Shots:

• The P-III trial (GEM-3) evaluating the efficacy & safety of B-VEC vs PBO in a ratio (1:1) in 31 patients aged ≥6mos. with genetically confirmed DEB for ~26wks.
• The trial met its 1EPs & showed complete wound healing was reported in 67.4% vs 21.6% @6mos. while 70.6% vs 19.7% @3mos. The therapy was found to be well tolerated with no drug-related SAEs or discontinuations due to treatment & the results were published in NEJM
• The US FDA has accepted the BLA for B-VEC while the therapy also received the FTD & RPD designation from the US FDA; RMAT for DEB and PRIME eligibility from the EMA. The company submits the MAA to the EMA and is currently working closely with the EMA through the MA validation

Ref: Globenewswire | Image: Krystal Biotech