News

Rituximab treatment leads to long-term remission in man with EBA

A man with severe, treatment-resistant epidermolysis bullosa acquisita (EBA) experienced sustained clinical remission for at least five years after rituximab therapy. Rituximab (sold in the U.S. as Rituxan and biosimilars) is an antibody that suppresses immune system activity by targeting immune B-cells — responsible for making antibodies, including…

RDEB cell therapy pz-cel on track for May FDA decision

Abeona Therapeutics said its gene-corrected cell therapy, called pz-cel (prademagene zamikeracel), for recessive dystrophic epidermolysis bullosa (RDEB), is on track for a May 25 decision from the U.S. Food and Drug Administration (FDA), and the company is addressing concerns raised during a plant inspection. The FDA conducted a…

Gene therapy applied as eye drops improves vision for boy with DEB

The gene therapy beremagene geperpavec (B-VEC), applied directly as eye drops, improved the vision in a boy with eye scarring related to recessive dystrophic epidermolysis bullosa (DEB), according to a case report. “Our data support further investigation of B-VEC in the care of patients with dystrophic epidermolysis bullosa with…

FDA review of RDEB cell therapy pz-cel remains on schedule

The U.S. Food and Drug Administration (FDA)’s review of pz-cel (prademagene zamikeracel), a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB), is progressing on schedule. The therapy’s developer Abeona Therapeutics announced it had conducted a biologics license application (BLA) mid-cycle meeting with the FDA and the agency…

2 arthritis medications may help relieve itching for DEB patients

Treatment with baricitinib or upadacitinib, two approved medications for rheumatoid arthritis, may be effective for relieving itching in some people with dystrophic epidermolysis bullosa (DEB). That’s according to a small study from Korea in which these medicines were found to reduce patient-rated itch severity scores — in some, by…