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.
Orchard Will Launch Lenmeldy At Just Five Centers
Gene Therapy Is World’s Priciest Drug
Orchard, which Kyowa Kirin bought in October, gave Lenmeldy a $4.25m list price, exceeding the upper bound of about $3.9m set by ICER.
More from New Products
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
• By
The small interference RNA therapeutic can be used by patients regardless of inhibitor status.
• By
The company is planning to launch oxylanthanum carbonate for chronic kidney disease patients on dialysis with hyperphosphatemia.
• By
After a long wait for patients, Vykat has become the first drug approved for Prader-Willi syndrome symptoms, opening the market up for Soleno and future challengers.
More from Scrip
• By
Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.
• By
The four-year-old firm said it plans to advance programs toward the clinic from the funding round, which comes just over a year after signing two major pharma partnerships.
• By
A Phase III trial testing the cortisol modulator showed a benefit on PFS and OS in patients with platinum-resistant ovarian cancer.