DRUGapprovedsmall molecule
amifampridine
Brand names: Firdapse, Ruzurgi
Mechanism
3,4-diaminopyridine. Blocks presynaptic Kv channels, prolongs action potential, enhances ACh release. Off-label NMJ symptomatic therapy in SMA.
Approved indications
LEMS
Related claims (11)
| Type | Predicate | Conf | Source |
|---|---|---|---|
| drug efficacy | Amifampridine treatment led to a statistically significant improvement in HFMSE (clinical trial result) | 59% | 35763114 |
| motor function | Amifampridine Phosphate improved Hammersmith Functional Motor Scale Expanded (HFMSE) scores in ambulatory patients with SMA Type 3 (clinical trial result) | 59% | NCT03781479_results |
| drug efficacy | Amifampridine treatment led to statistically significant improvement in HFMSE compared to placebo in ambulatory SMA Type 3 patients (clinical trial result). | 56% | PMID:35763114 |
| safety | Transient paresthesia occurred in 33.3% of amifampridine-treated patients as the only amifampridine-related adverse event (clinical trial result). | 56% | PMID:35763114 |
| drug efficacy | Amifampridine was effective in treating ambulatory SMA type 3 patients (clinical trial result) | 53% | 35763114 |
| safety | Amifampridine Phosphate was associated with adverse events in ambulatory patients with SMA Type 3 (clinical trial result) | 53% | NCT03781479_results |
| drug efficacy | Amifampridine Phosphate demonstrated efficacy in ambulatory patients with SMA Type 3 (clinical trial result) | 53% | NCT03781479_results |
| safety | No serious adverse events were reported with amifampridine in ambulatory SMA Type 3 patients (clinical trial result). | 50% | PMID:35763114 |
| drug efficacy | Amifampridine did not show statistically significant improvement in secondary outcomes (timed tests and quality of life) in ambulatory SMA Type 3 patients (clinical trial result). | 50% | PMID:35763114 |
| safety | Amifampridine was safe in treating ambulatory SMA type 3 patients (clinical trial result) | 45% | 35763114 |
| protein interaction | Amifampridine prolongs presynaptic NMJ terminal depolarization, enhancing neuromuscular transmission (mechanism of action). | 42% | PMID:35763114 |
Off-Target Findings (204)
T1: 5T3: 191T4: 8
| Target | Role | Tier | Boltz-2 iPTM | Chai-1 iPTM | Verdict |
|---|---|---|---|---|---|
| PAK4kinase | SMA | T1 | 0.952 | — | Primary SMA hit |
| STMN1 | SMA | T1 | 0.721 | — | Primary SMA hit |
| CHRNGion_channel | SMA | T1 | 0.458 | — | Primary SMA hit |
| GAP43 | SMA | T1 | 0.428 | — | Primary SMA hit |
| NCALD | SMA | T1 | 0.404 | — | Primary SMA hit |
| PTK6kinase | OFF-TARGET | T3 | 0.987 | — | Selectivity flag (off-target substrate) |
| PRKXkinase | OFF-TARGET | T3 | 0.984 | — | Selectivity flag (off-target substrate) |
| BTKkinase | OFF-TARGET | T3 | 0.983 | — | Selectivity flag (off-target substrate) |
| MAPK10kinase | OFF-TARGET | T3 | 0.982 | — | Selectivity flag (off-target substrate) |
| TGFBR1kinase | SMA | T3 | 0.979 | — | Selectivity flag (off-target substrate) |
| CDK5kinase | OFF-TARGET | T3 | 0.978 | — | Selectivity flag (off-target substrate) |
| PDPK1kinase | OFF-TARGET | T3 | 0.977 | — | Selectivity flag (off-target substrate) |
| RIPK2kinase | OFF-TARGET | T3 | 0.976 | — | Selectivity flag (off-target substrate) |
