Company behind drug for cystic fibrosis puts forward new proposal

Vertex, a pharmaceutical company, offers a drug call Orkambi for people with cystic fibrosis.

But the treatment, which costs £104,000 a year per patient, is not available on the NHS due to its price.

As previously reported in The News, a campaign started by two Portsmouth mums is trying to get Orkambi available for free.

Gemma Weir and Michelle Frank, from Paulsgrove, have daughters with cystic fibrosis which causes a thick mucus to clog up the lungs and digestive system.

Their petition received 113,000 signatures and will be debated in parliament.

Vertex have since submitted a new portfolio to the government.

A spokesman said: ‘We share the cystic fibrosis community’s sense of urgency on access to new Vertex medicines.

‘We are committed to working with the NHS to find a sustainable funding solution. That is why we have proposed to the government a bold new portfolio approach that could make our medicines available to patients as soon as possible.

‘It provides budget certainty and value to the NHS − and offers fair and equal access for patients to precision medicines that are tailored to specific genetic mutations.’

Until recently, the European Commission said Orkambi should only be used for people aged 12 or over. But earlier this year, the EU approved it for those six to 11.

The drug does not cure cystic fibrosis but stops the condition from getting worse.

In other countries it is state-funded.

Michelle, who’s six-year-old daughter Emma has cystic fibrosis, welcomed the latest proposal.

She said: ‘I am excited by Vertex’s proposal as it not only covers Orkambi but the other drugs in the pipeline.

‘I feel they have an important role to play with regards to costing for this drug, they shouldn’t price it out of the NHS’ financial abilities and should be keen to be able to offer it at a rate that is accessible for all patients.

‘The government have to now step in and make sure an agreement is made for eligible patients in the UK to have access to Orkambi as soon as possible.’

Gemma, who’s daughter Ivy, aged four, has cystic fibrosis, added: ‘I am very happy that Vertex have presented their new proposal and I urge NHS England to seriously consider what is on the table.

‘Maybe this is an indication that we as a country need to move with the times when it comes to precision medicines.’