Accelerated approval for patients with microsatellite instability-high or mismatch repair deficient solid tumors is the first time the US regulatory agency has granted an indication that does not specify the location of the tumor – a change that precision-medicine researchers have been eager to make.

Accelerated approval for patients with microsatellite instability-high or mismatch repair deficient solid tumors is the first time the US FDA has granted an indication that does not specify the location of the tumor – a change precision medicine researchers have been eager to make.

With FDA’s blessing, Stealth BioTherapeutics is working toward a pivotal trial that would address an underlying cause, mitochondrial myopathy, of roughly 270 very rare diseases, potentially leading to an aggregate approval in all of those unmet medical needs.