Commitment of trial participants in UK has far reaching benefits
The FDA, the American regulatory body, has this week approved everolimus for the treatment of AML (angiomyolipoma) in TSC adults – the first medicine to be made available for the renal lesions which affect up to 80% of people with the condition.
The fast track FDA approval was granted based on initial results from the worldwide EXIST-2 clinical trial, which includes UK patients – so you have made a vital contribution to this major step forward.
The initial results, currently being written up for publication, demonstrated that 42% of patients taking the study drug showed shrinkage of AML, compared to none of those taking placebo. Approval is conditional on continuation of the 4 year trial so that important follow-up data can be collected.
The announcement means that everolimus now has a US license for the treatment of 2 of the most debilitating aspects of TSC - SEGA and AML – with an active research program for its use in other TSC manifestations.
In the UK, everolimus is currently licensed only for SEGA, but there are multiple applications in the pipeline for licensing for AML outside the US. US approval for everolimus for AML is excellent news and increases our optimism that it will become available for UK AML patients in due course.
Posted on 28th April, 2012