Epidermolysis bullosa: FDA seeks to help development of new treatments
The FDA recently released guidance to help companies develop new treatments for epidermolysis bullosa (EB), which is a debilitating and often fatal rare disease.
The six-page draft guidance offers details on considerations for clinical trial design, including trial population (estimates suggest 25,000-50,000 people in the US have EB), discussion of efficacy endpoints (i.e. effects on patients’ signs or symptoms such as itching, pain, blister prevention and wound healing) and special considerations to maximize patient comfort (i.e. allowing photo or video documentation of wounds during routine dressing changes in the home).
“In EB, defective epithelial integrity in the skin leads to chronic and relapsing wounds that predispose patients to repeated infections and may result in unwanted fibrosis and deformities as well as cutaneous carcinogenesis. Skin lesions are associated with itching and pain, the latter aggravated during dressing changes,” the FDA explains.
Several EB treatments are currently making their way through companies’ pipelines.
Last month, Krystal Biotech evaluated four EB patients in a Phase II trial with its treatment KB103, finding that multiple open wounds closed after treatment, although one of the patients dropped out because the trial site was too difficult to reach. KB103 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and PRIME designation from the European Medicines Agency.
Krystal expects to begin a Phase III trial before the end of 2019.
In addition, in late May, the FDA granted an RMAT designation to Fibrocell Science’s FCX-007, which is a gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa. The company said it expects to complete data collection for the primary endpoint of a Phase III trial in the fourth quarter of 2020 and then file a biologics license application in 2021.
First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.
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