Home U.K. Cystic fibrosis: Two faces of wonder drug fight

Cystic fibrosis: Two faces of wonder drug fight

Cystic fibrosis: Two faces of wonder drug fight 1

Michelle Bamber, 37, is fighting for every breath at London’s Royal Brompton Hospital. Her mucus-filled lungs are struggling to work and only US firm Vertex’s Trikafta can save her. The wonder drug currently awaits an EU licence, but patients can qualify under Vertex’s “compassionate use” scheme.

The Daily Express can exclusively reveal 120 UK sufferers have been granted Trikafta, including Nicole Adams.

Michelle from Farningham, Kent, yesterday begged: “Willpower and determination have already taken me beyond life expectancy. But Trikafta could provide time to see my step-daughter grow, reach my 40th birthday.”

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Her husband Simon, 42, said: “This week her consultant gave us the dreaded conversation about preparing for life after Michelle’s death. We understand Vertex have a business to run but they have ethical and moral obligations too.

“But to know help is available yet unobtainable is nothing short of crippling.”

Last October we helped secure Vertex’s current drugs Orkambi and Symkevi – but Michelle has the wrong CF gene type. In fact her incredibly rare second CF gene may be why Vertex is hesitating.

We understand it fears the European Medicines Agency approval, due in June, will come with restrictions due to lack of data on patients with ultra-rare second genes. For a minority of sufferers Vertex’s compassionate scheme would be their only source of help.

Vertex chief Dr Jeffrey Leiden implied this in an email to us last Christmas: “Following the EMA’s appraisal, we will know the official licensed indication.”

Last night Vertex told us: “Our top priority… is to ensure that we are not putting patients at risk of unnecessary harm.”

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