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Orphan

Orphan Pharamaceuticals

Pheburane

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

Pheburane was approved by New Zealand’s MEDSAFE in October 2015. The Data Sheet can be accessed from here.The Consumer Medicine Information can be accessed here.

Pheburane was approved by Australia’s TGA in May 2017. The Product Information can be accessed here. The Consumer Medicine Information can be accessed here.

 

Nityr

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. The Consumer Medicine Information can be accessed here.

Nityr tablets was listed on the Life Saving Drugs Program (LSDP) in May 2019. Listing on the LSDP has made the product readily available to all patients diagnosed with hereditary tyrosinaemia type 1 . The full press release can be accessed here.

NITYR was approved by New Zealand’s MEDSAFE in January 2021. The Data Sheet can be accessed from here. The Consumer Medicine Information can be accessed here.

 

Ucedane

Ucedane contains 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.  

 

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.


Orpharma welcomes new opportunities to supply orphan drugs in the designated regions, please contact us for a confidential discussion.