News

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

The EB Research Partnership (EBRP) and Epidermolysis Bullosa Medical Research Foundation (EBMRF) granted $900,000 to Fibrocell, a gene therapy company, to support the clinical development of FCX-007 to possibly treat recessive dystrophic epidermolysis bullosa (RDEB). “We are grateful for this investment from EBRP and EBMRF that…

Abeona Therapeutics plans to start a Phase 3 trial in 2019 testing the cell therapy candidate EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB). The multi-center, randomized study, named VITAL, will compare treatment with EB-101 to untreated wounds in the same patient. In total, 10-15 patients…

Constant Pharmaceuticals recently announced plans to begin a development program for TXA127 for the treatment of epidermolysis bullosa (EB). The potential therapy is expected to enter Phase 2 clinical trials in EB patients in 2020. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA)…

A 1% diacerein ointment could be a safe and well-tolerated way to treat generalized severe epidermolysis bullosa simplex (EBS), according to a study involving two boys treated for four weeks. In vitro data suggested that the diacerein ointment was retained in the skin for a prolonged period of time. The study,…

Treatment with intravenous immunoglobulin (IVIG) and off-label rituximab can lead to complete remission of epidermolysis bullosa acquisita (EBA), according to a study that also illustrates the variable clinical and immunological presentations of EBA. The study, “Meta-analysis of the clinical and immunopathological characteristics and treatment outcomes in epidermolysis bullosa…

RegeneRx Biopharmaceuticals announced a clinical trial update on RGN-137, an investigational therapy for the treatment of epidermolysis bullosa (EB). The update was provided by GtreeBNT, RegeneRx’s partner for the development of RGN-137 in the United States. RGN-137 is a thymosin βeta-4 (Tβ4)-based dermal gel that being developed…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…

Fibrocell Science has completed a type C meeting with the U.S. Food and Drug Administration in which they discussed the design of a Phase 3 clinical trial to assess the efficacy of FCX-007, a gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). During the…

The first participant has been enrolled in a Phase 2 clinical trial testing AC-203 for the treatment of inherited epidermolysis bullosa (EB), the trial’s sponsor, TWi Biotechnology, announced. AC-203 (also known as CCP-020) is a proprietary topical formulation of 1% diacerein, a compound able to inhibit factors that contribute to…

A technique used to decipher the DNA sequence, called next-generation sequencing (NGS), was successfully used to identify two new mutations in a patient diagnosed with both simplex and junctional epidermolysis bullosa (EB). The findings were reported in the study “Next generation sequencing identifies double homozygous mutations in two…

The first two adults with recessive dystrophic epidermolysis bullosa (RDEB) treated with the topical gene therapy candidate KB103 experienced increased levels of functional human collagen 7 (COL7) protein and anchoring collagen fibril formation, a Phase 1/2 clinical trial shows. Moreover, wounds treated with topical KB103 closed in two weeks and…