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Kynamro (MIPOMERSEN)
Kynamro (Mipomersen) is an antisense oligonucleotide developed by Genzyme Corp and currently owned by Kastle Therapies LLC. It is a small molecule modality approved by the FDA in 2013 for the treatment of familial hypercholesterolemia in homozygous patients. Kynamro works by targeting a specific gene to reduce the production of apolipoprotein B, a key component of low-density lipoprotein (LDL) cholesterol. The drug is patented and has no generic manufacturers. Key safety considerations include liver toxicity and elevations in liver enzymes.
At a glance
| Generic name | MIPOMERSEN |
|---|---|
| Sponsor | Kastle Theraps Llc |
| Drug class | Antisense Oligonucleotide |
| Modality | Oligonucleotide |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2013 |
Approved indications
- Familial hypercholesterolemia - homozygous
Common side effects
Key clinical trials
- A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol (PHASE3)
- Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration (PHASE2)
- Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia (PHASE2)
- Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia (PHASE3)
- An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia (PHASE3)
- Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease (PHASE3)
- Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who Are Not on Apheresis (PHASE3)
- Safety and Efficacy of Mipomersen (ISIS 301012) As Add-on Therapy in High Risk Hypercholesterolemic Patients (PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
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 |
| FDA Orange Book | Patents + exclusivity |