Rimeporide Explained

Iupac Name:N-(2-methyl-4,5-bis(methylsulfonyl)benzoyl)guanidine
Legal Status:Experimental
Cas Number:187870-78-6
Atc Prefix:none
Pubchem:9799487
Chemspiderid:7975252
Chembl:2107802
Unii:QH6B4V5743
C:11
H:15
N:3
O:5
S:2
Smiles:Cc1cc(c(cc1C(=O)NC(=N)N)S(=O)(=O)C)S(=O)(=O)C
Stdinchi: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)
Stdinchikey:GROMEQPXDKRRIE-UHFFFAOYSA-N

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

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.

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 that aims at repositioning drugs for rare diseases., the substance has demonstrated efficacy in several animal models of Duchenne muscular dystrophy.[1]

It has also been recently tested in young boys with Duchenne muscular Dystrophy aged 6 to 11 years.[2]

See also

Other drugs for Duchenne muscular dystrophy

[3]

Notes and References

  1. Ghaleh B, Barthélemy I, Wojcik J, Sambin L, Bizé A, Hittinger L, Tran TD, Thomé FP, Blot S, Su JB . Protective effects of rimeporide on left ventricular function in golden retriever muscular dystrophy dogs . International Journal of Cardiology . 312 . 89–95 . August 2020 . 32199683 . 10.1016/j.ijcard.2020.03.031 . 214617920 .
  2. Previtali SC, Gidaro T, Díaz-Manera J, Zambon A, Carnesecchi S, Roux-Lombard P, Spitali P, Signorelli M, Szigyarto CA, Johansson C, Gray J, Labolle D, Porte Thomé F, Pitchforth J, Domingos J, Muntoni F . Rimeporide as a first- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy . Pharmacological Research . 159 . 104999 . September 2020 . 32535224 . 7482441 . 10.1016/j.phrs.2020.104999 .
  3. Ghaleh B, Barthélemy I, Wojcik J, Sambin L, Bizé A, Hittinger L, Tran TD, Thomé FP, Blot S, Su JB . 6 . Protective effects of rimeporide on left ventricular function in golden retriever muscular dystrophy dogs . International Journal of Cardiology . 312 . 89–95 . August 2020 . 32199683 . 10.1016/j.ijcard.2020.03.031 . 214617920 .