Kalydeco®

Question

Dear expert team,
I am a 24-year old girl suffering from CF. I come from Bulgaria, where there is no European standard therapy and no CF-experts either. That means that I get information about new drugs from German and English articles. I have read several times about Kalydeco® (for G551D). I know that it can already be bought in Germany. My first question is: how can someone from Bulgaria buy Kalydeco® from Germany and how much does it cost? This is very important for all patients with this mutation here in Bulgaria.
I have the mutation F508del and 3849+10KB. I have also read about other drugs like VX 809 and VX 770 in combination with Kalydeco®. To my understanding, they are still in the second experimental stage. Do you have actual information if they will be on the market, soon? Is there also a combination of these drugs that refer to my mutation?
Best regards,
N. A.

Answer

Dear questioner,
it is right that the drug Kalydeco® (Ivacaftor) got market authorization in the year 2012 in Germany and can be prescribed in this country in case of underlying indication (only CF-patients with at least one G551D mutation). In case of a mutation combination like yours (F508del und 3849+10KB), Kalydeco® cannot be used as an effect is not to be expected. Therefore Kalydeco® is not a therapeutic option for you. As you found out correctly, the company VERTEX that produces Kalydeco® is conducting furthter clinical studies with respective drugs at CF patients with the mutation F508del.
The results of these studies are not available, yet, therefore concrete statements cannot be made at the moment when and if at all a drug for CF patients with the F508del mutation that is comparable to Kalydeco® will achieve market authorization. This remains to be seen.
In order to answer your question completely, I will also comment on the possibility of purchase and the price of Kalydeco®. Kalydeco® can be purchased in case of an indication and with a vaild prescription in any German pharmacy. The drug is very expensive and the cost for a 4-week therapy are about 20 000.- Euros (about 240 000.- Euros per year). In case of Kalydeco®, it is a drug that should be taken throughout life, therefore the cost of therapy has to be multiplied with the years of therapy.
Your sincerely,
Dr. Dr. Christina Smaczny

05.02.2013