Rare Disease Report

FDA News: Orfadin Capsules for Tyrosinemia No Longer Require Refrigeration

MARCH 06, 2017
James Radke
The FDA has approved the in-use storage at room temperature (25°C or less) for up to 45 days for all Orfadin capsules (2 mg, 5 mg, 10 mg and 20 mg) to treat patients with hereditary tyrosinemia type-1 (HT-1).
The FDA has also approved an extended shelf life for Orfadin capsules 20 mg, from 24 to 36 months.
These approvals should make life easier for families trying to manage children with HT-1. The disease requires strict adherence to a low protein diet and the addition of Orfadin to be stored at room temperature allows families more flexibility. 
Before Orfadin became available, the survival rate in HT-1 was very low (i.e., 29% after two years for children who developed symptoms before two months of age). Since Orfadin has been on the market, survival rates have dramatically increased (93% after two years in patients with treatment initiation before two months of age).
In a news release, Jon Miller, President of the Network of Tyrosinemia Advocates (NOTA) said, “The approval for increased shelf life and extended out-of-refrigeration time may help make Orfadin an even more flexible treatment option.”
The new approval may also help HT-1 patients in areas where refrigeration is limited. Part of NOTA’s mission is to help HT-1 families in those areas of the world. Below, Jon Miller talks about that aspect of NOTA's mission.


Hereditary Tyrosinemia Type 1 (HT-1)

HT- I is caused by a deficiency of the enzyme fumarylacetoacetate hydrolase (FAH). The absence of FAH leads to an accumulation of toxic metabolic products in various body tissues, which in turn results in progressive damage to the liver and kidneys. Tyrosinemia type I affects males and females in equal numbers.

Treatment for HT-1 consists of a low phenylalanine and tyrosine diet plus Orfadin.  

Orfadin inhibits 4-hydroxyphenylpyruvate dioxygenase so that toxic by-products of tyrosine do not accumulate in the body.

In the video clips below, Jon Miller, founder of NOTA  and George Diaz, MD of Mount Sinai Hospital talk about the condition from both a parent's and clinician's perspective.

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