Amicus Therapeutics Advances Development of Investigational Therapy SD-101 for EPB

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Amicus Therapeutics, a company developing therapeutics for rare diseases, recently released an update of the company’s clinical trial testing SD-101 as a potential therapy for patients with epidermolysis bullosa.

The company’s investigational drug SD-101 is a skin cream designed for the treatment of skin blistering and lesions, and it has the potential to be the first approved therapy for patients with epidermolysis bullosa.

SD-101 was one of the first drugs to receive a breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of epidermolysis bullosa.

In previous Phase 2 clinical studies, SD-101 was shown to significantly improve wound closure across all major disease types.

SD-101 has been evaluated in the Essence study, a registration-directed Phase 3 clinical trial (NCT02384460). The trial’s  primary goal was the time to complete target wound closure and the proportion of patients experiencing complete closure of their target wound, both within three months.

Amicus has completed the final data collection for the primary outcome measures, and announced that more than 95 percent of the patients who completed the three-month primary treatment period were chosen to integrate an open-label extension study (NCT02670330).

In the study, participants apply SD-101 once a day to the entire body for up to 1,440 days (48 months). The study’s primary outcome is to demonstrate SD-101’s long-term safety and tolerability. Additional outcome measures include evaluating changes in body surface area regarding skin lesions and wound burden.

The date for the trial’s final data collection is set for May 2019.

According to John F. Crowley, chairman and CEO of Amicus Therapeutics, the company is committed to advance its lead clinical program in epidermolysis bullosa with SD-101 and hopes to achieve “several important milestones in the second half of this year,” Crowley said in a press release.

“I believe that today, more than ever before, Amicus is well-positioned to become a leading global biotechnology company with the potential to deliver significant benefits to people living with rare devastating diseases.” Crowley added.

Amicus also is developing therapies for other rare genetic conditions, including Fabry disease and Pompe disease.