VX-770 APPROVED and What That Means for Us!

I am so so excited to have received the news today that the FDA approved the drug VX-770, now called Kalydeco. It is the first drug that targets the underlying cause of Cystic Fibrosis, rather than just treating the symptoms. I'll tell you a little bit about the drug and what this news means for us.

*A lot of this info is coming straight from the CF Foundation website

Kalydeco was tested in CF patients with a specific mutation called G551D and showed remarkable results - improved lung function and lower sweat chloride levels and helps patients gain weight — all key indicators that the drug is working. In people with this mutation, the defective protein that causes CF 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.

Now that it has been approved, Kalydeco will be available to patients 6yrs and older with the G551D mutation by prescription, and while they aren't calling it the "cure", the quality of life for patients with this mutation taking this drug will be dramatically improved.

CF mutations fall into 5 classes based on the problem at the cellular level. The G551D mutation falls into Class 3. People with this class of mutation have a defective protein that moves to the right place at the surface of the cell but does not function correctly (see image below, compare the first picture, normal, to the Class III picture). 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. I mean look at how close a normal cell is to the Class 3 mutation. When this drug unlocks that locked gate, the cell works normally, removing the signs of CF. Its truly a medical breakthrough.

So while Drew does not have the G551D mutation that this drug was tested on and approved for, he does has another Class 3 mutation called R560T. There is belief that Kalydeco will correct all class 3 mutations, though it hasn't been specifically tested. Clinical trials are scheduled to begin in mid-2012 on people with other Class 3 or "gating" mutations. I have heard that this drug could cost as much as $200,000 a year, so I'm sure that without proper clinical testing on our mutation it will be extremely hard to get. Other drugs that Drew has taken haven't been tested on or approved for use in kids under age two, and with a little bit of work we were able to get those. I am very anxious to try this, either through clinical trials or just with a straight prescription and some followup tests to see if it is working for him.

I have had and continue to have a lot of hope in this drug. Your continued support and donations have made this drug possible, as Cystic Fibrosis received no federal funding. Your donations supported and developed this drug through the pipeline and through the FDA and will now change the lives of many people living with Cystic Fibrosis. Thank you for your past and present support. It means so so much to me. Visit our Great Strides Homepage to see what our everyday is like living with Cystic Fibrosis and how your continued support of our cause will help to cure Drew from this horrible disease.