seamus-conlan
Forum Replies Created
-
Kalydeco just got EU commission approval for the Cystic Fibrosis R117h gene alteration – children (please see below):
This is incredible news for my family and in particular my son Zack.
It is hard to find the words to express my gratitude for a decision such as this and my family wish to thank the EMA and EU Commission for the part they played in this approval.
This is great news not only for my son Zack but for all the other children in Europe who will benefit from this approval.
Zack now has the opportunity to gain access to Kalydeco as a child and this will allow him to manage the symptoms of CF.
-
Kalydeco just got EU commission approval for the Cystic Fibrosis R117h gene alteration – children (please see below):
This is incredible news for my family and in particular my son Zack.
It is hard to find the words to express my gratitude for a decision such as this and my family wish to thank the EMA and EU Commission for the part they played in this approval.
This is great news not only for my son Zack but for all the other children in Europe who will benefit from this approval.
Zack now has the opportunity to gain access to Kalydeco as a child and this will allow him to manage the symptoms of CF.
-
The efforts of the Cystic Fibrosis Foundation are encouraging and the new focus on Path to a cure is trying to address one of EmilyKG’s concerns of doing things faster. The Foundation is challenging potential collaborators to submit proposals that will accelerate the pace of progress in CF drug discovery and development and intends to allocate half a billion dollars to the effort through 2025(see full details below): Important to note that the CF Foundation Lab devotes more than half of its efforts to research focused on nonsense and other rare mutations.
-
I believe that funding is key here and all of the CF community must maintain focus on the 10% and follow the lead of the CF Foundation Lab which devotes more than half of its efforts to research focused on nonsense and other rare mutations. I believe the CF community more than any other group fully understand the complexities of CF and have most awareness of why the 10% cannot be left behind. Funding for research must still be top priority until we reach the objective of cure found.
I also wish to draw attention to another group who are getting left behind here purely because of their geographic location i.e. Children with the R117H gene alteration in Europe. Kalydeco is a game changer for children with R117H but they are denied access to this by the European Medicines Agency until they are 18 years old. This drug is available to children in the US and Australia from age 6 months and above. The reasons why the drug must be approved at 6 months are documented in the link below:
I believe that Professor Guido Rasi who is the Executive Director of the European Medicines Agency has ownership and accountability here. In the coming years, I will take no pleasure in saying to Guido “I told you so” when US research papers and US hospital data confirm the importance of Kalydeco to children with the CF severe phenotype R117H 5T as there is no way of recovering the irreversible lung damage to these children in Europe. My frustration is primarily based on this needless irreversible lung damage in the absence of Kalydeco. Kalydeco has tested safe for over 6 years now.
In my opinion, Europe should have aligned with the FDA approval to children in the US back in 2015. The EMA are presiding over a situation where children with Cystic Fibrosis (R117H) must needlessly accept irreversible damage to all the affected organs in the body–lungs, pancreas, sinuses, and the entire gut for a period of 18 Years. Kalydeco treats cystic fibrosis at the root cause of disease, by restoring CFTR protein function to all the affected organs in the body and the US and Australia realise that if you start this treatment early in life(US and Australia allow access at 6 months but working towards pre-natal pending safety studies) studies show that you can add decades to a child’s life. The EMA by denying access to this drug until 18 years are shortening life expectancies and possibly removing decades from these children’s lives. They are working against countries in Europe who have signed up to the NCD Countdown 2030 accord in an attempt to prevent and control Non-Communicable Diseases (NCDs) by fuelling an increase in premature deaths for Cystic Fibrosis children with the R117H gene alteration. There must be more accountability with these accord’s if they are to reduce premature deaths and the Kalydeco to R117H children is a good example of why Europe will continue to struggle to improve their ranking in this regard. In my opinion, there is also a question over drug approval governance with respect to Kalydeco and R117H at the EMA. This is due to the fact that a disparity of 6 months and 18 years between FDA and European approval is not good enough where children’s lives are at stake.
https://www.rte.ie/news/world/2018/0921/995127-chronic-diseases/