Treatment for Hereditary Tyrosinemia Type 1 Granted Shelf Life Extension

According to a story from Business Wire, the drug developer Cycle Pharmaceuticals recently announced that is has completed its goal to obtain the necessary data regarding the stability of its product nitisinone (marketed as NITYR™) for its shelf life to be extended by the US Food and Drug Administration (FDA). The shelf life while be extended from 24 months to 36. The drug is used as a treatment for hereditary tyrosinemia type 1.

About Tyrosinemia

Tyrosinemia is a metabolic disorder which prevents the body from processing the amino acid tyrosine. Without treatment, the disorder can be lethal. There are three different types of tyrosinemia with each being distinguished by which mutation causes it. Type 1 is caused by mutations of the FAH gene, type 2 is caused by mutations of the TAT gene, and type 3 is caused by mutations of the HPD gene. Type 1 is the most severe form of the disease; symptoms include jaundice, failure to thrive, bleeding, a cabbage-like odor, intellectual disability, vomiting, diarrhea, respiratory, liver, and kidney failure, abdominal pain, and softening and weakening of the bones. The best treatment for type 1 is a diet low in phenylalanine and tyrosine combined with nitisinone. A liver transplant is an option for patients that fail to respond to this treatment. To learn more about tyrosinemia, click here.

About Nitisinone

Hereditary tyrosinemia is a very rare disease, with less than 200 patients estimated in the US. Nitisinone has become a critical part of treatment for most patients with the condition, but concerns about its shelf life have been a burden to patients. Many patients had to keep the medication in a refrigerator, making situations such as travel or power outages a major problem. This new formulation will not require refrigeration and does not compromise the drug’s effectiveness. Aside from prior shelf life concerns, nitisinone is a generally convenient drug to use; it is small, tasteless tablet of small size which makes it convenient for children and adults. It can also be disintegrated in water and ingested, which is useful for infants.

Nitisinone was recently approved for use by the European Medicines Agency (EMA) and has become a widespread, first line treatment for tyrosinemia in many countries around the world.


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