HCL saves 27 patients by restarting production of an unobtainable drug

The Hospices Civils de Lyon have cured several patients by producing a drug whose production had ceased in 2019 worldwide, in particular because of its very high cost, reports AFP.

It all started in the fall of 2020, when an immunocompromised teenager suffered from severe diarrhea after a liver transplant. An HCL biologist had then highlighted the presence of a fungus from the family of microsporidia, potentially fatal in such a case.

The only remedy was fumagillin, an antiparasitic used since the 1950s. However, at the central pharmacy of Lyon hospitals, stocks were exhausted, the production of this drug having ceased.

The active ingredient found in Hungary

Toxic and unstable, this molecule requires significant protective measures in the laboratory and storage at a temperature of -80°C. Fripharm, the HCL pharmaceutical manufacturing, research and innovation platform, is looking for it from its Chinese, Indian and European suppliers. And ends up finding, in Hungary, 300 grams of active ingredient used to produce the drug.

The material is sent to Lyon, where hospital pharmacists draw an oral suspension for the boy, who recovers in two weeks in August 2021. Since then, 26 other patients – from Lyon, Paris, Grenoble, Clermont-Ferrand, Rennes, Nantes and Bordeaux – could be treated like this.

Public or private aid called in to remedy the shortage

The shortage of fumagillin is global, underlines to AFP Professor Fabrice Pirot, coordinator of the HCL. The team therefore set out to find a start-up to sustain the production of the drug. Several candidates have come forward in France to manufacture a pilot batch, but the launch cost – around one million euros – requires external aid, public or private.

“Without this remedy, immunocompromised patients, including all transplant patients, victims of microsporidia, have almost no chance of recovering,” says Dr. Meja Rabodonirina, biologist consulted on the case of the teenager. It is unthinkable not to save patients when we know how to do it. »

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