Rimeporide

Chemical compound From Wikipedia, the free encyclopedia

Rimeporide is an experimental drug for the treatment of Duchenne muscular dystrophy, being developed by the EspeRare foundation.[1] it has been granted orphan drug status by the European Medicines Agency.[2]

ATC code
  • none
Legal status
  • Experimental
Quick facts Clinical data, ATC code ...
Rimeporide
Clinical data
ATC code
  • none
Legal status
Legal status
  • Experimental
Identifiers
  • N-(2-methyl-4,5-bis(methylsulfonyl)benzoyl)guanidine
CAS Number
PubChem CID
ChemSpider
UNII
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC11H15N3O5S2
Molar mass333.38 g·mol−1
3D model (JSmol)
  • Cc1cc(c(cc1C(=O)NC(=N)N)S(=O)(=O)C)S(=O)(=O)C
  • InChI=1S/C11H15N3O5S2/c1-6-4-8(20(2,16)17)9(21(3,18)19)5-7(6)10(15)14-11(12)13/h4-5H,1-3H3,(H4,12,13,14,15)
  • Key:GROMEQPXDKRRIE-UHFFFAOYSA-N
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Mechanism of action

The substance blocks an ion pump called sodium–hydrogen antiporter 1. While the exact mechanism is unknown, it is speculated that inhibition of this pump reduces pH, sodium and calcium overload in cells of patients with Duchenne muscular dystrophy.[1]

History

Rimeporide was designed as a treatment for chronic heart failure. It was tested in seven Phase I studies clinical trials in patients with congestive heart failure and some degree of renal insufficiency. Subsequently, the drug was licensed to EspeRare, a Swiss nonprofit organisation[3] that aims at repositioning drugs for rare diseases. As of May 2015, the substance has demonstrated efficacy in several animal models of Duchenne muscular dystrophy.[4]

It has also been recently tested in young boys with Duchenne muscular Dystrophy aged 6 to 11 years.[5]

See also

Other drugs for Duchenne muscular dystrophy

[6]

References

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