Propionic acidemia is an inherited organic acidemia caused by propionyl-CoA carboxylase deficiency, leading to toxic metabolite accumulation and recurrent metabolic decompensation.
Moderna partners with Recordati on propionic acidemia therapy for up to $160 million, while FDA Commissioner defends mRNA vaccine support following funding cuts and regulatory challenges.
Moderna reported Q4 2025 revenue of $700 million and full-year revenue of $1.9 billion, beating estimates while cutting operating expenses by $2.2 billion. The FDA refused to file the company's seasonal flu vaccine application.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05769621 |
A Retrospective Study to Characterize Participants With Propionic Acidemia |
RECRUITING | |
| NCT05687474 |
Baby Detect : Genomic Newborn Screening |
COMPLETED | |
| NCT05438485 |
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia |
TERMINATED | |
| NCT05130437 |
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study |
RECRUITING | PHASE1/PHASE2 |
| NCT05040178 |
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics |
RECRUITING | |
| NCT04836494 |
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia |
TERMINATED | PHASE1 |
| NCT04732429 |
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia |
TERMINATED | PHASE2 |
| NCT04176523 |
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach |
RECRUITING | |
| NCT04159103 |
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia |
RECRUITING | PHASE1/PHASE2 |
| NCT03655223 |
Early Check: Expanded Screening in Newborns |
ENROLLING_BY_INVITATION |
