FDA issues Complete Response Letter for Disc Medicine’s bitopertin
The FDA issued a Complete Response Letter for Disc Medicine’s bitopertin NDA in EPP, saying additional evidence is needed. The agency wants Phase 3 APOLLO data before deciding.
EPP
DiseaseDisease Profile
EPP in disease context refers to erythropoietic protoporphyria, a rare inherited porphyria caused by defects in heme biosynthesis leading to protoporphyrin accumulation. The disease typically presents with severe painful photosensitivity from childhood and may involve hepatobiliary complications in a subset of patients. It is one of the more common porphyrias in children.
- Category
- Inherited porphyria (heme biosynthesis disorder)
- Prevalence
- Estimated incidence is about 2 to 5 per 1,000,000 and prevalence ranges roughly from 1:75,000 to 1:200,000 in reported populations
Related News
FDA Issues Complete Response Letter for Disc Medicine's Bitopertin in EPP
Disc Medicine received a complete response letter from the FDA for bitopertin in erythropoietic protoporphyria, with the agency requesting Phase 3 APOLLO trial results before approval. The company expects to respond by mid-2027.
FDA Issues Complete Response Letter for Disc Medicine's Bitopertin in EPP
The FDA issued a Complete Response Letter for Disc Medicine's bitopertin application for erythropoietic protoporphyria, citing concerns over surrogate endpoints despite review under the accelerated approval pathway and Commissioner's National Priority Voucher pilot program.
FDA Denies Accelerated Approval for Bitopertin in EPP, Awaits Phase 3 Trial Results
The FDA issued a Complete Response Letter for bitopertin in erythropoietic protoporphyria, acknowledging the drug lowers PPIX levels but requiring Phase 3 APOLLO trial results to demonstrate clinical benefit before approval.
FDA Issues Complete Response Letter for Disc Medicine's Bitopertin in EPP Treatment
The FDA issued a Complete Response Letter for Disc Medicine's bitopertin New Drug Application for erythropoietic protoporphyria, requiring results from the ongoing Phase 3 APOLLO study before making an approval decision. Topline data is expected in Q4 2026.
Disc Medicine Receives FDA Complete Response Letter for Bitopertin EPP Treatment
Disc Medicine received a complete response letter from the FDA for bitopertin as a treatment for erythropoietic protoporphyria. The FDA acknowledged the drug lowers PPIX levels but requires evidence from the ongoing Phase 3 APOLLO study before approval.
Related Clinical Trials
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05005975 |
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) |
RECRUITING | PHASE3 |
| NCT04402489 |
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria |
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Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact |
COMPLETED |