Date: 2015-07-28
Type of information: Granting of the orphan status in the EU
Product name: sarizotan hydrochloride
Compound: sarizotan hydrochloride
Therapeutic area: Neurodegenerative diseases - Neurodevelopmental diseases - Rare diseases - Genetic diseases
Action mechanism: 5-HT1A receptor agonist/D2 receptor antagonist. Sarizotan has been licensed from Merck KGaA. Sarizotan is a full 5-HT1a agonist and a dopamine D2-like agonist/partial agonist. This compound is a highly selective compound for specific serotonin or dopamine receptors that modulates the activity of these neurotransmitters in the brain. Sarizotan has been associated with a 70 to 85 percent reduction of apneas and hyperventilation episodes in preclinical testing with both acute and chronic dosing. Sarizotan has been fully characterised in preclinical studies evaluating its toxicological effects and metabolic profile, without any significant safety concerns.
Company: Newron Pharmaceuticals (Italy)
Disease: Rett syndrome
Latest news: * On July 7, 2015, the FDA has granted orphan drug designation for sarizotan for the treatment of Rett syndrome. Newron is currently in advanced discussions with regulatory authorities in Europe, the US and Canada on a clinical development program. * On 16-18 June, 2015, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending sarizotan hydrochloride for designation as orphan medicinal product for 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: 2015-07-07
Orphan status UE: 2015-07-28
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
