Amicus Shifts Focus After EB Treatment Misses Endpoints in Phase 3

Mathew Shanley

Yesterday, it was announced that the ESSENCE study, a Phase 3 clinical trial of SD-101 for the treatment of Epidermolysis Bullosa (EB), missed its co-primary and key secondary endpoints.

When compared with a placebo, SD-101 did not show statistical significance in reducing the time taken for wounds to close in patients with EB.

EB patients have delicate, papery, thin skin that becomes so fragile, it can fall apart at the touch, much like the wings of a butterfly. This “butterfly skin” makes patients subject to blisters, easy tearing and poor healing of wounds. The most severe iteration of the disorder, recessive dystrophic EB is due to a mutation in the COL7A1 gene which is responsible for encoding collagen VII.

There are currently no approved drugs for the condition. At present, standard of care entails the bandaging and cleaning of open wounds to prevent infection and pain management.

Amicus, the developer of the drug, has made clear its plan to proceed without any additional investments in further clinical studies.

“In keeping with our Amicus mission, we have a strong commitment to the EB community and will work closely with investigators and other leading experts to understand and to share these data. We would like to sincerely thank the patients, families, clinical investigators, regulators and our Amicus team involved in this EB program,” said Amicus CEO John Crowley in a press release.

“In seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail. But we would rather be the first to fail, than the last to try. Our vision at Amicus remains steadfast and is focused on building a leading global biotechnology company that delivers significant benefits for people living with rare diseases.”

The company has stated its intention to share an analysis of data from the ESSENCE study with key stakeholders throughout the EB community as it shifts its focus back to gaining approval for Migalastat for the treatment of Fabry.

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