Treatment of hereditary Tyrosinemia Type 1 (HT-1 )

 What is HT1 ?

Tyrosinemia type ( HT-1 ) is caused when the body is unable to break down tyrosine

which is one of the protein’s core building block. When the tyrosine is unable to

break down; toxic substances are formed within blood known as succinylacetone.

This clearly shows that if Tyrosinemia isn’t treated, it can cause liver and kidney

damage and other neurological issues.

How many people are affected by Tyrosinemia & is it inherited from both the parents?

Tyrosinemia affects 1 in 100,000 people and must be inherited from both the parents.

Does a specific gender is more prone to have HT-1 ?

Both Male and Female are equally prone to having HT- 1 .

Are people affected by Tyrosinemia never eat proteins ?

No that’s not true though low – protein diet is required in such cases to cause build

up of too much protein in child’s body but they still small amount of protein and

amino acids are required in the body .

Does HT-1 can be cured permanently ?

No not necessarily , those who have HT-1 will likely to manage for entire life .

What is Nitisone ?

It is first drug which has been approved for the treatment of HT-1

Does the dose of Orfadin (Nitisinone )remains same throughout the life ?

The dose of Orfadin depends on the body weight changes of a body , this is a

reason blood tests become necessary to quantify the levels the tyrosine in the blood

.Are there any other versions of Nitisinone available other than Orfadin ?

Yes , Nobel’s Pharma also manufacturer Nitisinone known as Tisinon which is

available in strength’s of 5mg and 10 mg which comes in a pack of 60 capsules .

In what Dosage forms and strengths does Orfadin comes ?

Orfadin comes in capsule form of 2mg , 5 mg , 10 mg and 20 mg . Apart from that it

also comes in oral suspension 4mg/ml .

Can Nitisinone be procured by the patients if not available or approved in

one’s own country ?

Yes , Indian pharma network  Named Patient program help’s patient to procure the

medicines which are not available or approved in one’s own country. Indian pharma network  provides access to both Orfadin (nitisinone) which is marketed by the

company Swedish Orphan Biovitrum International AB, Sweden and Tisinon

(nitisinone)by Nobel Pharma which is a Turkish brand and manufactures drugs for

unmet medical needs.

Read More- Hereditary tyrosinemia type 1 (HT-1) medicine

Venetoclax: Briefed information about the medication

 On May 15'th, 2019, the Food and Drug Administration approved Venetoclax for chronic lymphocytic leukemia. It is used with another drug named obinutuzumab (when CLL has not been previously treated). Or venetoclax is used with another drug named rituximab (when CLL has come back or has not responded to treatment). Venetoclax is also used by itself when CLL has a chromosome deletion and has come back or has not responded to treatment; or has no chromosome deletion and other therapies are not available. 

Chronic Lymphocytic Leukemia is a type of cancer that typically affects the lymph nodes and WBCs known as “B lymphocytes”. In this disease, unhealthy B lymphocytes amplify rapidly and live a bit longer. This causes an increase in the number of unhealthy B lymphocytes in the blood.

Venetoclax works to inhibit a protein in the body known as BCL-2, which helps malignant cells

 survive. Inhibiting BCL-2 helps to destroy and reduce the number of malignant cells.

Ingredients in Venclexta

The medicine Venclexta contains active ingredient as Venetoclax, and as a non-medicinal ingredients it contains: colloidal silicon dioxide, calcium phosphate dibasic, copovidone, polyethylene glycol, polysorbate 80, iron oxide yellow, polyvinyl alcohol, sodium stearyl fumarate, talc and titanium oxide. Venetoclax 50 mg tablets also contain iron oxide black and iron oxide red.

Dosage Forms & Strengths

The medication Venetoclax typically comes in the form of Tablets as following dosage strengths:

• venetoclax 10 mg tablets
• venetoclax 50 mg tablets
• venetoclax 100 mg tablets

Dosing Regimen

The proposed dosing regimen of Venetoclax is always taken exactly as your health specialist tells you. Drink plenty of water while on treatment with Venetoclax to help reduce the risk of getting TLS. Take the tablets through a meal and water persistently everyday. Swallow the pills whole. Healthcare professionals recommend chewing/crushing/breaking the pills. Your doses of Venetoclax may be reduced in some cases, including in case you have severe liver complications, or you are on with certain drugs that may interact with Venetoclax. Your therapy may be interrupted or dose lowered in case you have certain side effects.

Usual dose: Your health specialist will start Venetoclax at a low dose for 7-days. Your health specialist will gradually increase the dose of Venetoclax over the next 4 weeks to the full standard dose. The usual dosage is as follows: 

The starting dose is 20 mg (2 × 10 mg tablets) once daily for 7 days. The dose will be enhanced to 50 mg (1 × 50 mg tablet) once daily for 1-week. The dose will be enhanced to 100 mg (1 × 100 mg tablet) once daily for 1-week. The dosage will be enhanced to 200 mg (2 × 100 mg tablets) once daily for 1-week. The dose will be enhanced to 400 mg (4 × 100 mg tablets) once a day.

