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DCR-PHXC
DCR-PHXC is an RNA interference therapeutic that silences the PHXC gene to reduce production of a disease-causing protein.
DCR-PHXC is an RNA interference therapeutic that silences the PHXC gene to reduce production of a disease-causing protein. Used for Phytanic acid storage disease (Refsum disease) or related lipid metabolism disorder.
At a glance
| Generic name | DCR-PHXC |
|---|---|
| Also known as | Nedosiran, nedosiran |
| Sponsor | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company |
| Drug class | RNA interference therapeutic |
| Target | PHXC mRNA |
| Modality | Small molecule |
| Therapeutic area | Genetic/Metabolic Disease |
| Phase | Phase 3 |
Mechanism of action
DCR-PHXC uses Dicerna's proprietary dicer-substrate RNA (dsRNA) technology to trigger RNA interference (RNAi) and degrade messenger RNA encoding the target protein. This approach aims to reduce pathogenic protein levels in patients with genetic or acquired diseases driven by PHXC overexpression or dysfunction.
Approved indications
- Phytanic acid storage disease (Refsum disease) or related lipid metabolism disorder
Common side effects
- Injection site reactions
- Transient liver enzyme elevation
- Immune activation (interferon-related)
Key clinical trials
- Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD (PHASE2)
- Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function (PHASE2)
- Long Term Extension Study in Patients With Primary Hyperoxaluria (PHASE3)
- Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria (PHASE1)
- Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients (PHASE1)
- A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- DCR-PHXC CI brief — competitive landscape report
- DCR-PHXC updates RSS · CI watch RSS
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company portfolio CI