An innovative genetic drug to treat cystic fibrosis (CF) has been approved by the new Specialist Commissioning Groups (SCGs) for use in the NHS.
Prescription of Kalydeco (ivacaftor) for CF patients in England aged six and over who have the G551D gene mutation will be funded by the four SCGs.
The decision follows the targeted drug’s approval by the FDA in January 2012 and by the EMA in May 2012.
Kalydeco, from biotechnology company Vertex, is the first licensed drug to treat the cause of CF: a defective gene that inhibits fluid transfer in the lungs.
Of the 9,000 people with CF in the UK, 4% carry the G551D mutation. The approval of Kalydeco will stimulate the development of other targeted drugs.
The Scottish Medicines Consortium aims to reach a decision on the drug by 14 February. In England, the drug will be made available via CF care services.
Ed Owen, Chief Executive of the Cystic Fibrosis Trust, said: “We are extremely grateful to the Department of Health’s Clinical Priorities Advisory Group and the SCGs in making this decision so quickly. It is heartening that the needs of people with CF have been properly recognised.”
While the decision only directly affects CF patients with the G551D mutation, he said, it was “good news for the whole of the CF community”, as “it provides a strong signal that the NHS is willing to fund future transformational therapies – at a time when the future pipeline in drug development is looking so promising.”
In over 90% of people with CF in the UK, the genetic mutation has been identified, paving the way for targeted drug therapy.
At present, half of those living with CF are likely to die from lung damage before the age of 41.