- Cancer Biology & Biomarkers
- Chromatography & Mass Spectrometry
- Contract Research, Clinical Trials and Outsourcing
- Drug Discovery
- Drug Targets
- Flow Cytometry
- Informatics & Lab Automation
- Ingredients, Excipients and Dosages
- Microbiology & RMMs
- NIR, PAT & QbD
- Raman Spectroscopy
- Screening, Assays & High-Content Analysis
- Thermal Processing
- Events & Workshops
European Commission approves Orfadin oral suspension
29 June 2015 • Author: Victoria White
The European Commission (EC) has approved the oral suspension formulation of Sobi’s Orfadin (nitisinone) for the treatment of Hereditary Tyrosinaemia type-1 (HT-1).
HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver and kidney failure and can be fatal if untreated.
People with HT-1 have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver failure, renal dysfunction and neurological complications. In the most common form of the disease, symptoms arise within the first six months of the child’s life. Orfadin blocks the breakdown of tyrosine, thereby reducing the amount of toxic by-products in the body.
Oral suspension of Orfadin facilitates accurate dosing for infants and small children
Before pharmacological treatment was available, fewer than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday. Treatment with Orfadin combined with dietary restriction of tyrosine and phenylalanine and more widespread new-born screening, leading to early diagnosis have dramatically improved outcomes for HT-1 patients.
“The European Medicines Agency has called for more child-friendly formulations and the new oral suspension formulation is a demonstration of Sobi’s commitment to the needs of the infants and children diagnosed with HT-1 early in life,” says Birgitte Volck, Chief Medical Officer of Sobi. “An oral suspension of Orfadin for paediatric use will facilitate accurate dosing for infants and small children, and contribute to improving quality of life for patients and caregivers.”
The oral suspension will be made available to patients throughout Europe in H2 2015.
ABB Analytical Measurement ACD/Labs ADInstruments Ltd Advanced Analytical Technologies GmbH Analytik Jena AG Astell Scientific Ltd B&W Tek Bachem AG Bibby Scientific Limited Bio-Rad Laboratories BioNavis Ltd Biopharma Group Black Swan Analysis Limited Charles Ischi AG | Kraemer Elektronik Cherwell Laboratories CI Precision Cobalt Light Systems Coulter Partners CPC Biotech srl Dassault Systèmes BIOVIA DiscoverX Edinburgh Instruments Enterprise System Partners (ESP) EUROGENTEC F.P.S. Food and Pharma Systems Srl IDBS JEOL Europe L.B. Bohle Maschinen + Verfahren GmbH Lab M Ltd. LabWare Linkam Scientific Instruments Limited Molins Technologies Multicore Dynamics Ltd Nanosurf New England Biolabs, Inc. Panasonic Biomedical Sales Europe B.V. PerkinElmer Inc ReAgent Russell Finex Limited Source BioScience Takara Clontech Tornado Spectral Systems Tuttnauer Watson-Marlow Fluid Technology Group Wickham Laboratories Limited Xylem Analytics YMC Europe GmbH Yusen Logistics