European CHMP recommends approval of glucarpidase to reduce toxic plasma methotrexate concentration
The CHMP has recommended the granting of a marketing authorisation under exceptional circumstances for this detoxifying agent that converts methotrexate (MTX) to its inactive metabolites, for use in adults with delayed MTX elimination or at risk of MTX toxicity.
Source:
European Medicines Agency
SPS commentary:
In exceptional circumstances, an authorisation may be granted subject to certain specific obligations, to be reviewed annually. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicinal product, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.
In this case, the positive recommendation for this orphan product was based on clinically important reductions in methotrexate concentration in at least 62% of treated patients with a median reduction of > 98% in methotrexate concentration occurring within 15 minutes following glucarpidase administration, as observed in four compassionate-use, open-label multicentre studies.