This petition is an appeal to Vertex Pharmaceuticals, the manufacturer of the latest targeted therapy for patients with cystic fibrosis.

Cystic fibrosis is an incurable rare genetic disease that affects the entire body and leads to early mortality. In 2021 in Ukraine, the largest number of children died from cystic fibrosis, compared to other rare diseases.

Despite the proven effectiveness and long-term use of gene corrector drugs (Trikafta/Kaftrio+Kalideco) by neighboring European countries, these drugs are not registered in Ukraine and, unfortunately, are not available for treatment. This is not acceptable in the 21st century. In the center of Europe, there is a country, where children die because of the lack of medicine, which already exists and helps prevent the destruction of the body from a terrible disease.

During the martial law in Ukraine, the registration of drugs takes place according to a simplified procedure and is FREE! The term from submission of documents to entry into the register of registered medicines is 12 working days.

Currently, accelerated registration procedures are in force in Ukraine for drugs that have already passed inspection and registration in other countries, defined in the regulatory legal acts of Ukraine.

Yes, an accelerated registration option is provided for drugs that have already been approved by the EMA. For such drugs, the period of specialized inspection is shortened to 45 working days, and part of the Dossier can be submitted electronically. In addition, a report from the EMA with the evaluation of the drug is submitted with the package of documents.

We, thousands of families, parents of children with cystic fibrosis, and patients appeal to the Vertex Company with a request to submit documents to the Ministry of Health of Ukraine for the registration of drugs of target therapy Trikafta/Kaftrio, which are currently the most effective for the treatment of cystic fibrosis.
This will help save the lives of thousands of patients who are fighting for life and health during the war. The number of deaths without life-saving drugs is increasing, while the Vertex Company evaluates the possibilities of expanding access to the drug in Ukraine.
Irreversible losses from the war and hostilities experienced by our families urge us to act decisively against cystic fibrosis.