Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Topical beremagene geperpavec, Open Label Topical Beremagene Geperpavec (B-VEC), Open Label Beremagene Geperpavec
beremagene geperpavec · 3 trials · 4 indications
Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.
The primary wound was defined as a responder wound that met either of the following conditions: * Complete wound healing on Week 22 and Week 24, or * Complete wound healing on Week 24 and Week 26. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
One wound is a responder if the reduction from baseline in wound surface is ≥90%.
Time to wound closure was defined as the time from the first treatment to Complete Wound Closure (≥90% reduction in wound surface area from baseline)
Duration of wound closure
| Arm | Type | Description |
|---|---|---|
| B-VEC | EXPERIMENTAL | Open label B-VEC topical treatment of DEB wounds. |
| Placebo | PLACEBO_COMPARATOR | Matching masked inactive topical gel |
| Topical beremagene geperpavec | EXPERIMENTAL | HSV1-COL7A1 vector (KB103) |
| Name | Type | Description |
|---|---|---|
| Open Label Topical Beremagene Geperpavec (B-VEC) | BIOLOGICAL | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
| Topical Beremagene Geperpavec | BIOLOGICAL | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
| Placebo | OTHER | Matching masked inactive topical gel |
| Placebo gel | BIOLOGICAL | Placebo gel |
Inclusion Criteria: * Willing and able to give consent/assent * Clinical diagnosis of epidermolysis bullosa * Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1. * Age: 2 months of age and older at the time of informed consent/assent * Women of childbearing age must...
Beremagene geperpavec is an investigational gene therapy being developed for the treatment of dystrophic epidermolysis bullosa, a rare inherited skin disorder. It is designed to be applied topically to the skin. The drug is currently in Phase 3 clinical development and has received an orphan drug designation from the FDA.
Beremagene geperpavec targets COL7A1, the gene encoding type VII collagen. It is an exogenous gene therapy, meaning it delivers a functional copy of the COL7A1 gene to patients' cells. This approach aims to address the underlying genetic cause of dystrophic epidermolysis bullosa, which is associated with defects in type VII collagen.
Beremagene geperpavec is being developed by Krystal Biotech, Inc., which trades on the Nasdaq under the ticker symbol KRYS. Krystal Biotech is a biotechnology company focused on gene therapies for rare diseases.
Beremagene geperpavec is in Phase 3 clinical development. It is an investigational therapy and has not been approved by the FDA. The development program includes completed Phase 1 and Phase 3 trials, and the drug has received orphan drug designation from the FDA for dystrophic epidermolysis bullosa.
Beremagene geperpavec has been studied in three completed clinical trials: NCT04917874, a Phase 3 long-term treatment study; NCT04491604, a Phase 3 efficacy and safety trial; and NCT03536143, a Phase 1/2 study. All trials enrolled patients with dystrophic epidermolysis bullosa and were conducted in the United States.
Yes, beremagene geperpavec is also known as B-VEC, which stands for beremagene geperpavec. Other names used include topical beremagene geperpavec and open label topical beremagene geperpavec. These terms refer to the same investigational gene therapy developed by Krystal Biotech for dystrophic epidermolysis bullosa.