If you are taking venetoclax dosage alone, you will stay on the 400 mg per day dose, which is the standard dosage, for as long as required. 

Venetoclax in combination with Obinutuzumab: You will start Venetoclax dosing after receiving your first cycle of obinutuzumab doses. You will receive Venetoclax for 12 months.

Venetoclax in combination with rituximab: You will start your rituximab following the initial 5 weeks of Venetoclax. You will receive the medication Venetoclax for 2 years.

Possible Side Effects from Venclexta    

These are not the entire possible side effects you may feel while on venetoclax. In case you have any venetoclax side effects not listed here, contact your health specialist immediately.

• diarrhea
• constipation
• nausea
• vomiting
• decreased appetite
• weight loss
• stomach pain
• swelling of arms, hands, legs, and feet
• weakness
• mouth sores
• shortness of breath
• rash
• fever
• headache
• dizziness
• feeling tired
• cough
• muscle, bone, back, neck and joint pain
• itching

Venetoclax may be responsible for causing abnormal exam and blood test results. Your health specialist will do certain tests prior to and during the treatment. A health specialist will interpret the results. They will let you know in case there are any abnormalities in tests that might require treatment.

Storage and Handling 

• One should store the vials of Venetoclax at 2–8°C (36–46°F) in the original carton to protect from light. 
• Venetoclax is an antineoplastic product so one should handle this medicine with precautions. 

Price and Availability in India

The Venetoclax price is absolutely reasonable. The prices of this medication usually depend on the pharmaceutical firm or company or wholesaler you visit. Venetoclax is available as a brand name medicine only, a generic version is not yet available. 

Nitisinone: Hereditary tyrosinemia type 1 (HT-1) medicine

 The medicine Orfadin contains Nitisinone, an FDA-approved drug to treat patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Nitisinone is a competitive inhibitor drug of 4-hydroxyphenyl-pyruvate dioxygenase, an enzyme upstream of fumarylacetoacetate hydrolase (FAH) in the tyrosine catabolic pathway. 

Dosage and administration

The drug Nitisinone comes in the standard strengths of 2 mg, 5 mg, 10 mg, and 20 mg capsules to administer orally.

 Following are the administration and dosage instructions:

• The recommended initial dosage of Nitisinone is 0.5 mg/kg orally twice daily.
• One should titrate the dose for individual patients based on biochemical and clinical responses.
• One should adjust the dose of Nitisinone capsules individually.
• Healthcare professionals should monitor plasma and urine succinylacetone concentrations, liver function parameters, and alpha-fetoprotein levels.
• If succinylacetone is still detectable one month after the start of nitisinone treatment, increase the Nitisinone dosage to 0.75 mg/kg twice daily. 
• The patient may need a maximum dosage of 1 mg/kg orally twice daily based on evaluating all biochemical parameters.  
• If the biochemical response is satisfactory, the dosage should be adjusted only according to body weight gain.
• In addition to the tests above, during the initiation of therapy or if there is a deterioration in the patient’s condition, it may be necessary to follow all available biochemical parameters more closely. 

Side-effects of Nitisinone

The common side-effects of Nitisinone 20 mg, 10 mg, 5 mg, and 2 mg are:

•  Elevated tyrosine levels
• Thrombocytopenia (deficiency of platelets)
• Leukopenia (decrease in white blood cells)
• Conjunctivitis (inflammation in the eye)
• Corneal opacity 
• Keratitis (inflammation in the cornea)
• Photophobia 
• Eye pain
• Blepharitis (inflammation of eyelids)
• Cataracts
• Granulocytopenia (less no. of a type of white blood cells)
• Epistaxis (bleeding nose)
• Pruritus (skin allergy)
• Exfoliative dermatitis 
• Dry skin
• Maculopapular rash 
• Alopecia (baldness)

Price of Nitisinone

The price of Nitisinone in India is reasonably reasonable, and one can purchase this medicine from any certified generic medicine supplier. Certified pharmaceutical organizations like Indian Pharma Network help to connect the patients with certified suppliers, wholesalers, and dealers of Nitisinone in India and worldwide. 

FAQ

What are the ingredients in the medicine Orfadin capsules?

The active ingredient in the Orfadin capsules is Nitisinone, with the inactive ingredient pregelatinized starch. 

What are the brand names for the drug Nitisinone?

The innovator brand names for Nitisinone are Orfadin, Tisinone, and Nityr. 

How much does Orfadin cost?

The cost of Tisinone (or Orfadin) is sensibly reasonable, and one can purchase this medication from any licensed generic medicine supplier. For more information regarding the drug Nitisinone, contact us on 18008891064.