Date: 2015-08-10
Type of information: Granting of the orphan status in the EU
Product name: trofinetide - NNZ-2566 - glycyl-L-2-methylprolyl-L-glutamic acid
Compound: glycyl-L-2-methylprolyl-L-glutamic acid
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases
Action mechanism: peptide hormone. NNZ-2566/trofinetide is an analog of glypromate (GPE) which is derived from IGF-1 and occurs naturally in the brain. GPE affects glial cells (astrocytes and microglia) while IGF-1 itself mostly affects neurons. IGF-1 is a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and for responding to injury and disease. Glypromate comprises the last three peptides of IGF-1 and trofinetide is Neuren’s chemically modified form of GPE that can mimic GPE’s natural function in the brain. The small modification results in the drug having an increased half-life in the circulation, better stability for easier storage and shipping, and suitability for use as an oral medication, whereas GPE itself and IGF-1 can only be administered by injection. Neuren conducted a Phase 2 double-blind placebo-controlled clinical trial in subjects aged 16 to 45 years with Rett syndrome, in which treatment with trofinetide showed clinical improvement in many of the core symptoms of Rett syndrome. The benefit was observed in both clinician and caregiver assessments. In 2016, Neuren will conduct a Phase 2 trial in subjects aged 5 to 15 years to test higher doses in this younger population and to confirm the optimum doses for a susequent Phase 3 trial in subjects aged 5 to 45 years.
Company: QRC Consultants (UK)/Neuren Pharmaceuticals (Australia)
Disease: Rett syndrome
Latest news: * On 14-16 July 2015, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion recommending glycyl-L-2-methylprolyl-L-glutamic acid for designation as orphan medicinal product for treatment of Rett syndrome. * On February 11, 2015, the FDA has granted orphan drug designation for glycyl-L-2-methylpropyl-L-glutamic acid for the treatment of Rett syndrome.
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: 2015-02-11
Orphan status UE: 2015-08-10
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
