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NI-0501
NI-0501 is a Small molecule drug developed by Swedish Orphan Biovitrum. It is currently in Phase 2 development. Also known as: Gamifant.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | NI-0501 |
|---|---|
| Also known as | Gamifant |
| Sponsor | Swedish Orphan Biovitrum |
| Modality | Small molecule |
| Phase | Phase 2 |
Approved indications
Common side effects
Key clinical trials
- Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists (PHASE1, PHASE2)
- Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study (PHASE2)
- A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients (PHASE4)
- First-in-human Study of Interferon-y PET Imaging to Assess Response to Immunotherapy (PHASE1)
- Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE (PHASE3)
- A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia (PHASE2)
- Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients (EARLY_PHASE1)
- A Study of Emapalumab for Pediatric Aplastic Anemia (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:
- NI-0501 CI brief — competitive landscape report
- NI-0501 updates RSS · CI watch RSS
- Swedish Orphan Biovitrum portfolio CI
Frequently asked questions about NI-0501
What is NI-0501?
Who makes NI-0501?
Is NI-0501 also known as anything else?
What development phase is NI-0501 in?
Related
- Manufacturer: Swedish Orphan Biovitrum — full pipeline
- Also known as: Gamifant
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing