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Translarna

  • Brand Name – Translarna
  • Active Ingredient – Ataluren
  • Strength – 125mg, 250mg, 1000mg in sachet
  • Originator Name/Marketing-authorisation Holder – PTC Therapeutics

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What is Translarna?

Translarna is a medicine consisting of ataluren. Ataluren is a protein restoration therapy that enables the formation of a functioning protein in patients having genetic disorders caused by a nonsense mutation. And a nonsense mutation is an alteration in the genetic code that prematurely stops the synthesis of an essential protein; it results in a disorder determined by which protein can’t be expressed in its entirety and which is no longer functional, like dystrophin in Duchenne muscular dystrophy.

What is the usage of Translarna?

It helps treat Duchenne muscular dystrophy (Duchenne), a rare and fatal genetic disorder that results in progressive muscle weakness from childhood and leads to early death in the mid-twenties due to cardiac and respiratory failure. Dystrophin is severe to the structural stability of skeletal, diaphragm, and cardiac muscles. Patients with Duchenne may lose the ability to walk as early as age ten, followed by loss of the use of the arms. Duchenne patients afterwards experience life-threatening lung complications, requiring the need for ventilation support and heart-related complications.

Dosage and Administration

Ataluren comes as an oral suspension with strengths of 100mg, 250 mg, and 1000 mg granules for oral administration.

Administration

  • Healthcare professionals will recommend the dosage according to the patient’s medical history and response towards the medicine.
  • Administer the medicine orally every day in 3 doses.
  • Take the first dose of the medicine in the morning, the second at midday, and the third in the evening. Professionals recommend 6 hours of dosing intervals between morning and midday doses, 6 hours between midday and evening doses, and 12 hours intervals between the evening and the first doses on the next day.
  • The recommended dosage by professionals is 10 mg per kg body weight of the patient in the morning, 10 mg per kg body weight at midday, and 20 mg per body weight in the evening.
  • Administer the medicine orally after mixing it into a suspension in a liquid or semi-solid food. Open Translarna sachet at the time of dose preparation. Mix all the sachet contents with at least 30 ml of liquid (water, milk, fruit juice) or 3 tablespoons of semi-solid food. Mix the prepared dose well before administration. The amount of liquid or semi-solid food can be increased based on patient preference. Patients should take the entire dose.

Overdose
In the case of Healthy volunteers, receiving a single oral dose of 200 mg/kg of Translarna ataluren experienced low-grade symptoms such as headache, nausea, vomiting, and diarrhoea. Professionals observed no serious adverse reactions in these subjects. In case of an overdose, healthcare professionals provide supportive medical care and closely observe the patient’s clinical status.

Side effects

Most of the medicine has side effects, and this medicine has some side effects too.
These side effects are classified as:

Very Common
Taking this medicine has a very common side effect that is vomiting.

Common
Decreased appetite, hypertriglyceridemia, Headache, Hypertension, Cough, epistaxis, Nausea, upper abdominal pain, flatulence, abdominal discomfort, constipation, Rash erythematous, pain in extremity, musculoskeletal chest pain, Haematuria, enuresis, Pyrexia and weight decreased are common side effects.

Frequency not known
The Changes in lipid profile (increased triglycerides and cholesterol) and changes in renal function tests (increased creatinine, blood urea nitrogen, cystatin C) are side effects whose frequency is not known.

Precautions and Warnings

Patients do not have nonsense mutations.
Patients should have a nonsense mutation in the dystrophin gene as part of their underlying disease state, determined by genetic testing. One should not treat patients who do not have a nonsense mutation with ataluren.

In case of renal impairment
There are reports on the increase in ataluren exposure and ataluren metabolite in patients with severe renal impairment. The toxicity of the metabolite is unknown yet. Higher ataluren exposure was related to a potential decrease in efficacy. Therefore, professionals should treat patients with severe renal impairment or end-stage renal disease with ataluren only if the anticipated clinical benefit exceeds the potential risk and should be closely monitored for possible metabolite toxicity and decrease in efficacy. Professionals should consider lower ataluren dose.

Changes in lipid profile
Reports of some patients observed changes in lipid profile (increased triglycerides and cholesterol) in clinical trials; therefore, professionals recommend monitoring that total cholesterol, LDL, HDL, and triglycerides on an annual basis in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients receiving PTC ataluren.

In Case of Hypertension with use of concomitant systemic corticosteroids
Case studies show hypertension using concomitant systemic corticosteroids in some patients in clinical trials; experts recommend monitoring the resting systolic and diastolic blood pressure every 6 months in nmDMD patients receiving ataluren concomitantly with corticosteroids, or more frequently as per patient’s medical reports.

Monitoring of Renal function
Professionals observed small increases in mean serum creatinine, blood urea nitrogen (BUN), and cystatin C of nmDMD, and in the controlled studies. They recommended monitoring serum creatinine, BUN, and cystatin C every 6 to 12 months in nmDMD patients receiving ataluren.

In the case of Aminoglycosides
Professionals observed Aminoglycosides had been shown to reduce the readthrough activity of ataluren in vitro. In addition, ataluren was found to increase nephrotoxicity of intravenous aminoglycosides. Avoid the co-administration of these medicinal products with ataluren. Since the mechanism by which ataluren increases the nephrotoxicity of intravenous aminoglycosides is unknown, professionals do not recommend concomitant use of other nephrotoxic medicinal products with ataluren. And they advise carefully monitoring the renal function if this is unavoidable (e.g. vancomycin to treat MRSA).

Handling and storage
  • Administer each prepared dose immediately after preparation.
  • Discard the prepared dose if not consumed within 24 hours of preparation if kept refrigerated (2 to 8°C) or within 3 hours at room temperature.

The medicine comes in heat-sealed laminated aluminium foil sachet: polyethylene terephthalate (child resistance), polyethylene (colouring and polyester/foil bond), aluminium foil (moisture barrier), adhesive (polyurethane class) and the copolymer of ethylene and methacrylic acid (sealant resin for packaging integrity).

Frequently asked questions (FAQs)

Naglazyme is a formulation of galsulfase. Naglazyme is used in treating patients with Mucopolysaccharidosis type IVA. It comes in the form of an injection of the strength of 5 mg/5ml.

Translarna is a medicine consisting of ataluren. Polydextrose (E1200), Macrogol Poloxamer Mannitol (E421), Crospovidon, Hydroxyethyl cellulose, Artificial vanilla flavour: maltodextrin, artificial flavours and propylene glycol, Silica, colloidal anhydrous (E551) and Magnesium stearate are the inactive ingredients of the medicine.

Administer the medicine orally every day in 3 doses.

PTC Therapeutics is the innovator and Ikris Pharma Network is an authorized partner of it, who can help access this medicine in India for the patient through legal procedure. To know Translarna price and more, contact Ikris at toll-free no. 18008891064 or write at info@ikrispharmanetwork.com.

In India, you can buy this medicine with the help of Ikris Pharma Network, an authorized partner of “PTC Therapeutics” for this medicine.

Discard the prepared dose if not consumed within 24 hours of preparation if kept refrigerated (2 to 8°C) or within 3 hours at room temperature.