What is Translarna and ataluren used for?
Translarna is a medicine containing the active substance ataluren . It is indicated for the treatment of Duchenne muscular dystrophy in patients over 5 years of age who are able to walk. Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function. Translarna is used in the restricted group of patients with Duchenne dystrophy due to a specific genetic defect (called "nonsense mutation") in the dystrophin gene. Because the number of patients with Duchenne muscular dystrophy is low, the disease is considered 'rare' and Translarna was designated an 'orphan medicine' (a medicine used in rare diseases) on 27 May 2005.
How is Translarna used - ataluren?
Translarna can only be obtained with a prescription and treatment should be started by specialist physicians experienced in the management of Duchenne / Becker muscular dystrophy. Before starting Translarna therapy, patients must have a blood test to confirm that the disease is caused by a nonsense mutation and that it is therefore suitable for treatment with Translarna. Translarna is available in the form of granules (100, 250 and 1 000 mg) to be taken by mouth mixed with liquid or semi-solid food (such as yogurt). Translarna is taken three times a day at the recommended dose of 10 mg / kg (10 mg per kilogram body weight) in the morning, 10 mg / kg at noon and 20 mg / kg in the evening (for a total dose of 40 mg / kg). For more information, see the package leaflet.
How does Translarna - ataluren work?
Patients with Duchenne muscular dystrophy have a dystrophin deficiency, a protein found in muscles. Because this protein helps protect the muscle from damage during contraction and relaxation, in patients with Duchenne muscular dystrophy the muscles are injured and lose their function over time. Duchenne muscular dystrophy can be caused by a series of genetic abnormalities. Translarna is used in patients whose disease is due to the presence of certain defects (called nonsense mutations) in the dystrophin gene, which prematurely block the production of a normal dystrophin protein, giving rise to a dystrophin protein with a shorter form, which does not is able to function properly. Translarna acts in these patients allowing the protein producing apparatus present in the cells to overcome the defect so that the cells can produce a functional dystrophin protein.
What benefit has Translarna - ataluren shown during the studies?
Translarna was examined in a main study involving 174 patients with ambulatory duchenne muscular dystrophy, in which two doses of Translarna (40 mg / kg daily and 80 mg / kg daily) were compared with placebo ( fictitious treatment). The main measure of effectiveness was the change in distance patients could walk in six minutes after 48 weeks of treatment. Although a first analysis of the results of all the data collected from the study did not reveal significant differences in the distances traveled by patients treated with Translarna and by subjects treated with placebo, further analyzes showed that walking ability was reduced less in the group treated daily with 40 mg / kg of Translarna compared to the placebo group: after 48 weeks of therapy, subjects treated daily with 40 mg / kg of Translarna were able to walk on average 31.3 meters more than patients treated with placebo. This beneficial effect of the lower dose has also been corroborated by improvements in other parameters of effectiveness, including those directly related to patients' daily activities. However, no improvement was observed with the higher dose (80 mg / kg / day).
What is the risk associated with Translarna - ataluren?
The most common side effects with Translarna (which may affect more than 1 in 10 people) are nausea, vomiting and headache. Translarna should not be used concomitantly with certain antibiotics known as aminoglycosides, if given intravenously. For the full list of all side effects and restrictions with Translarna, see the package leaflet.
Why has Translarna - ataluren been approved?
The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Translarna's benefits are greater than its risks and recommended that it be approved for use in the EU. Despite the limited data available and the fact that no benefits emerged with the highest dose of 80 mg / kg per day, the CHMP was of the opinion that the daily dose of 40 mg / kg of Translarna had been shown to delay progression of the disease and that the security profile was not a cause for concern. The committee also recognized the severity of Duchenne muscular dystrophy and the unmet medical demand of patients suffering from this condition. Translarna has obtained a "conditional approval". This means that further information on the medicine, which the company is required to provide, will be available in the future. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly
What measures are being taken to ensure the safe and effective use of Translarna - ataluren
A risk management plan has been developed to ensure that Translarna is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Translarna, including the appropriate precautions to be followed by healthcare professionals and patients. Further information is available in the summary of the risk management plan.
What measures are being taken to ensure the safe and effective use of Translarna - ataluren?
A risk management plan has been developed to ensure that Translarna is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Translarna, including the appropriate precautions to be followed by healthcare professionals and patients. Further information is available in the summary of the risk management plan.
More information on Translarna - ataluren
On 31 July 2014, the European Commission issued a marketing authorization for Translarna, valid throughout the European Union. For more information on treatment with Translarna, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist. The summary of the opinion of the Committee for Orphan Medicinal Products related to Translarna is available on the Agency's website: ema.Europa.eu/Find medicine / Human medicines / Rare disease designation. Last update of this summary: 08-2014
