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PERHEXILINE
Perhexiline is a marketed drug that selectively blocks the muscarinic acetylcholine receptor M4, positioning it in a niche segment of the pharmaceutical market. Its key strength lies in its unique mechanism of action targeting the M4 receptor, which differentiates it from competitors such as amantadine, amiodarone, and amitriptyline. The primary risk is the key composition patent expiry in 2028, which could lead to increased competition from generics.
At a glance
| Generic name | PERHEXILINE |
|---|---|
| Drug class | perhexiline |
| Target | Carnitine O-palmitoyltransferase 1, liver isoform, Carnitine O-palmitoyltransferase 2, mitochondrial, Cytochrome P450 2D6 |
| Modality | Small molecule |
| Therapeutic area | Cardiovascular |
| Phase | FDA-approved |
| First approval | 1974 |
Approved indications
Common side effects
Key clinical trials
- Role of Perhexiline in Hypertrophic Cardiomyopathy (PHASE2)
- Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure (PHASE2)
- Efficacy, Safety, and Tolerability of Perhexiline in Subjects With Hypertrophic Cardiomyopathy and Heart Failure (PHASE3)
- Perhexiline Therapy in Heart Failure With Preserved Ejection Fraction Syndrome (PHASE2)
- Metabolic Manipulation in Chronic Heart Failure (PHASE2)
- Myocardial Protection With Perhexiline in Left Ventricular Hypertrophy (PHASE2,PHASE3)
- Perhexiline Therapy in Patients With Hypertrophic Cardiomyopathy (PHASE2)
- Metabolic Support With Perhexiline to Protect Myocardium Undergoing Coronary Artery Surgery (PHASE2,PHASE3)
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |