"For families with more than one child suffering from nonsense mutation Duchenne muscular dystrophy, having only one child enrolled in our clinical trials with access to Translarna, can cause great distress for parents," said
"Families of children with Duchenne have wanted companies to be responsive in the case of siblings," added
About Translarna™ (ataluren)
Translarna, discovered and developed by
Abbreviated Prescribing Information:
Indication: Translarna™ (active ingredient: ataluren) is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. Dosing: Translarna is available as granules for oral suspension in sachets of 125 mg, 250 mg or 1000 mg. The recommended total daily dose of Translarna is 40 mg/kg body weight, split in 3 doses - 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Ingredients: Active ingredient: ataluren. Excipients: polydextrose (E1200), macrogol, poloxamer, mannitol (E421), crospovidone, hydroxyethyl cellulose, artificial vanilla flavor: maltodextrin, artificial flavours and propylene glycol, silica, colloidal anhydrous (E551), magnesium stearate. Contraindications: Translarna is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients. Should not be co-administered with intravenous aminoglycosides, and concomitant use of other nephrotoxic agents is not recommended. Special warnings and precautions for use: Patients must have a nonsense mutation in the dystrophin gene as determined by genetic testing. Patients who do not have a nonsense mutation should not receive ataluren. Patients with renal and hepatic impairments should be closely monitored. In nmDMD patients receiving ataluren, it is recommended that total cholesterol, LDL, HDL, triglycerides, serum creatinine, BUN, and cystatin C be measured on an annual basis, or more frequently as needed based on clinical status. It is also recommended that resting systolic and diastolic blood pressure be monitored every 6 months in nmDMD patients receiving ataluren concomitantly with corticosteroids, or more frequently as needed based on clinical status. Caution should be exercised when ataluren is coadministered with medicinal products that are substrates or inducers of UGT1A9, inhibitors of BCRP, or substrates of OAT1, OAT3, or OATP1B3. Medicinal product subject to medical prescription. Treatment with Translarna should only be initiated by specialist physicians with experience in the management of DMD. Adverse reactions: The most frequent adverse reactions that were very common at the recommended dose were nausea, vomiting, and headache. Common adverse events include: decreased appetite, weight loss, dizziness, high blood pressure, cough, nosebleed, constipation, diarrhoea, wind, regurgitation, stomach discomfort, stomach pain, rash, arm or leg pain, cyst in the kidney, abnormally frequent urination, involuntary urination, abnormal urine colour, fever, tiredness. Authorised for marketing in the
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA is copied from DNA through the transcription process. PTC has received conditional marketing authorization in the European Economic Area for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.
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