This Thursday, January 29, the independent medical journal Prescrire distinguished Orphacol (cholic acid), a French drug intended to treat a rare genetic disease of liver which mainly affects children. This genetic disease involves “rare hereditary deficits in the synthesis of primary bile acids” and is “most often fatal”, the review states.
The “golden pill” was therefore awarded to Orphacol, an award that the magazine had not awarded since 2007.
This annual distinction rewards a drug that brings “real therapeutic progress” for patients. For lack of new truly innovative drugs, the journal had refrained from awarding this prize for the previous 8 years.
The cholic acid drug “greatly increases the life expectancy” of children, “provided treatment is started early,” the review says.
Long used in France, Orphacol had however undergone many modifications before being able to receive a marketing authorization in 2013.
The role of cholic acid in the treatment of certain hereditary bile acid deficits was discovered in 1993 by the Assistance Publique des Hospitals de Paris (APHP), which then sold its operating rights to the French laboratory CTRS in 2007. The latter had registered cholic acid under the Orphacol brand and obtained a temporary authorization for use in France. However, the laboratory had to fight for several years with the European Commission before winning its case with the General Court of the European Union in Luxembourg, in 2013.
Other drugs are included in the “Honor Roll”
But although he obtained the “Golden Pill”, this drug is not the only one to have obtained the considerations of the Prescribe magazine. It also added to its “Roll of Honor” three drugs bringing “a clear improvement for certain patients compared to the therapeutic means already available”. These are Glivec (the active substance of which is imatinib) to treat acute lymphoblastic leukemia in children, Malacef (containing intravenous artesunate) prescribed for severe attacks of malaria and Solvaldi (treatment with sofosbuvir), the famous and very expensive drug to treatHepatitis C.
Prescrire specifies, however, that in the case of the latter, “uncertainties surround its long-term clinical efficacy and its adverse effects”, while estimating that “its exorbitant price, disconnected from research and manufacturing costs endangers the insurance united. “
After several years without major breakthroughs in the world of medicine, the journal welcomes this 2015 ranking, which it describes as “good vintage”.
“Let’s hope for patients that this year will not be just an exception but the start of a lasting trend” can be read on the journal’s website.
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