Put simply, it represents the most important breakthrough in the treatment of cystic fibrosis for many years. For the first time ever, following licensing decisions in the US in February and Europe in July, there is now an approved treatment that directly affects the genetic defect that causes cystic fibrosis.
For those with the G551D mutation of cystic fibrosis - for which Kalydeco is targeted - its impact is potentially life-changing. But its development brings real hope to everyone that further transformational treatments can be discovered for other genetic types in the next few years.
But licensing doesn't automatically mean access for what is a very high-cost drug. That is why we have been leading a campaign to ensure that the drug is funded within the NHS - and just a few weeks ago, we received the excellent news that all eligible patients in England will have access to Kalydeco from the beginning of 2013. We are pushing hard to ensure that Scotland, Wales and Northern Ireland follow England's lead soon.
That Kalydeco exists owes much to the foresight and imagination of our sister charity in the US, the Cystic Fibrosis Foundation (CFF). The CFF funded its early development work in partnership with a commercial company - and we need to learn from this as we improve our own approach to research here in the UK.
There have been research advances here in the UK too, and in June the first doses in the Gene Therapy Consortium's Phase 2b clinical trial were administered to patients. This trial will continue to the end of 2013 and we should have results sometime in 2014. The trial is a major milestone in the development of gene therapy research and all those in the cystic fibrosis community who have raised money for this work over the last decade can feel enormous pride in what has been achieved.
Going forward, we are going to be much more clever - and hard-headed - about how we fund research to ensure that the work we support has the best possible chance of getting into clinics as soon as possible. And in 2012 we took the first steps towards this with the appointment of Dr Janet Allen as our new Director of Research. She is working hard on developing an innovative and ambitious strategy to be published at the Wellcome Trust in April 2013.
Janet's arrival was only one of a number of changes at the Trust in 2012. In July, George Jenkins became our Chair and - following five months as an interim - I was made the permanent chief executive in October. It's a genuine privilege to do this job, and we are all focused on promoting the interests of everyone with cystic fibrosis and their families.
Improving the way we communicate with our core supporters, as well as to clinicians, politicians and the general public, is a key part of further changes we are making - and in the spring we plan to launch a new brand that will demonstrate the inspiration, passion and determination that lies behind everything we all do.
There is a great deal of other work going on too, not least the roll-out of our new Quality Improvement Programme in 2013 to drive up standards in CF care across the UK.
But I want to take this opportunity to thank you, our supporters, for your tireless efforts raising money for and awareness of cystic fibrosis. The Trust can only exist with your energy and generosity, and we are determined to repay that commitment by making 2013 a year of real achievement and progress.
Have a very happy new year!