Date: 2017-08-29
Type of information: Granting of the Breakthrough Therapy status
Product name: EB-101
Compound: ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism:
- gene therapy. EB-101 is an autologous, ex-vivo gene therapy in which COL7A1 is transduced into autologous keratinocytes for the treatment of recessive dystrophic epidermolysis bullosa.
Company: Abeona Therapeutics (USA - NY) Sermes Planificacion (Spain)
Disease: recessive dystrophic epidermolysis bullosa
Latest news:
- • On August 29, 2017, Abeona Therapeutics announced that the FDA has granted Breakthrough Therapy designation status to the EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa. The Breakthrough Therapy designation is based on data from the Phase 1/2 EB-101 clinical trial, which demonstrated significant wound healing (greater than 50% healed) in treated wounds for over two years. Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for this particular designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. This is the first Breakthrough Therapy designation for Abeona since the FDA initiated the program in 2013.
- • On May 30, 2017, Abeona Therapeutics announced that the FDA has granted Rare Pediatric Disease Designation for Abeona’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa, including recessive dystrophic epidermolysis bullosa.
- • On May 24, 2017, the FDA has granted Orphan Drug Designation (ODD) for Abeona’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa, including recessive dystrophic epidermolysis bullosa.
- • On February 27, 2017, EB-101 (ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene) has been granted orphan drug designation in the EU for patients with recessive dystrophic epidermolysis bullosa. A phase 1/2 clinical trial with gene-corrected skin grafts is ongoing.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2017-05-24
Orphan status UE: 2017-02-27
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
