ICX-RHY

Unassigned

New Medicines

Vavelta · Recessive dystrophic epidermolysis bullosa (RCEB) - second-line

Information

Vavelta
Advanced therapy medicinal product (ATMP)
Ember Therapeutics
Ember Therapeutics

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
None
Yes
Dec 19 · No longer listed in Ember Therapeutics pipeline [8].
Nov 18 · Vavelta is listed as PII in the Ember Therapeutics pipeline [7].
Mar 16 · Ember Therapeutics has acquired Vavelta from Intercytex and is planning to resume clinical development [6]
Feb 16 · Intercytex suspended development because of insufficient funds [6]
Mar 15 · PII trial ongoing [3]
Aug 13 · ISRCTN67757229 (ICX-DERBY-2) has completed and is no longer recruiting, study results not yet posted [5].
Sep 10 · Orphan designation (EU/3/10/774) granted by the European Commission to Intercytex Ltd (UK) for allogeneic human dermal fibroblasts for treating EB [2].
Sep 10 · PII in the UK [1].

Category

A suspension of human allogeneic dermal fibroblasts in cell storage medium [1].
The prevalence of all forms of EB in the UK is estimated to be 15 per million population, with DEB affecting 6.4 per million population. Published estimates suggest that RDEB accounts for around 15% of all EB cases, which equates to approximately 126 pts in England & Wales. However, expert opinion suggests that this number could be up to 600 pts. There is currently no cure for EB. Treatment aims to protect the skin & stop blister formation, promote healing, & prevent complications [1].
Recessive dystrophic epidermolysis bullosa (RCEB) - second-line
Intradermal

Trial or other data

Jan 16 · Intercytex announces that current experience of injecting ICX-RHY in 14 individuals with RDEB indicates that this therapy can deliver a disease-modifying intervention without the adverse events associated with topical application and should provide a major step forward in delivering translational research to benefit patients [4].
Mar 13 · PII (EUDRACT2010-023121-38-GB) trial investigating ICX-RHY in 25 UK pts aged 18-50 years with RDEB is expected to complete. Pts must have 5 open skin erosions each with surface area between 5 and 50cm2 on limbs or trunk. They will be randomised to one series of ICX-RHY 0.25ml (20x106 cells/ml) injections intradermally per linear cm of erosion margin or placebo 0.25ml intradermally, with 26 week follow-up. The primary outcome is time to wound closure; secondary outcomes are Wound surface area; erosion pain; wound recurrence; adverse events; clinical assessment [1].