NICE recommends PTC Therapeutics’ Translarna to treat Duchenne muscular dystrophy
pharmafile | April 18, 2016 | News story | Sales and Marketing | DMD, Duchenne Muscular Dystrophy, NICE, PTC Therapeutics, Translarna, regulation
Shares in drug maker PTC Therapeutics (Nasdaq: PTCT) jumped after the National Institute for Health and Care Excellence (NICE) backed its Translarna (ataluren) to treat muscle wasting disease in paediatric patients.
The UK regulator has called the drug a ‘step change’ in the management of Duchenne muscular dystrophy (DMD), which causes progressive muscle wasting and is usually fatal by age 30.
Sir Andrew Dillon, chief executive of NICE, said: “NICE acknowledges that ataluren represents a significant cost to the NHS at a time of increased pressure on funding and has considered this carefully against the uncertainties of its potential long-term benefits. This is why the committee has recommended the drug be made available for an initial period of five years, under strict conditions to allow more data to be gathered on its efficacy, before the guidance is reviewed and a further decision made on whether funding should be continued.”
Dillon added: “Ataluren is a medicine that for the first time is aimed at the root cause of the disease and has the potential to offer benefits to people with the condition and their families,” adding, “Because of its very high cost, it is important that details of the financial and other arrangements to enable this new medicine to be offered to patients on the NHS are discussed and agreed between the company and NHS England, and set out in a managed access agreement.”
However, the regulators have placed a five-year limit for the recommendation to evaluate further efficacy data. Dr Peter Jackson, chair of the NICE High Specialised Technologies Evaluation Committee said: “The committee could not have recommended ataluren without the agreement to limit its use to five years while more data are gathered. If those data show that the drug is less effective in the longer term and doesn’t provide good value, the NHS is not committed to funding the drug in the long-term.”
PTC and NHS England are in the process of finalizing a Managed Access Agreement (MAA) outlining financial and clinical details surrounding the use of Translarna, including a financial arrangement. The MAA is expected to allow PTC to collect further data on the efficacy of Translarna for the treatment of nmDMD over a five-year period with NICE guidance to be reviewed again at the end of that period.
Translarna was approved by the European Commission in August 2014 to treat nmDMD and is currently available to patients in 23 countries through either expanded access programs or commercial sales.
Primarily affecting males, DMD is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD lose the ability to walk from as early as 10 years of age and experience life-threatening lung and heart complications in their late teens and early twenties.
Shares in PTC Therapeutics closed up 43.2% to $8.92 Friday on the Nasdaq.
Anjali Shukla
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