Medication for treating CF

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Information about Kalydeco and VX-809. Improvements in Lung Function

Improvements in Lung Function in F508del mutations - An update

December 18, 2012

Victory as ground-breaking, Kalydeco gets funding go ahead in England

The Cystic Fibrosisi Trust has warmly welcomed the decision by the NHS to fund Kalydeco (ivacaftor) from 1 January 2013. This is for patients in England with cystic fibrosis (CF) aged six and over and who have at least one copy of the G551D mutation.

The decision, announced by the North of England Specialist Commissioning Group (SCG), on behalf of the four SCGs in England, comes after a campaign by families and the CF Trust to ensure this transformational drug is made available to patients as soon as possible.

Ed Owen, Chief Executive at the CF Trust, said: "We are delighted with this decision to fund Kalydeco for all those patients who will benefit. It is a tribute to the hard work and determination of families, clinicians and the wider CF community. 

"We are extremely grateful to the Department of Health's Clinical Priorities Advisory Group and the SCGs in making this decision so quickly. It is heartening that the needs of people with CF have been properly recognised. We are also grateful to the role of the manufacturer of Kalydeco, Vertex, in assisting this process.

"Today's decision is a fantastic early Christmas present for those affected by the G551D mutation. But it's good news for the whole of the CF community too, as it provides a strong signal that the NHS is willing to fund future transformational therapies - at a time when the future pipeline in drug development is looking so promising."

George Jenkins, Chairman of the CF Trust said:
"This is wonderful news before Christmas. The CF Trust works tirelessly on behalf of all people with cystic fibrosis. This decision is a testament to the value and importance of our role in championing the needs of people with cystic fibrosis."

The CF Trust represented patient's views from the outset, and was part of the Cystic Fibrosis Clinical Reference Group that recognised Kalydeco's transformational benefit to patients. 

Kalydeco is the first licensed drug that treats the fundamental defect in people with cystic fibrosis. Kalydeco is effective for the four percent of patients in the UK with this G551D mutation in cystic fibrosis. 

Full statements from the SCGs and Vertex are available on the CF Trust website. NHS Commissioners are liaising with cystic fibrosis services in order to ensure effective implementation of this decision from 1 January 2013. The advice to patients and their families wanting further information about receiving Kalydeco is to contact their CF Consultant.

This decision is only applicable to those patients that live in England. The CF Trust is continuing to press for this to be made available in each of the devolved nations. Scotland is due to make its decision on 14 January 2013, it is hoped that the Scottish Medicines Consortium will take note of the positive decision in England. Decisions are yet to be made in Wales and Northern Ireland.

Source: CF Trust

December 3, 2012

Vertex Pharmaceuticals has won approval from Canadian regulators to market its treatment for cystic fibrosis, Kalydeco. The drug was approved in January by the U.S. Food and Drug Administration and is the first CF drug approved to treat the causes of the disease, rather than just the symptoms.

The Cambridge, Mass-based biotechnology company (Nasdaq: VRTX) said the Canadian approval will increase the potential market for the drug by just 100 patients. That’s because Kalydeco is only indicated for CF patients with a specific genetic mutation that is found in 4 percent of people with the disease. There are estimated to be 1,200 CF patients with the mutation in the United State. The drug is priced at approximately $300,000 per year.

“Kalydeco is an important step toward our ultimate goal of developing new medicines that target the underlying cause of cystic fibrosis for more people with this life-shortening disease,” said Dr. Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex, in a prepared statement. “We are working closely with federal, provincial and territorial governments and private health insurers to bring Kalydeco to all eligible Canadians with cystic fibrosis who have the G551D mutation.”

The drug has been found to help patients breathe easier and gain weight. The most common serious side effects include abdominal pain, increased liver enzymes and low blood sugar, which occurred in less than 1 percent of patients.

Kalydeco also was approved by European regulators in July, and is currently under review by Australian regulators for possible approval. In addition to expanding the geographic reach of the drug, Vertex is studying the drug in other subsets of CF patients to determine if the drug could be approved for wider use.

Source: Boston Business Journal

On June 28, 2012, Vertex Pharmaceuticals announced the final results of a Phase 2 clinical trial of Kalydeco™ in combination with VX-809, showing significant improvements in lung function in people with the most common CF mutation, Delta F508.

Both Kalydeco (formerly called VX-770 and also known by its generic name, Ivacaftor and VX-809, a CF drug in development, are designed to treat the root cause of cystic fibrosis. Vertex released preliminary results from the Phase 2 trial earlier this year.  

The 56-day study enrolled 109 people, ages 18 and older, with one or two copies of Delta F508. People in the study with two copies of Delta F508 (the most common CF mutation) who received the highest dose of VX-809 combined with Kalydeco showed the greatest improvement in lung function. That means an absolute improvement in lung function of 6.1 percentage points within group (p < 0.001) and 8.6 percentage points compared to placebo (p < 0.001) in homozygous patients receiving combination treatment (Day 28 to 56) with highest study dose of VX-809 -600mg.

Based on the final results, Vertex plans to begin a pivotal trial of the combination treatment in people with two copies of Delta F508 in early 2013.

Participants with one copy of the Delta F508 mutation also showed improvements in lung function, compared with those who received a placebo — though smaller than the improvements seen in those with two copies of Delta F508. Vertex plans to conduct additional studies of Kalydeco and VX-809 in those with one copy of Delta F508.

Adverse events were similar between treatment and placebo groups; most events were mild to moderate.

In people with the G551D mutation of CF ages 6 and older, Kalydeco helps improve the function of a defective protein, called CFTR, which causes the sticky mucus and symptoms in CF. In people with the G551D mutation, the defective protein moves to the right place at the surface of the cell but does not function correctly. Instead, it acts like a locked gate, preventing the proper flow of salt and fluids in and out of the cell. Kalydeco helps unlock that gate and restore the function of the CFTR protein, allowing a proper flow of salt and fluids on the surface of the lungs. This helps to thin the thick, sticky mucus caused by CF that builds up in the lungs.

As we announced here before on this site, the European Medicines Agency (EMA) announced on May 25 that it has recommended the approval of the CF drug Kalydeco™ for people with this G551D mutation aged 6 years and older in the European Union. The recommendation is being sent to the European Commission, which has the authority to approve medicines in the European Union. The European Commission generally follows the recommendation of the EMA and typically makes a decision on approval of a drug within three to four months. The FDA approved Kalydeco in January 2012 for people with the G551D mutation of CF ages 6 and older. 

The DeltaF508 mutation is more complex and therefore a combined therapy is tried out. In people with the F508del mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809, known as a CFTR corrector, aims to increase CFTR function by increasing the trafficking, or movement, of CFTR to the cell surface. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR at the cell surface.

Vertex developed Kalydeco and VX-809 with significant financial, clinical and scientific support from the US Cystic Fibrosis Foundation.

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