A drug being developed to treat cystic fibrosis has just been granted Fast Track Designation by the US Food and Drug Administration (FDA), reports PR Newswire. This shows that previous trials of the drug have been successful, and that the FDA recognises the potential of the drug to help patients with cystic fibrosis. These designations provides several benefits to the company developing the drug that will help them to carry out further testing, and, hopefully, make the drug widely available.
Cystic fibrosis (CF) is an inherited genetic condition that causes mucus to build up in the digestive system and lungs. It is caused when the CFTR gene mutates, causing it to produce a dysfunctional form of the CFTR protein. CFTR proteins are usually involved in regulating mucus levels, but because the protein does not work in patients with CF, the mucus builds up. Symptoms usually begin in childhood, and the disease gradually worsens over time. The sticky mucus build-up in the lungs puts patients at risk of serious infections and breathing issues. The mucus also builds up in the pancreas, which is a digestive organ. A large amount of mucus can prevent digestive enzymes from reaching the gut and breaking down food. This stops CF patients from processing nutrients, and can lead to malnutrition.
CF is a lifelong condition that there are not currently any cure for. However, the disease can be managed using several treatment options. Some of the mucus can be moved through regular exercise techniques and airway clearance techniques that use controlled breathing. Some medicines can also be used to improve symptoms, including antibiotics to manage lung infections, and drugs to reduce and thin mucus to help clear build-ups. However, effective drug treatments remain limited.
A new drug to treat CF is under development by Proteostasis Therapeutics, Inc., and has just been granted Fast Track Designation by the U.S. Food and Drug Administration. This means that the FDA is keen to support the development of the drug, because it is believed to have the potential to fulfil an unmet need for patients with life-threatening illnesses. The Fast Track Designation means that the FDA will prioritise communications with the drug developers in order to speed up its development and testing. The Cystic Fibrosis Therapeutics Development Network, the Clinical Trial Network, and the research branches of the Cystic Fibrosis Foundation and the European Cystic Fibrosis Society have also endorsed the new drug.
The potential drug is comprised of three parts that work together to treat the dysfunctional CFTR protein, which causes the symptoms of CF. One of the elements is completely new, but the other two are variations on previous drugs. The president of Proteostasis Therapeutics, Inc., Meenu Chhabra, hopes that this potential new drug could particularly help certain members of the CF community that find limited benefits from existing treatments, including F508del heterozygotes and F508del homozygotes.
The Fast Track Designation for this drug will speed up the process of further testing to make sure that it is safe and effective for patients with cystic fibrosis.
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