEL is committed to facilitating treatment access for SCENESSE through their Support Programs.

For information on Patient Assistance Programs visit the UPA Resource Hub

Investigational Treatments

  • Disc Medicine is investigating Bitopertin as a treatment for EPP
  • FDA is sponsoring study of Cimetidine as a treatment for EPP/XLP
  • Mitsubishi Tanabe is investigating Dersimelagon (MT-7117) as a treatment for EPP/XLP

If you are interested in learning more about treatments or participating in clinical research, contact UPA at info@porphyria.org or 800-868-1292.