FDA Approves Once Daily NITYR for Patients with HT-1


This past week, Cycle Pharma announced FDA approval of a once daily dose of NITYR for patients with hereditary tyrosinemia type 1 (HT-1). According to its prescribing information, NITYR is:

a hydroxyphenyl-pyruvate dioxygenase inhibitor for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Patients may take up to 2mg/kg NITYR daily. Currently, the orally administered therapy is approved for patients age 5 or older. It works by assisting with tyrosine regulation, preventing plasma tyrosine levels from rising too high and causing detrimental symptoms. Patients taking NITYR should have undetectable serum and urine succinylacetone levels following prior nitisinone treatment.

Are you a patient with HT-1 who is interested in trying NITYR? First, speak to your doctor to see if this might offer an effective solution. NITYR is offered through the Free NITYR Program, which allows patients to try NITYR for free for one month. Learn more here.

Hereditary Tyrosinemia Type 1 (HT-1)

Ultimately, HT-1 is a subset of tyrosinemia, a rare inherited metabolic disorder that prevents the body from properly processing essential amino acids. Tyrosine is one such amino acid and plays a role in many proteins. Since the body is unable to process this protein, tyrosine levels drastically rise throughout the body to the point of toxicity. Without treatment, this can be fatal.

Symptoms vary based on tyrosinemia subtype. HT-1 usually appears during infancy. Patients experience diarrhea, failure to thrive, jaundice, rickets, an inability to hold down food, and changes in mood or behavior. In more severe cases, infants may also experience kidney dysfunction. In HT-2, symptoms include changes in mood or behavior, sensitivity to light, skin hardening, ocular discomfort, and developmental delays. Finally, HT-3, the rarest form of tyrosinemia, is characterized by seizures, cognitive delays, and vertigo.

Learn more about HT-1 and other subsets of tyrosinemia here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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