Rimeporide is an experimental

drug A drug is any chemical substance other than a nutrient or an essential dietary ingredient, which, when administered to a living organism, produces a biological effect. Consumption of drugs can be via insufflation (medicine), inhalation, drug i ...

for the treatment of

Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. The onset of muscle weakness typically begins around age four, with rapid progression. Initially, muscle loss occurs in the thighs and pe ...

, being developed by the EspeRare foundation. it has been granted

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

status by the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

Mechanism of action

The substance blocks an ion pump called

sodium–hydrogen antiporter 1 The sodium-hydrogen antiporter 1 (NHE-1) also known as sodium/hydrogen exchanger 1 or SLC9A1 (SoLute Carrier family 9A1) is an isoform of sodium–hydrogen antiporter that in humans is encoded by the ''SLC9A1'' gene. Function The Na+/H+ antip ...

. While the exact mechanism is unknown, it is speculated that inhibition of this pump reduces pH,

sodium Sodium is a chemical element; it has Symbol (chemistry), symbol Na (from Neo-Latin ) and atomic number 11. It is a soft, silvery-white, highly reactive metal. Sodium is an alkali metal, being in group 1 element, group 1 of the peri ...

and

calcium Calcium is a chemical element; it has symbol Ca and atomic number 20. As an alkaline earth metal, calcium is a reactive metal that forms a dark oxide-nitride layer when exposed to air. Its physical and chemical properties are most similar to it ...

overload in cells of patients with Duchenne muscular dystrophy.

History

Rimeporide was designed as a treatment for

chronic heart failure Heart failure (HF), also known as congestive heart failure (CHF), is a syndrome caused by an impairment in the heart's ability to fill with and pump blood. Although symptoms vary based on which side of the heart is affected, HF typically pre ...

. It was tested in seven Phase I studies

clinical trial Clinical trials are prospective biomedical or behavioral research studies on human subject research, human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel v ...

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

References

{{reflist, 35em, refs= {{Cite journal, journal=Österreichische Apothekerzeitung, volume=69, issue=11, page=12, title=Neue Wirkstoffe – Rimeporid , vauthors = Spreitzer H , date=26 May 2015, language=German {{cite web, url=http://esperare.org/en/our-mission-and-vision, title=Our mission and vision, publisher=EspeRare, access-date=23 July 2015 {{cite news, url=http://esperare.org/en/news/esperares-rimeporide-receives-orphan-drug-designation-duchenne-muscular-dystrophy, title=EspeRare's Rimeporide receives Orphan Drug Designation in Duchenne Muscular Dystrophy, publisher=EspeRare, date=4 May 2015 Muscular dystrophy Orphan drugs Acylguanidines Benzosulfones Benzamides

Mechanism of action

History

See also

Other drugs for Duchenne muscular dystrophy

References