Disc Medicine, Inc. (Disc), A Clinical-Stage Biopharmaceutical Company, Announced That The Us Food And Drug Administration (Fda) Granted Orphan Drug Designation To Bitopertin For The Treatment Of Erythropoietic Protoporphyria (Epp). Bitopertin Is An Investigational Oral, Selective Inhibitor Of Glycine Transporter 1 (Glyt1) Designed To Modulate Heme Biosynthesis, And Has Been Shown In Preclinical Studies To Reduce Accumulation Of Protoporphyrin Ix (Ppix), The Toxic Metabolite That Causes Disease Pathology In Epp Patients. It Is Currently Being Studied In Two Ongoing Phase 2 Studies In Epp, Aurora (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, J.D., Ph.D., Chief Executive Officer 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." Fda Orphan Drug Designation May Be Granted To Investigational Drugs Or Biological Products Which Show Promise In Treating Rare Medical Diseases Or Conditions That Affect Fewer Than 200,000 People In The United States. By Receiving Orphan Drug Designation, Bitopertin Can Benefit From Certain Development Incentives And Seven Years Of Market Exclusivity, Subject To Regulatory Approval. Epp Is A Rare, Debilitating And Potentially Life-Threatening Diseases Caused By Mutations That Affect Heme Biosynthesis, Resulting In The Accumulation Of A Toxic, Photoactive Intermediate, Ppix. This Causes Severe Reactions When Patients Are Exposed To Sunlight, Characterized By Excruciating Pain, Edema, Burning Sensations And Potential Blistering And Disfigurement. Ppix Also Accumulates In The Hepatobiliary System And Can Result In Complications Including Gallstones, Cholestasis, And Liver Damage In 20-30% Of Patients And In Extreme Cases Liver Failure. Current Standard Of Care Involves Extreme Measures To Avoid Sunlight, Including Restricting Outdoor Activities To Night Time, Use Of Protective Clothing And Opaque Shields, And Pain Management. This Has A Significant Impact On The Psychosocial Development, Quality Of Life, And Daily Activities Of Patients, Particularly In Young Children And Families. There Is Currently No Cure For Epp And Only One Fda-Approved Therapy, A Surgically Implanted Synthetic Hormone Designed To Stimulate Melanin Production Called Scenesse (Afamelanotide). Bitopertin Is A Clinical-Stage, Orally Administered Inhibitor Of Glyt1 That Is Designed To Modulate Heme Biosynthesis. Glyt1 Is A Membrane Transporter Expressed On Developing Red Blood Cells And Is Required To Supply Sufficient Glycine For Heme Biosynthesis And Support Erythropoiesis. The Safety Profile And Effects Of Bitopertin On Heme Biosynthesis Were Previously Established In A Comprehensive Clinical Program Comprising Over 4,000 Individuals Across Multiple Clinical Studies. Disc Is Planning To Develop Bitopertin As A Potential Treatment For A Range Of Hematologic Diseases Beginning With Epp And X-Linked Protoporphyria (Xlp). In Preclinical Models Of Epp And Xlp, Bitopertin Was Shown To Significantly Decrease Ppix, A Toxic Intermediate Of Heme Biosynthesis Which Is The Underlying Cause Of The Disease. Bitopertin Is An Experimental Agent And Is Not Approved For Use As A Therapy In Any Jurisdiction Worldwide. Disc Obtained Global Rights To Bitopertin Under A License Agreement From Roche In May 2021. Disc Is A Clinical-Stage Biopharmaceutical Company That Is Dedicated To Transforming The Lives Of Patients With Hematologic Disorders.
