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,4-diaminopyridine
,4-diaminopyridine is a Potassium channel blocker Small molecule drug developed by National Center for Research Resources (NCRR). It is currently in Phase 3 development for Lambert-Eaton myasthenic syndrome (LEMS), Myasthenia gravis.
3,4-diaminopyridine blocks potassium channels on nerve terminals, prolonging action potentials and increasing acetylcholine release at the neuromuscular junction.
3,4-diaminopyridine blocks potassium channels on nerve terminals, prolonging action potentials and increasing acetylcholine release at the neuromuscular junction. Used for Lambert-Eaton myasthenic syndrome (LEMS), Myasthenia gravis.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
CNS / neurology attrition
-3.0pp
CNS drugs have historically high Phase 3 failure rates (notably in Alzheimer disease + major depression).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +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 | ,4-diaminopyridine |
|---|---|
| Sponsor | National Center for Research Resources (NCRR) |
| Drug class | Potassium channel blocker |
| Target | Voltage-gated potassium channels (presynaptic) |
| Modality | Small molecule |
| Therapeutic area | Neurology |
| Phase | Phase 3 |
Mechanism of action
3,4-diaminopyridine acts as a potassium channel blocker that enhances neuromuscular transmission by prolonging the duration of the action potential at presynaptic nerve terminals. This leads to increased calcium influx and greater acetylcholine release into the synaptic cleft. The drug is used to treat disorders of neuromuscular transmission where acetylcholine release is impaired.
Approved indications
- Lambert-Eaton myasthenic syndrome (LEMS)
- Myasthenia gravis
Common side effects
- Paresthesia
- Tremor
- Abdominal pain
- Seizures
Key clinical trials
- Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism (PHASE2, PHASE3)
- Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
- Treatment Use of 3,4-Diaminopyridine
- Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) (PHASE3)
- Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia
- 3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)
- Use of 3,4-Diaminopyridine in the Treatment of Lambert-Eaton Syndrome
- Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients (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:
- ,4-diaminopyridine CI brief — competitive landscape report
- ,4-diaminopyridine updates RSS · CI watch RSS
- National Center for Research Resources (NCRR) portfolio CI
Frequently asked questions about ,4-diaminopyridine
What is ,4-diaminopyridine?
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Who makes ,4-diaminopyridine?
What drug class is ,4-diaminopyridine in?
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What does ,4-diaminopyridine target?
Related
- Drug class: All Potassium channel blocker drugs
- Target: All drugs targeting Voltage-gated potassium channels (presynaptic)
- Manufacturer: National Center for Research Resources (NCRR) — full pipeline
- Therapeutic area: All drugs in Neurology
- Indication: Drugs for Lambert-Eaton myasthenic syndrome (LEMS)
- Indication: Drugs for Myasthenia gravis
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing