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CF Drugs Update: July 2018

This is a brief summary of some recent developments in CF drug therapy availability (and likely future availability) in Ireland.

Orkambi
The roll out of Orkambi for 6-11 year olds in Ireland is going very well, including since extended use was approved in March 2018 (previously Orkambi was only licensed for 12 year olds and upwards).

CFI understands there has been an almost a 100% uptake in Orkambi among the 6-11 cohort of patients, representing over 110 patients. This is on top of the circa 380 patients aged 12 years and over already on Orkambi.

This means that the total CF population on Orkambi at present is 490 or about 37% of the CF population in Ireland. This represents a high proportion of those with two copies of the F508del  gene alteration, especially since those aged 0-5 are presently excluded from Orkambi on age grounds. Further extensions of Orkambi are covered by the pipeline deal agreed between the HSE and Vertex in April 2017.

Plans are presently well advanced to further extend Orkambi to 2-5 year olds and this will likely be considered in the new year by the European Medicines Agency (EMA) and then by Ireland through a rapid review by the NCPE. It should be noted that this and other decisions may be delayed by the move of the EMA from London to the Netherlands as a result of Brexit.

To date, Orkambi has been approved in Germany; the Netherlands; Austria; Italy, France, Ireland and Sweden. England NHS turned it down in July 2018 for a second time, which will also  impact on PWCF in N. Ireland who will continue to be denied this medication. Australia and Canada are likely to make a decision in the next couple of months. It should be noted that the Irish pipeline deal is reflected in the agreements with the Netherlands and Sweden.

Smydeco
Vertex have recently released promising results from studies into two drugs, a double combination therapy called Symdeko, and a triple combination therapy. The double combination therapy ​(Symdeko) uses a combination of ivacaftor and tezacaftor. The drug has been tested on people with CF with two copies of the F508del mutation and those with one copy of F508del and another mutation, and results have been positive.

The triple combination therapy uses a combination of ivacaftor, tezacaftor and one of three new compounds, and could work in people with one copy of the F508del mutation and one copy of a 'minimal function mutation' - approximately 90% of the CF population.

Symdeko was approved by the Food and Drug Administration (FDA) in the United States in February 2018 and may be approved by the EMA as early as February 2019. Under the pipeline deal this drug would be made available in Ireland after a rapid review by the NCPE. It is likely that some patients presently on Orkambi will be switched to Smydeko, but the criteria for switching is not yet clear.

VX 659 plus tezacaftor and ivcaftor and VX445 plus tezacaftor and ivcaftor
These two separate triple combination therapies are in phase 3 clinical trials at present following very promising results from phase 2. The target genotypes are or PWCF with one F508del and one minimal function gene alteration who don’t currently have a CFTR precision medicine – we estimate around 100-150 patients in Ireland may fall into this cohort- about 10% or more of the total CF population. These 2 therapies are presently outside the ‘pipeline deal’ but CFI will of course seek to have them included once the results of the Phase 3 trials become verified. Phase 2 trials showed a fantastic increase in FEV1 of 13 percentage points from baseline, which would put this drug therapy in the Kalydeco range of effectiveness for that marker, although it will also be important to use other markers to assess its full impact.

Conclusion
In short, CFI envisages that up to 90% of people with CF, and perhaps more, will be on one of the above ground-breaking CFTR drugs in the next few years. We predict that, all going to plan, this is going to bring a further major boost on both life expectancy and quality of life for people with CF in Ireland. Important as these new and innovative drugs are, CFI reminds our patients and families that they need to continue to adhere to the fully range of therapies, exercise and physiotherapy that has been prescribed for their care by their CF team. CFI thanks the CF centres in Ireland who have been involved in clinical trials and other research related to CFTR drug therapies, as we know this contribution has been important.

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Anti-Biotics: New Colobreathe Capsule and Tymbrineb available in Ireland
There have also been some important recent developments in relation to anti-biotics for people with CF in Ireland.

