Lenmeldy: an example of what it takes to get an ultrarare disease drug across the FDA finish line

With scores of gene therapies for ultrarare diseases languishing in pipelines, FDA’s approval of Orchard’s metachromatic leukodystrophy gene therapy offers a case study in a path forward.

The Orchard Therapeutics unit of Kyowa Kirin Co. Ltd. (Tokyo:4151) announced Monday that FDA granted approval to atidarsagene autotemcel, under the brand name Lenmeldy, based on data from 37 patients, none of whom were treated in a randomized-controlled trial. Instead, the data were pieced together from two single-arm, open-label studies and a European expanded access program. All the patients were treated at one site, the Ospedale San Raffaele in Milan, Italy, and the results were compared with natural history. ...