mipomersen
Trade name: kynamro
Nucleic acidapprovedOrphan Drug FDA
Approved
Jan 29, 2013
Mipomersen is a withdrawn drug that was used to treat homozygous familial hypercholesterolemia and is administered by subcutaneous injection. There is a serious risk of liver damage from this drug and it was only prescribed in the context of a risk management plan. — Wikipedia
Clinical trial activity
19 trials · 4 clinical orgs · 2 marketing orgs
Phase 1
5
Phase 2
14
Phase 3
7
Phase 4
0
Earliest trial started Aug 1, 2005 (NCT00216463)
Timeline
2000s
- Jan 1, 2005
Kastle Therapeutics, LLC — Earliest Phase 2 Sponsor(trial)
- May 23, 2006
Orphan Drug Designation
- Jan 1, 2007
Kastle Therapeutics, LLC — Earliest Phase 3 Sponsor(trial)
2010s
- Jan 1, 2010
Kastle Therapeutics, LLC — Earliest Phase 1 Sponsor(trial)
- Jan 29, 2013
Genzyme — Earliest FDA Approval
- Jan 29, 2013
Genzyme — NDA Organization
- Jan 29, 2013
Kastle Therapeutics, LLC — NDA Secondary Org
Indications
Approved for
Studied for
- Atherosclerosis · Phase 3
- Congenital Abnormalities · Phase 3
- Coronary Artery Disease · Phase 3
- Coronary Disease · Phase 3
- Dyslipidemias · Phase 3
- Genetic Diseases, Inborn · Phase 3
- Healthy Volunteers · Phase 1
- Heterozygote · Phase 3
- Homozygous Familial Hypercholesterolemia · Phase 3
- Hypercholesterolemia · Phase 3
- Hyperlipidemias · Phase 3
- Hyperlipoproteinemias · Phase 3
- Hypobetalipoproteinemias · Phase 2
- Hypolipoproteinemias · Phase 2
- Infant, Newborn, Diseases · Phase 3
- Lipid Metabolism Disorders · Phase 3
- Lipid Metabolism, Inborn Errors · Phase 3
- Metabolic Diseases · Phase 3
- Metabolism, Inborn Errors · Phase 3
Mechanism of action
- Apo-B 100 mRNAANTISENSE INHIBITOR
Apo-B 100 mRNA antisense inhibitor
It's an antisense oligonucleotide against apo b-100, the principal apoliprotein of LDL and its metabolic precursor VLDL
- Apolipoprotein B-100INHIBITOR
APOB inhibitor
Approval history
- approvedJan 29, 2013
Chemistry & pharmacology
Loading structure…
SMILES
COCCO[C@@H]1[C@H](SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)[C@H](OCCOC)[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)[C@H](OCCOC)[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(N)ncnc43)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(=O)[nH]c(N)nc43)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(=O)[nH]c(N)nc43)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(=O)[nH]c(N)nc43)[C@H](OCCOC)[C@@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cc(C)c(N)nc3=O)[C@@H](OCCOC)[C@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cnc4c(N)ncnc43)[C@@H](OCCOC)[C@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cc(C)c(N)nc3=O)[C@@H](OCCOC)[C@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cc(C)c(N)nc3=O)[C@@H](OCCOC)[C@H]2O)[C@@H](COP(=O)(O)S[C@H]2[C@@H](OCCOC)[C@H](n3cc(C)c(N)nc3=O)O[C@@H]2COP(=O)(O)S[C@H]2[C@@H](OCCOC)[C@H](n3cnc4c(=O)[nH]c(N)nc43)O[C@@H]2CO)O[C@H]1n1cc(C)c(N)nc1=O- Mol. weight
- 7177.25 g/mol
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Discontinued
- Multi-specific
- No
Oral
No
Parenteral
Yes
Topical
No
Black box warningFirst in class
Sources
- ChEMBLCHEMBL2219536 ↗
- WikipediaMipomersen ↗
- NCATS9GJ8S4GU0M ↗
- ChEMBLCHEMBL502097 ↗
Also known as
- isis301012
- isis 301012
- isis 301012
- isis 301012
- isis 301012
- isis-301012
- isis-301012
- isis-301012
- isis-301012
- kynamro
- kynamro
- kynamro
- kynamro
- kynamro
- mipomersen
- mipomersen
- mipomersen
- mipomersen
- mipomersen
- mipomersen
- mipomersen
- mipomersen
- mipomersen
- mipomersen sodium
- mipomersen sodium
- mipomersen sodium
- mipomersen sodium
- mipomersen sodium
- mipomersen sodium
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT01598948 | Phase 3 | Aug 1, 2012 | Ludwig Maximilians University |
| NCT01475825 | Phase 3 | Dec 1, 2011 | Genzyme, Kastle Therapeutics, LLC |
| NCT01414881 | Phase 1 | Sep 1, 2011 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT01299298 | Phase 1 | Jan 1, 2011 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT01133366 | Phase 1 | May 1, 2010 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT01090661 | Phase 1 | Mar 1, 2010 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT01061814 | Phase 1 | Jan 1, 2010 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00794664 | Phase 3 | Jan 1, 2009 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00770146 | Phase 3 | Nov 1, 2008 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00707746 | Phase 2 | Oct 1, 2008 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00706849 | Phase 3 | Jul 1, 2008 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00694109 | Phase 3 | Apr 1, 2008 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00607373 | Phase 3 | Jul 1, 2007 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00477594 | Phase 2 | May 1, 2007 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00362180 | Phase 2 | Jul 1, 2006 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00281008 | Phase 2 | Feb 1, 2006 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00280995 | Phase 2 | Jan 1, 2006 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00231569 | Phase 2 | Sep 1, 2005 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
| NCT00216463 | Phase 2 | Aug 1, 2005 | Genzyme, Ionis Pharmaceuticals, Inc., Kastle Therapeutics, LLC |
Showing 19 of 19 trials
Page 1 / 1
Organizations
Research & Development (4)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Genzyme | For profit | 18 | 0 | 3 | 2005 |
| Kastle Therapeutics, LLC | For profit | 18 | 18 | 3 | 2005 |
| Ionis Pharmaceuticals, Inc. | For profit | 17 | 0 | 3 | 2005 |
| Ludwig Maximilians University | Academic/Hospital | 1 | 1 | 1 | 2012 |
4 organizations
Page 1 / 1
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Genzyme | For profit | NDA | Jan 29, 2013 |
| Kastle Therapeutics, LLC | For profit | NDA2 | Jan 29, 2013 |