More than three years after receiving approval in Europe, recent Kyowa Kirin Co., Ltd. acquisition Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel) has finally won US Food and Drug Administration approval as well, for children with early-onset metachromatic leukodystrophy (MLD). Now, the task for the company is to roll the gene therapy out to a population of only a few dozen patients, with a price far in excess of what a drug pricing watchdog had expected.
The FDA approved Lenmeldy 18 March – which the European Commission approved under the brand name Libmeldy in December 2020...
