Rising leader Mårten Steen, spoke with In Vivo about translating science into business, building precision medicine firms from the ground up and the importance of high-energy leadership.

Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."

In a series of articles, Scrip takes a look at some of the key novel products expected to reach the market in 2022 in a cross-section of therapy areas. Here, with input from Biomedtracker, are nine new products for endocrine and metabolic disorders that could make their debuts next year.

Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.