Epidermolysis Bullosa: FDA Seeks to Help Development of New Treatments

Regulatory NewsRegulatory News | 08 July 2019 |  By 

The US Food and Drug Administration (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The six-page draft guidance offers details on considerations for clinical trial design, including trial population (estimates suggest 25,000-50,000 people in the US have EB), discussion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,” FDA explains.

Several EB treatments are currently making their way through companies’ pipelines.

Last month, Krystal Biotech evaluated four EB patients in a Phase 2 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 FDA and PRIME designation from the European Medicines Agency.

Krystal expects to begin a Phase 3 trial before the end of 2019.

In addition, in late May, 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 (RDEB). The company said it expects to complete data collection for the primary endpoint of a Phase 3 trial in the fourth quarter of 2020 and then file a biologics license application in 2021.

And Abeona Therapeutics won RMAT designation in January 2018 for its gene therapy to treat RDEB. A Phase 1/2 clinical trial met its primary safety and efficacy endpoints, the company said. Enrollment in a Phase 3 study is expected in mid-2019.

Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations: Guidance for Industry

Updated 7/8/19 with information on Abeona Therapeutics.


© 2023 Regulatory Affairs Professionals Society.

Tags: EB, Fibrocell, Krystal

Discover more of what matters to you

No taxonomy