The Japanese pharma has taken over commercialization of the rare disease drug from Ultragenyx under a planned transition and as part of a broader growth strategy in North America.

SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.

After shutting down its trial for a leukemia conditioning agent due to a patient death, Magenta’s review of strategic alternatives concludes with a combination with complement inhibitor-focused Dianthus.

Quick takes on Ultragenyx’s CEO lobbing the US FDA for rare disease policy changes, the agency naming a new deputy assistant commissioner for media affairs, the former CEO of AAM starting a consulting firm, and more.