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How Nitisinone Copay Assistance Works
1 – Check Your Coverage/Benefits Verification
Our team of expert billers finds the best avenues of coverage that minimize out-of-pocket costs.
2 – Transfer Prescription to AmeriPharma
We process your prescription by working with your previous pharmacy or prescriber, making the transition quick and easy.
3 – Prior Authorization
Our team of specialists obtains approval from your insurance companies within 24 to 72 hours.
4 – Copay Assistance & Financial Aid
We secure financial aid and decrease copays, out-of-pocket expenses, and high deductibles. To date, AmeriPharma Specialty Care has secured $55 million in financial assistance for our patients.
5 – Nursing Care Coordination
AmeriPharma puts your schedule and home environment first when scheduling and coordinating one of our specialized nurses for your in-home infusions.
6 – Delivery Coordination
Medications are always delivered in strict compliance with the specific requirements for immune globulin shipping. Next-day and overnight cold-chain deliveries are coordinated around your schedule.
What Is Nitisinone?
Nitisinone is a synthetic inhibitor used to treat a rare and lifelong genetic disorder called hereditary tyrosinemia type 1 (HT-1). Patients with tyrosinemia type 1 are usually unable to properly break down tyrosine (an amino acid) due to a deficiency of a specific enzyme. The failure of tyrosine breakdown leads to the buildup of tyrosine and its toxic metabolites in the tissues or other body organs. The accumulation of these toxic components causes severe and progressive organ damage, in particular, liver and kidney cancer in HT-1 patients.
Nitisinone slows down the progression of HT-1 by preventing the formation and accumulation of toxic metabolites in the liver and kidney.
Nitisinone is sold under the brand names Orfadin and Nityr and can only be obtained through a doctor’s prescription.
How Does Nitisinone Work?
Typically, our body breaks down protein into smaller components called amino acids. These amino acids are further broken down in a series of steps via various enzymes. Fumarylacetoacetate hydrolase (FAH) is an enzyme needed for the final breakdown of tyrosine and releases substances that the body utilizes to perform different functions.
People who are born with HT-1 lack the FAH enzyme. Their bodies only metabolize a small amount of tyrosine and produce large amounts of toxic metabolites. Nitisinone works to stop the tyrosine breakdown pathway by working as an inhibitor (a component that stops the enzyme activity).
Mode of Action
Nitisinone acts as an inhibitor of hydroxyphenylpyruvate dioxygenase (HPPD), an enzyme involved in the third step of the tyrosine breakdown pathway. Nitisinone blocks the action of this third enzyme, so the reaction won’t proceed further and make toxic metabolites in HT-1 patients.
Copay and Financial Assistance
AmeriPharma Specialty Care alleviates financial burdens for patients and their families
Advanced software locates funding sources to match you with top-dollar foundation programs
One of our copay assistance specialists will assist with the application process
Automatic updates will be sent to you and your physician on the status of the funding
Nitisinone Side Effects
Nitisinone may cause unwanted side effects. Some side effects are common and usually go away during treatment. In other cases, however, side effects could be severe and potentially life-threatening.
The following are some of the side effects you may experience after taking nitisinone suspension, tablets, or capsules:
- Headache, diarrhea, or upset stomach due to glycerol content of nitisinone oral suspension
- A severe allergic reaction with symptoms such as hives; rash; and swelling of lips, tongue, and throat
- Hair thinning
- Vision changes, and eyes becoming sensitive to light (photophobia)
- Eye redness, irritation, and pain
In more severe cases, when you don’t eat diets restricted in tyrosine and phenylalanine, you may experience some adverse side effects due to elevated levels of tyrosine, which has been associated with toxicity to the eyes, skin, and the nervous system. Serious adverse symptoms include:
- Dry skin
- Keratitis (corneal ulcers)
- Corneal opacities
- Conjunctivitis (pink eye)
- Painful patch of thick skin (hyperkeratotic plaques) on the palms or soles
- Leukopenia and severe thrombocytopenia
The dose of nitisinone can vary from patient to patient and is determined by their body weight and their health status. The recommended starting dose for adult and pediatric patients is 0.5mg/kg twice a day for 4 weeks (1 month).
The dose of nitisinone is adjusted according to the level of succinylacetone (toxic metabolite) in the serum or urine of the HT-1 patient. For instance:
- If succinylacetone is still detectable in the serum of HT-1 patients after taking a stable dose (0.5 mg) for four weeks, then the dose is increased from 0.5 to 0.75 mg/kg twice daily.
- Once the succinylacetone is undetectable in the serum of HT-1 patients, the dose is adjusted according to the patient’s body weight. The maximum dose of nitisinone capsules and oral suspension is usually not more than 1 or 2 mg/kg body weight and is taken once a day.
It is recommended to consult your healthcare provider if your condition deteriorates after switching dosing from twice a day to once a day.
If you missed your nitisinone dose, take it immediately. If it is almost time for your next dose, skip the missed dose and take only the next dose. Do not take a double dose.