Teva Pharmaceuticals Ireland have launched a new Colobreathe (colistimethate sodium) capsule which is now available in Ireland. This new capsule is designed ‘to help minimise capsule fracture on administration when used with the Turbospin inhaler’. 

CFI have been informed by Teva, when used with the Turbospin inhaler, the new capsule ‘adds to Colobreathe’s existing benefits of portability and simplicity of use’.  For people with CF, this can mean less time spent in preparing and administering medication and as with similar time saving devices and it therefore supports adherence. A nurse support programme associated with Colobreathe can be obtained by calling 1800 804494 or by emailing This email address is being protected from spambots. You need JavaScript enabled to view it.

Teva Pharmaceuticals have also informed CFI they have launched a new generic medicine called Tymbrineb (tobramycin) nebuliser solution.  Tymbrineb will be available in Ireland from 22 June 2018, and is indicated for the long-term management of chronic pulmonary infection due to Pseudomonas aeruginosa (in patients 6 years and older).

CFI understands that this generic form of tobramycin and is an outcome of the patent for tobramycin expiring in Ireland, thus bringing competition into the market for this important and widely used anti-biotic. CFI and for their continuing support of the CF annual medical and scientific meeting in Killarney over many years.

A note on Patents and Drug Therapies
CFI supports the responsible and planned use of generic medicines and biosimilars in Ireland to reduce the cost of drug therapies for all patients, including for those with CF. CFI has also campaigned with other patient groups on this issue (see references to further information below).

Patents for drug therapies in Ireland (and the EU) normally last 20 years. Once this period is over, other drug companies can enter into competition and bring in what is known as ‘generic drugs’ that are often at a fraction of a price of the original patent drug. The price of the original patented drug also falls after the end of the patent period.

In 2012 Ireland had the third highest per person spending on drugs of all 25 countries in the EU where data was available. Ireland was 43% above the average and Ireland’s increases have been among the highest, nearly tripling from 2000-2008 (Briody)

From an industry perspective, pharma contend that they often only get only 8-12 years on patent because it takes so long for drugs to be reimbursed. They also point out to job losses arising from the ‘cliff’ at the end of patent. International human rights groups such as Oxfam and Medicines Sans Frontiers have contended that there is need to reform the patent system to reduce the cost of drugs. Industry contends that they need strong incentives to invest in drug therapies,  given the very high costs and risks associated with research and development. It should be noted  the price of generics has declined in time relative to the price of the patented drugs. The discount was 35% in 2010 to about 50-60% in recent years in Ireland. This shows that drug prices can be reduced by more effective intervention by Government at a national policy level.

For more information visit

J. Briody. ‘How Drugs Prices are set in Ireland’ www.publicpolicy.ie http://www.publicpolicy.ie/wp-content/uploads/Pharmaceutical-Prices-in-Ireland-J-Briody-Draft-2.pdf

MRCG and IPPOSI. ‘Steering a course to avoid the drug iceberg’ (Two Reports) http://www.mrcg.ie/go/news/in_the_news/mrcg-ipposi-meeting-report-the-drug-iceberg


Statement from Cystic Fibrosis Ireland
Continuing lack of deal for Orkambi in England for people with cystic fibrosis will also impact on Northern Ireland

Date of issue: July 5, 2018

Cystic Fibrosis Ireland (CFI) is very disappointed to learn today (5 July 2018) that there continues to be no agreement for the provision of Orkambi and future cystic fibrosis (CF) drugs in England as negotiations have once again broken down without agreement between NHS England and Vertex Pharmaceuticals, despite a new deal being put on the table.

The failure to find an agreement also impacts on people with CF in Northern Ireland as it has been made clear by the Northern Ireland Secretary of State that they will be guided by decisions on new medications by NHS England. 

Philip Watt, CEO of CFI, stated:

“The absence of an Assembly in Northern Ireland means that there is very limited opportunity for regional pressure to be brought to bear on behalf of patients in Northern Ireland, except by individual politicians and the fantastic work of our colleagues in the UK-based patient group Cystic Fibrosis Trust, including its CEO David Ramsden.

“We know from our experience in Ireland, that CF patients in the UK and their families will feel devastated today by this cruel and harsh decision.

“The decision not to fund Orkambi appears to be final and is all the more inexplicable given the fact that the drug was recently approved in the Netherlands and Sweden. Both of these countries adopted the innovative ‘Irish solution’ of providing Orkambi and providing rapid access to future drugs that are already showing excellent results in advanced clinical trials, as part of the one deal. Sweden, in particular, has a very similar drug approval system to the UK.

“We would call on the Taoiseach, Minister for Health and politicians in the Republic of Ireland and Northern Ireland to speak out and show solidarity on this issue. While this decision will be devastating news throughout the UK for those with CF and their families, it will be particularly devastating for those in Northern Ireland who know that these innovative and ground-breaking drugs are available only a few miles south of the border and our thoughts and support are with them today.”

ENDS

For more information:

Philip Watt, CEO, Cystic Fibrosis Ireland, email This email address is being protected from spambots. You need JavaScript enabled to view it.  / telephone 087 637 0557

Issued on behalf of Cystic Fibrosis Ireland by Don Delaney, d2 communications, email This email address is being protected from spambots. You need JavaScript enabled to view it. / telephone 087 793 3249


Click here to read the statement issued by CF Trust.

 

Sweden adapts Irish drug therapy deal to make Orkambi available

Philip Watt, CEO of Cystic Fibrosis Ireland welcomes the decision to make the ground-breaking CF drug therapy Orkambi available to CF patients in Sweden. In doing so, the Swedish authorities and Vertex (pharmaceuticals) have adapted the pioneering long term access programme that was approved in Ireland in April 2017. This long term access programme not only provides patients access to Orkambi but also CF drugs not yet approved but which are in at advanced clinical trial stage and which may be even better than Orkambi.

The CF drug Orkambi is now available in Austria, Germany, Ireland, Italy, the Netherlands, Sweden and the U.S.

Read the full statement from Vertex below:


Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor) 

June 18, 2018

- The agreement allows for reimbursement of ORKAMBI for people who have two copies of the F508del mutation from July 1 -

- A framework for assessment and access to our future cystic fibrosis medicines is included as part of the agreement -

LONDON--(BUSINESS WIRE)--Jun. 18, 2018--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of theF508del mutation, ages six and older, will be reimbursed in Sweden after concluding the three-party negotiations with TLV and the county councils. Reimbursement is effective from July 1. The innovative, long-term access agreement also provides a framework for the assessment and access of our future CF medicines.

“We are delighted that people with CF in Sweden will join the thousands of others around the world who are already benefitting from our CF medicines,” said Simon Bedson, International General Manager at Vertex. “We commend the Swedish authorities for partnering with us on an innovative, long-term access agreement. In countries where Vertex remains actively involved in reimbursement discussions, we encourage these health authorities and governments to match the commitment to innovation shown in Sweden to secure access for all patients who may benefit.”

CF is a devastating rare disease that causes continuous damage to multiple organs from birth. In the lungs, a build-up of sticky mucus causes progressive and permanent damage, severe infections and ultimately premature death. In addition to Sweden, countries where lumacaftor/ivacaftor is available to all eligible patients include Austria, Germany, Ireland, Italy, the Netherlands and the U.S.

About Cystic Fibrosis 

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

About ORKAMBI® (lumacaftor/ivacaftor) and the F508del mutation

In people with two copies of theF508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of theF508del mutation are easily identified by a simple genetic test.

ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is available as tablets and is typically taken twice per day.

For complete product information, please see the Summary of Product Characteristics that can be found onwww.ema.europa.eu.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row. 

For additional information and the latest updates from the company, please visitwww.vrtx.com.

(VRTX-GEN)

View source version on businesswire.com: https://www.businesswire.com/news/home/20180618005495/en/

Source: Vertex Pharmaceuticals Incorporated