PTC124 approval?

Question

Dear expert team,

as far as I know, the drug is currently in phase III, which is supposed to end by mid-2012.

Can you predict roughly how long it will take until the drug is approved and how likely it is to get approved at all?

In case it does not get approved, would it be possible to obtain the drug from another country such as the United States or Israel?

I am sorry for asking so many questions, but the drug is really important to me.

Many thanks in advance.

Answer

Hello,

we received the following answer from Dr. Uta Düesberg (together with Dr. Jutta Bend) from the German CF patient organization:

“…unfortunately we could not get any information from the manufacturer other than that they are ‘in talks’ with the European government agencies about the approval and cannot provide any details as to when ataluren will be available in Germany. (See also PTC’s answer to our question: ‘We are in discussions with regulatory authorities in the EU but it is too early to predict when we might have approval. When we have more information, one of the ways in which we will be sharing it is through our email update list. I invite you to visit our website and sign up for free updates at: www.ptcbio.com/contact_us.’) Perhaps you would be interested in subscribing to the mailing list for updates?

Unfortunately I cannot provide any information on whether it will be possible to obtain ataluren in other countries. You would have to talk to your treating doctor about this.” [end of Dr. Düesberg’s answer]

Concerning the part of your question on receiving the drugs in other countries in case of non-approval, I consulted with Dr. Smaczny of Frankfurt University Hospital. She says that “the treating doctor decides about his/her patient’s therapy. In exceptional cases, it could well be possible that drugs that have not been approved in Germany are used with a patient (off-label use). This has to be arranged with the patient carefully; the responsibility is with the doctor, and the coverage of costs has to be approved in advance by the cost bearer. For the doctor, the decision for off-label use comes with a lot of responsibility. A drug that has not been approved in Germany but is already being used in other countries often can be ordered by foreign pharmacies, but not all drugs can be obtained this way. I do not know whether ataluren can be ordered or not. If applicable to your case, the doctor who would agree to take responsibility would have to research this question accordingly.”

Kind regards,
Annette Pfalz for ECORN-CF

22.03.2012