The American laboratory Vertex Pharmaceuticals will ultimately not continue the clinical trials of a drug against cystic fibrosis in France, which is nevertheless among the countries most affected by this genetic disease.
1,500 patients will ultimately not be able to benefit from it as planned. The American laboratory Vertex Pharmaceuticals announced Monday, February 1 that the phase 3 clinical trials scheduled for 2018 for the evaluation of a triple therapy targeting the causes of cystic fibrosis will ultimately not take place in France. A decision that provokes the ire of those fighting against this deadly genetic disease, which gradually destroys digestive and respiratory functions.
“A real loss of luck”
“For patients suffering from cystic fibrosis, being excluded from this research is a real loss of opportunity. This situation, which sounds like a condemnation for those involved in the fight against cystic fibrosis in France, is unacceptable for both patients and carers “, are indignant together in a free forum Isabelle Durieu, leader of the Muco CFTR Filière, Pierre Lemarchal, president of the Association Grégory Lemarchal and Pierre Guérin, president of Vaincre la Mucoviscidose.
All assimilate the decision of the American laboratory to a real “blackmail”, while Vertex Pharmaceuticals justifies its resolution by the impossibility of reaching an agreement with the French State on the price of a previous treatment against cystic fibrosis tested in France, l ‘Orkambi, whose reimbursement to patients would cost too much to social security. The suspension of clinical trials would, according to associations, only a means of pressure exerted by Vertex Pharmaceuticals on the French state in order to win the negotiations.
7,200 patients in France
The clinical trials of the laboratory Vertex Pharmaceuticals, which will take place in the United States and in some countries in Europe, aim to save patients years of life expectancy by delaying the decline of respiratory functions. Cystic fibrosis is the most common serious hereditary genetic disease in Europe and North America, affecting around one in 2,500 births (France has 7,200 patients *). It leads to lethal respiratory damage, which current treatments still cannot overcome, despite a significant improvement in patients’ life expectancy (estimated at 47 years in 2005 in France, this was only age 7 in 1965).
The treatment of respiratory damage currently uses “respiratory physiotherapy, antibiotic therapy, treatments with bronchodilators, anti-inflammatory drugs and mucolytic thinners”, as the Pasteur Institute explains, specifying that gene therapy did not, despite many trials, not yet proven to beat cystic fibrosis. On average, only one in 10 patients currently reach the age of 40.
* The incidence varies according to the regions from 1/2500 in the north-west to 1/10000 in the south-east with approximately 2 million heterozygous carriers throughout the country (source: Institut Pasteur).
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