| TECkinase | OFF-TARGET | T3 | 0.975 | — | Selectivity flag (off-target substrate) |
| AURKCkinase | OFF-TARGET | T3 | 0.974 | — | Selectivity flag (off-target substrate) |
| GRK5kinase | OFF-TARGET | T3 | 0.972 | — | Selectivity flag (off-target substrate) |
| IKBKBkinase | OFF-TARGET | T3 | 0.972 | — | Selectivity flag (off-target substrate) |
| GRK1kinase | OFF-TARGET | T3 | 0.970 | — | Selectivity flag (off-target substrate) |
| CSKkinase | OFF-TARGET | T3 | 0.969 | — | Selectivity flag (off-target substrate) |
| PRKG1kinase | OFF-TARGET | T3 | 0.969 | — | Selectivity flag (off-target substrate) |
| ITKkinase | OFF-TARGET | T3 | 0.968 | — | Selectivity flag (off-target substrate) |
| CDKL1kinase | OFF-TARGET | T3 | 0.967 | — | Selectivity flag (off-target substrate) |
| HTR5Agpcr | OFF-TARGET | T3 | 0.964 | — | Selectivity flag (off-target substrate) |
| CHUKkinase | OFF-TARGET | T3 | 0.964 | — | Selectivity flag (off-target substrate) |
| CDK7kinase | OFF-TARGET | T3 | 0.964 | — | Selectivity flag (off-target substrate) |
| BRAFkinase | OFF-TARGET | T3 | 0.963 | — | Selectivity flag (off-target substrate) |
| CAMK1Dkinase | OFF-TARGET | T3 | 0.962 | — | Selectivity flag (off-target substrate) |
| PAK2kinase | OFF-TARGET | T3 | 0.961 | — | Selectivity flag (off-target substrate) |
| HRH2gpcr | OFF-TARGET | T3 | 0.961 | — | Selectivity flag (off-target substrate) |
| NEK3kinase | OFF-TARGET | T3 | 0.960 | — | Selectivity flag (off-target substrate) |
| AMHR2kinase | OFF-TARGET | T3 | 0.960 | — | Selectivity flag (off-target substrate) |
| FGFR4kinase | OFF-TARGET | T3 | 0.959 | — | Selectivity flag (off-target substrate) |
| CSNK1Dkinase | OFF-TARGET | T3 | 0.959 | — | Selectivity flag (off-target substrate) |
| MARK4kinase | OFF-TARGET | T3 | 0.959 | — | Selectivity flag (off-target substrate) |
| DRD3gpcr | OFF-TARGET | T3 | 0.958 | — | Selectivity flag (off-target substrate) |
| MAP3K11kinase | OFF-TARGET | T3 | 0.957 | — | Selectivity flag (off-target substrate) |
| FLT3kinase | OFF-TARGET | T3 | 0.956 | — | Selectivity flag (off-target substrate) |
| CHRM5gpcr | OFF-TARGET | T3 | 0.953 | — | Selectivity flag (off-target substrate) |
| MAST4kinase | OFF-TARGET | T3 | 0.952 | — | Selectivity flag (off-target substrate) |
| PTK2kinase | OFF-TARGET | T3 | 0.952 | — | Selectivity flag (off-target substrate) |
| MAST3kinase | OFF-TARGET | T3 | 0.952 | — | Selectivity flag (off-target substrate) |
| ADRA1Dgpcr | OFF-TARGET | T3 | 0.951 | — | Selectivity flag (off-target substrate) |
| STK4kinase | OFF-TARGET | T3 | 0.951 | — | Selectivity flag (off-target substrate) |
| ADRA2Cgpcr | OFF-TARGET | T3 | 0.950 | — | Selectivity flag (off-target substrate) |
| ADRA2Bgpcr | OFF-TARGET | T3 | 0.949 | — | Selectivity flag (off-target substrate) |
| MAPK6kinase | OFF-TARGET | T3 | 0.949 | — | Selectivity flag (off-target substrate) |
| AAK1kinase | OFF-TARGET | T3 | 0.949 | — | Selectivity flag (off-target substrate) |
| HRH4gpcr | OFF-TARGET | T3 | 0.948 | — | Selectivity flag (off-target substrate) |
| ARAFkinase | OFF-TARGET | T3 | 0.947 | — | Selectivity flag (off-target substrate) |
| MAPK7kinase | OFF-TARGET | T3 | 0.947 | — | Selectivity flag (off-target substrate) |
Showing top 50 of 204 targets.