Bitopertin Granted Orphan Drug Status for Erythropoietic Protoporphyria

The Food and Drug Administration (FDA) has granted Orphan Drug designation to bitopertin for the treatment of erythropoietic protoporphyria (EPP).

EPP is a rare, life-threatening disease caused by a deficiency of the enzyme ferrochelatase, which leads to an accumulation of protoporphyrin IX. The disease is characterized by intense pain, edema, burning sensations, and in rare cases, blistering, when patients with EPP are exposed to sunlight. 

Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 (GlyT1) designed to modulate heme biosynthesis by limiting glycine uptake. In preclinical studies, bitopertin had demonstrated to significantly decrease protoporphyrin IX.

The Company is currently investigating the efficacy, safety, and tolerability of bitopertin in patients with EPP in two ongoing phase 2 studies, AURORA ( Identifier: NCT05308472) and BEACON (ACTRN12622000799752). 

“Receiving orphan drug designation for bitopertin is incredibly encouraging and validates our commitment to bring a potential new treatment to EPP patients,” said John Quisel, JD, PhD, CEO and President of Disc. “We are eagerly awaiting the results of our ongoing Phase 2 trials and look forward to collaborating with the FDA to progress bitopertin through clinical development.”


Disc Medicine receives FDA Orphan Drug designation for bitopertin for the treatment of erythropoietic protoporphyria. News release. Disc Medicine. Accessed December 27, 2022.

This article originally appeared on MPR

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