News

Rigosertib shows promise in trial for RDEB patients with advanced SCC

Rigosertib may hold promise for recessive dystrophic epidermolysis bullosa (RDEB) associated with advanced squamous cell carcinoma (SCC), with two patients achieving complete treatment responses in the skin, according to initial clinical trial data. The findings were presented at the European Academy of Dermatology and Venereology (EADV) annual congress,…

debra of America launches registry to better understand EB

A new registry for those with epidermolysis bullosa (EB) and their families, from the patient advocacy organization debra of America, is aiming to help both them and scientists to better understand this group of rare skin disorders. The ultimate goal, according to the nonprofit, is to develop new…

Abeona seeks FDA approval for EB-101 cell therapy for RDEB

Abeona Therapeutics is seeking U.S. Food and Drug Administration (FDA) approval for EB-101, an investigational cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). The biologics license application (BLA) submitted by the company was filed along with a request for priority review, which, if granted, would shorten the…

Eddie Vedder lends his support to 1st Venture Into Cures Summit

Eddie Vedder of the rock band Pearl Jam will perform two concerts to support EB Research Partnership (EBRP) following its inaugural Venture Into Cures Summit and Dinner, set for Oct. 22 in Seattle, Washington. The organization funds research into treatments for epidermolysis bullosa (EB), a group of…

Itch is common in RDEB; available treatments don’t help much

Itch is a common symptom with recessive dystrophic epidermolysis bullosa (RDEB) and available treatments are generally inadequate for managing it. That’s according to “Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study,” published in the Orphanet Journal of Rare Diseases. “The…

Abeona poised to seek FDA approval of EB-101

Abeona Therapeutics is preparing to request U.S. approval of EB-101, a cell therapy candidate for treating recessive dystrophic epidermolysis bullosa (RDEB). The announcement follows a successful pre-biologics license application (BLA) meeting with the U.S. Food and Drug Administration (FDA). “With the constructive feedback from the FDA now in-hand,…

Paradigm Therapeutics acquires rights to SD-101 cream

Paradigm Therapeutics has acquired worldwide rights to SD-101, an investigational cream that’s being developed as a whole-body therapy to promote wound healing and ease pain and itching in all forms of epidermolysis bullosa (EB). The company now plans to meet with the U.S. Food and Drug Administration (FDA)…

Biological therapies may help manage hard-to-treat EBA

The use of biological anti-inflammatory therapies effectively controlled epidermolysis bullosa acquisita (EBA) in four people with the disease, a recent study reports. “According to our clinical experience and literature data in severe EBA cases with rapid progress, biologicals should be considered promptly,” the researchers wrote. They added that the…

2 cases highlight immunotherapy challenges in RDEB-linked cancer

Researchers have highlighted the challenges of treating two cases of aggressive squamous cell carcinoma (SCC), a form of skin cancer associated with recessive dystrophic epidermolysis bullosa (RDEB), with immunotherapy when surgery was no longer an option. One patient, who had many genetic mutations within cancer cells, responded well to…

Abeona provides new data to FDA on EB-101 ahead of meeting

Abeona Therapeutics has submitted a data briefing package on its cell therapy candidate EB-101 to the U.S. Food and Drug Administration (FDA) ahead of a meeting with the agency planned for this month. The meeting’s purpose is to discuss Abeona’s planned application seeking approval of EB-101 to treat recessive …