An improved formulation of phenylbutyrate, Pheburane is the only taste-masked formulation available for the treatment of Urea Cycle Disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase and argininosuccinate synthetase. Phenylbutyrate is well-known for its extremely unpleasant bitter taste, which may compromise compliance to treatment, especially in children.
Pheburane has been orphan designated by the Therapeutic Goods Administration
An improved formulation of nitisinone, Nityr is the only tablet formulation available for the treatment of patients with hereditary tyrosinaemia type 1 in combination with dietary restriction of tyrosine and phenylalanine.
Nityr has been orphan designated by the Therapeutic Goods Administration
Nityr was approved by Australia’s TGA in June 2018. The Product Information can be accessed here.
Prohippur is an improved formulation of sodium benzoate, it is the only taste-masked formulation being developed for the treatment of Urea Cycle Disorders.
Prohippur has been orphan designated by the Therapeutic Goods Administration.
Ucedane is an improved formulation of carglumic acid, it is the only room temperature formulation being developed for the treatment of a metabolic condition.
Ucedane has been orphan designated by the Therapeutic Goods Administration.
Citrupress is an intravenous form of L-citrulline. Completed clinical studies have shown its potential to treat pulmonary hypertension developed after heart surgeries, which can occur due to the lack of citrulline production.
Approximately 20 percent of children who have heart surgery with use of the bypass pump develop pulmonary hypertension, or high blood pressure in the lungs. In early studies it has been found that Citrupress prevented pulmonary hypertension from developing.
Cholic acid capsule
A paediatric orphan drug for the treatment of an ultra rare disorder of bile acid metabolism leading to cholestatic hepatitis and progressive liver failure that has been developed and will be submitted to regulatory agencies for evaluation in the designated jurisdictions in the upcoming months. Clinical studies have shown:
- Reversal of the pathogenic process with improvement of liver function tests and histology
- Reversal of failure to thrive with return to normal weight parameters for age
- Excellent tolerance with no drug-related adverse events
Cholic acid capsules has been orphan designated by the Therapeutic Goods Administration.
In addition, 8 orphan products are under development. For more details please refer to our pipeline.
Orpharma welcomes new opportunities to supply orphan drugs in the designated regions, please contact us for a confidential discussion.