Abeona Therapeutics

Ultragenyx submitted a BLA to the FDA for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. The BLA is supported by data showing UX111's ability to reduce heparan sulfate levels in the CSF and improve cognitive development. UX111 is well-tolerated and has various designations in the U.S. and EU. Ultragenyx also dosed the first patient in the Aspire study for Angelman syndrome.

November's dermatology highlights include FDA approval of bimekizumab for HS, resubmissions for dupilumab and pz-cel, obesity's link to psoriasis, JNJ-2113's efficacy in psoriasis, stress's impact on skin aging, and factors affecting HS diagnostic wandering.

FDA approves PTC Therapeutics' eladocagene exuparvovec for AADC deficiency, marking the first brain-administered gene therapy in the U.S. Abeona Therapeutics' BLA for EB-101 accepted by FDA. Neurogene's NGN-401 trial for Rett syndrome faces critical immune response issue. Adicet Bio initiates phase 1 trial for ADI-100 in lupus nephritis. CSL Behring to close Pasadena R&D facility, shifting focus from ex vivo gene therapy. Anixa Biosciences and Moffitt Cancer Center dose first patient in phase 1 trial for ovarian cancer therapy. Answer ALS and Cedars-Sinai release ALS data repository. MyoGene receives FDA ODD and RPD designations for MyoDys45-55 for Duchenne muscular dystrophy.

FDA approves Kebilidi, the first gene therapy for aromatic L-amino acid decarboxylase deficiency; FDA accepts resubmission for pz-cel to treat recessive dystrophic epidermolysis bullosa; AAP and AHA update drowning resuscitation guidance; identifying drug-resistant epilepsy patients in primary care; ADHD treatment clinical pearls for primary care providers.

FDA to decide on pz-cel (prademagene zamikeracel) approval for recessive dystrophic epidermolysis bullosa (RDEB) by April 29, 2025. Abeona Therapeutics resubmitted BLA after earlier concerns about manufacturing. Clinical trials showed improved wound healing and pain relief in RDEB patients.

Halozyme offers to buy Evotec for €2B; Bluebird records first revenue from Lyfgenia; BeiGene rebrands as BeOne Medicines; Adaptimmune to lay off 33% of staff; Leerink Partners adds senior M&A directors; Abeona awaits FDA decision on pz-cel.

Abeona Therapeutics' BLA for pz-cel, an RDEB treatment, was accepted by the FDA with a PDUFA date of April 29, 2025. This resubmission follows a CRL in April 2024, addressing CMC issues. The BLA is supported by phase 3 VIITAL study data showing significant wound healing and pain reduction in RDEB patients. If approved, pz-cel would be the second advanced therapeutic for RDEB, following Krystal Biotech's B-VEC approval in 2023.

Abeona Therapeutics' resubmitted BLA for pz-cel, an investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), was accepted by the FDA with a PDUFA target action date of April 29, 2025. Pz-cel aims to deliver the COL7A1 gene to produce collagen VII, potentially becoming the first gene therapy for RDEB.

FDA accepts BLA resubmission for pz-cel to treat RDEB, with a target action date of April 25, 2025. Pz-cel, an autologous COL7A1 gene-corrected epidermal sheet, demonstrated wound healing and pain reduction in phase 3 and phase 1/2a studies. The therapy aims to provide VII expression at wound sites via a stably integrated copy of the COL7A1 gene.

Abeona Therapeutics announced the FDA accepted its resubmitted BLA for pz-cel to treat recessive dystrophic epidermolysis bullosa, with a PDUFA date of April 29, 2025. The acceptance is based on positive phase 3 and 1/2a data showing sustained wound healing and pain reduction.