orphanix GmbH covers all key aspects of drug identification, evaluation, research and development. It offers medicinal products solving a high unmet medical need in the paediatric orphan drug space, ready-to-implement in the partner's pipeline through licensing and/or co-development.
Our lead product under development- Retinolx addresses two severe diseases of very preterm infant that are without adequate treatment until today.
- Bronchopulmonary Dysplasia (BPD) is a life-threating lung disease leading to long-term consequences.
- Retinopathy of Prematurity (ROP) is a serious eye-disease and the most common cause of childhood blindness.
- Around 1% of births globally are very preterm and potentially affected by BPD and ROP.
- To date there has not been an authorised medicine available to administer to preterm infants. orphanix fills this need.
Retinolx is a parenteral medicinal product (intravenous/ intramuscular) based on Retinol (Vitamin A) specifically developed for preterm infants in the neonatal intensive care unit.
International neonatology guidelines recommend Retinol for prevention of BPD. Cochrane Meta-analysis shows that parenteral Vitamin A supplementation during the first 4 weeks of life significantly reduces BPD and ROP risk.
AOP Orphan Pharmaceuticals AG and orphanix GmbH have joined forces to develop Retinolx for defined territories to prevent bronchopulmonary dysplasia.
Founder & Chief Therapeutic Development Officer - AOP Orphan Pharmaceuticals AG
"We are excited to work with orphanix to develop and commercialize this potentially life-changing treatment. Projects like this are the reason I founded AOP Orphan."