Cystic Fibrosis

Jim Cunningham Excerpts

Tuesday 8th December 2015 Westminster Hall

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

It is a pleasure to serve under your chairmanship, Sir Edward. I warmly welcome the Minister, who, I am afraid, is very familiar with what I am speaking about today; I hope he gives me an A for effort and persistence. Given that we have spent so much time discussing access to Translarna, perhaps in his winding-up speech he will have some good news for me and my constituent.

I congratulate the hon. Member for Dudley North (Ian Austin). I am absolutely delighted that he secured this debate on access to medicines for people with cystic fibrosis and other rare diseases. Like me, he knows how important this issue is for families up and down England. I have been looking at the issues surrounding Duchenne muscular dystrophy for what seems like many years—in truth, it has been for just over a year. Only 90 boys affected by the disease in England are eligible for this drug, and the number is slightly larger across the whole of the United Kingdom.

Duchenne muscular dystrophy is a devastating condition that leads to full-time wheelchair use between the ages of eight and 11. It is a progressive, muscle-wasting disease that eventually affects the muscles involved in the respiratory and cardiac functions. Sadly, few with the condition live to see their 30th birthday. I have been working with Muscular Dystrophy UK, which fights causes to do with muscle-wasting conditions. I pay tribute to that organisation for all the support and help it gives. It not only informs Members of Parliament, but helps people affected by those diseases. My constituent, young Archie Hill, is an inspiration to everybody in this area. He has been campaigning for many years, and he and his family are indefatigable in their efforts to get the right medicine at the right time to these boys.

[Mr Jim Cunningham (Coventry South) (Lab)]

I congratulate my hon. Friend the Member for Dudley North (Ian Austin) on securing this timely debate. As the right hon. Member for Chesham and Amersham (Mrs Gillan) will recall, some months ago we all went to Downing Street to petition to get something done about muscular dystrophy. I am sure she would agree that one of the big problems is that even if the new treatments are okay, there is always a long run-in, in which negotiations take place between the Government and the pharmaceutical companies.

[Mrs Gillan]

The hon. Gentleman is absolutely right. I pay tribute to the other colleagues in the House who took part in that petition. That truly cross-party effort aimed to draw attention to the drugs that are not readily and fully available to our constituents. I was grateful that it was a cross-party delegation, because such things are much stronger when they take place in an atmosphere of good co-operation across the board rather than a political atmosphere. We saw parliamentarians at their best, so I thank the hon. Gentleman for attending that lobby at No. 10 Downing Street, which was inspired partly by Muscular Dystrophy UK and partly by the families it supports.

The issue for me is the drug that the hon. Member for Dudley North referred to. Translarna is its trademark name; it is called ataluren. It is produced by a company called PTC Therapeutics, which calls it its “lead product candidate” for these disorders. I know that the Minister is familiar with PTC Therapeutics, and I hope that in his winding-up speech he will refer to any contact he has had with the company. One of the issues surrounding the efficacy and licensing of the drug is the cost, so I hope the Minister will update us on that situation.

PTC Therapeutics states that the drug is a

“novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation…and cystic fibrosis caused by a nonsense mutation…Ataluren was granted conditional marketing authorization in the European Union under the trade name Translarna”.

I believe that it is already available in France, Germany, Italy and Spain. It is the first treatment approved for the underlying cause of Duchenne muscular dystrophy, which is a complicated condition.

Nonsense mutations are implicated in a variety of genetic disorders. They create a premature stop signal in the translation of the genetic code contained in the mRNA. That prevents the production of full-length, functional proteins. The company says that

“ataluren interacts with the ribosome, which is the component of the cell that decodes the mRNA molecule and manufactures proteins, to enable the ribosome to read through premature nonsense stop signals on mRNA and allow the cell to produce a full-length, functional protein. As a result…ataluren has the potential to be an important therapy for muscular dystrophy, cystic fibrosis and other genetic disorders for which a nonsense mutation is the cause of the disease.”

The importance of access to Translarna cannot be overstated. Boys such as my constituent Archie Hill have been waiting since August 2014 for a decision on whether Translarna will be approved in England. As I said, it is the first licensed drug to tackle an underlying genetic cause of Duchenne’s. It would help to keep Archie and these other boys walking for longer and potentially delay the onset of the devastating symptoms affecting the heart and lungs that I referred to earlier.

NICE’s appraisal of the drug is ongoing, but the families have not yet been made aware of when guidance will be issued, leaving them facing an anxious wait over the Christmas period. Over the time I have known Archie and his family, I have seen his mobility decrease; it is depressing to see such an active, energetic, lively, intelligent young man, who has his life before him, being denied a drug that could well keep him active for longer and improve his quality of life.