A drug used to treat children with a rare genetic condition who are unable to produce bile and risk fatal liver failure has received international recognition for its potential to prolong life.
Cholic acid (Orphacol) was named “Pilule d’Or” or “Golden Pill” by the French medical journal Prescrire in its annual awards, which recognise new drugs or new indications for drugs already on the market that represent “tangible progress” for patients. It is the first time in six years that the top Golden Pill honour has been made.
The journal says the drug, licensed across the European Union in 2013, has the potential to prolong life considerably if given early enough to children with bile acid deficiency. Previously, it was only available on prescription in France on compassionate grounds.
Prescrire has added three other drugs to its 2014 honours board. They are imatinib (Glivec), which it says significantly prolongs survival in children suffering from a rare form of acute lymphoblastic leukaemia; intravenous artesunate (Malacef), a treatment for severe attacks of malaria and sofosbuvir (Sovaldi), an antiviral used in chronic hepatitis C.
The journal also highlighted as “noteworthy” the addition of coating to sodium phenylbutyrate granules (Pheburane). The coating disguises their unpleasant taste which interfered with compliance in the treatment of patients with a rare urea synthesis disorder.