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.

The European Medicines Agency has recommended approval of the first drug to treat the underlying genetic cause of cystic fibrosis (CF).

Kaydeco (ivacaftor) from Vertex Pharmaceuticals has been recommended by the agency for people with CF aged over 6 who carry the G551D gene mutation.

The drug has the potential to help an estimated 1,100 people in Europe in whom this mutation causes CF.

In people with the G551D mutation, Kalydeco helps the defective protein synthesised by the mutant gene to function more normally.

In two major placebo-controlled phase III studies, patients taking Kalydeco showed sustained improvements in lung function, gained weight and were 55% less likely to suffer exacerbations.

Stuart Elborn, President of the European Cystic Fibrosis Society, said: “While there has been great progress in cystic fibrosis treatment during the last few decades, we are still only treating the symptoms and complications of the disease. Kalydeco is a fundamentally different approach to the way we treat cystic fibrosis because it targets the underlying cause of the disease.”

“Since 1998, Vertex has been committed to developing new medicines to treat the underlying cause of cystic fibrosis,” commented Peter Mueller, Chief Scientific Officer and Executive VP of Global R&D at Vertex. “We look forward to working with the EMA to bring Kalydeco, our first new medicine in Europe, to people with CF as quickly as possible.”

CF is a life-threatening genetic disease affecting 35,000 people in Europe.

A genetically targeted drug for cystic fibrosis (CF), applicable to patients with a specific gene mutation, has received early approval from the FDA.

Kalydeco (ivacaftor) from US company Vertex Pharmaceuticals has been shown to strengthen lung tissue in the 4% of CF sufferers who have the G551D mutation.

The drug was granted approval in three months, one of the fastest FDA approvals ever.

The decision shows that the US market is receptive to drugs that target narrowly-defined patient populations – a ‘personalised medicine’ strategy that is the reverse of the ‘blockbuster’ paradigm.

Vertex developed Kalydeco through its collaboration with Cystic Fibrosis Foundation Therapeutics, a non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation in the US.

CF is a life-limiting genetic condition that causes lung tissue inflammation and excessive mucus production, leading to dangerous infections.

According to Vertex, Kalydeco is the first drug to address the underlying causes of CF rather than treat the symptoms.

The company plans to develop further targeted drugs to treat a wider range of CF patients.

“Kalydeco is an excellent example of the promise of personalised medicine – targeted drugs that treat patients with a specific genetic makeup,” commented FDA Commissioner Margaret A. Hamburg.

“The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”

Robert Beall, CEO of the Cystic Fibrosis Foundation, said the new drug “provides us with a roadmap for exploring additional targeted approaches to treatment for all people with cystic fibrosis”.