The U.S. Food and Drug Administration has reversed another of its drug rejections, allowing gene therapy developer Regenxbio to resubmit its treatment, according to BioPharma Dive.
The move is part of a broader pattern. BioPharma Dive reports that the agency is working through a backlog of rejections issued during the tenure of FDA official Makary — decisions that, in several cases, appeared to contradict agreements the agency had previously struck with drugmakers. Regenxbio's resubmission marks "another step" in that cleanup, the outlet says.
The therapy in question targets Hunter syndrome, a rare genetic disorder. Because Regenxbio is being permitted to resubmit rather than start over, the reversal suggests the original rejection is being treated as inconsistent with what the company had been led to expect.
Beyond the specifics of one company's filing, the episode points to instability in how recent regulatory decisions were made. When a rejection is walked back because it clashed with an earlier understanding between the FDA and a manufacturer, it raises questions about the consistency of the review process during that period — and about how many other rejected drugs may follow the same path back to consideration.
Few further details are available from the source summary alone, including the timeline for resubmission or the FDA's reasoning.
Why it matters: For patients with rare diseases and the companies developing treatments for them, predictable and consistent FDA decision-making is the difference between a therapy reaching the market and being shelved — and this reversal signals the agency is now revisiting a string of contested calls.