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.

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


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.

So­cial im­age: An­drew Harnik, AP Im­ages

Author

Zachary Brennan

managing editor, RAPS

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