Type of information: Granting of the orphan status in the EU
Product name: ABO-202 (AAV-CLN1)
Compound: self-complementary adeno-associated virus serotype 9 expressing human codon-optimized CLN1 under control of CBh
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases - Neurodegenerative diseases
- gene therapy. ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease.
Company: Abeona Therapeutics (USA - NY)
Disease: infantile Batten disease or infantile neuronal ceroid lipofuscinosis (INCL)
- • On April 25, 2018, the European Commission has granted Orphan Drug Designation (EMA/OD/013/18) for Abeona’s gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children.
Preclinical data from the program were recently presented at the WORLDSymposium held in San Diego and an update will be presented at the American Society for Gene and Cell Therapy (ASGCT) later this year. Key findings reported included: The data demonstrate that a single intravenous, single intrathecal (IT) or combination therapy utilizing both administration routes of a self-complementary adeno-associated virus encoding the human CLN1 gene to CLN1 mice significantly increased their survival, improved behavior and reduced motor deficits.Higher doses further improved these observations, suggesting that methods increasing total CNS exposure may be beneficial and provided some survival and behavioral benefit to symptomatic mice.A combination approach delivering ABO-202 by both intravenous and intrathecal routes of administration further increased survival efficacy and improved potential treatment options for older animals with advanced disease manifestations.
- • On February 12, 2018, Abeona Therapeutics announced that the FDA has granted orphan drug designation to its ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of infantile Batten disease.This inherited fatal genetic disease primarily affects the nervous system in newborns and progresses rapidly.
- ABO-202, developed with Steven Gray, Ph.D. and the support of The Saoirse Foundation, Taylor's Tale, Garrett the Grand Batten Fighter, Hayden's Batten Disease Foundation, and the Batten Disease Support and Research Association, is anticipated to enter clinical trials in 2018. The preclinical data were presented last week at the WORLDSymposium held in San Diego. Key findings included:
- CLN1 mice recapitulate the major features of the human disease manifestations;
The data demonstrate that a single intrathecal (IT) injection of self-complementary adeno-associated virus 9 (scAAV9) encoding the human CLN1 gene to CLN1 mice at 1 week and 1 month (pre-symptomatic) significantly increased their survival, improved behavior and reduced motor deficits.
Higher IT doses further improved these observations, suggesting that methods increasing CNS exposure may be beneficial and provided some survival and behavioral benefit to symptomatic INCL mice.
A combination approach delivering ABO-202 by both intravenous and intrathecal routes of administration further increased survival efficacy 50% and improved potential treatment options for older animals with advanced disease manifestations.
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2018-02-07
Orphan status UE: 2018-05-25
Pediatric exclusivit _USA:
Pediatric exclusivity UE: