The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences’ gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to go through development at a faster pace and hopefully reach patients in need quicker.

About Cystic Fibrosis

Cystic fibrosis is a genetic disorder that is characterized by progressive damage to the respiratory and digestive systems. Those with cystic fibrosis do not have the slippery mucus that is normally found in the lungs. Instead they have thick and sticky mucus which builds up in their system. This buildup causes clogs in the airways, which then traps bacteria and causes breathing problems, infections, lung damage, and respiratory failure. It can also block digestive enzymes, which makes it difficult to absorb nutrients. Cystic fibrosis is a recessive disorder, meaning that the mutated gene must be passed down by both parents. The gene responsible for this condition affects the protein that regulates salt movement. The mutation in the gene varies in severity as well.

Symptoms of cystic fibrosis affect the respiratory and digestive systems. They include persistent coughing and wheezing, shortness of breath, difficulty exercising, frequent lung infections, stuffy noses, trouble with gaining weight, constipation, male infertility, salty-tasting skin, and exercise intolerance.

About the Designations

The orphan drug designation is reserved for medications that treat rare diseases, meaning they impact less than 200,000 people in the United States. The developer is given benefits, such as financial incentives, exclusive marketing rights, and fee waivers.

The rare pediatric disease designation is reserved for any therapy that treats those under the age of 18. Similar benefits are given for this designation, and the developer also receives a priority review voucher, which makes the approval process quicker.

About SPIRO-2101

SPIRO-2101 is a gene therapy that is inhaled. It is intended to replace the damaged cystic fibrosis transmembrane conductance regulator (CFTR) gene through the use of a harmless virus, adeno-associated virus.

This gene therapy will treat about 10% of patients with CF, as it aims to treat those with class 1 mutations, along with those who cannot be treated with existing CFTR modulators. These patients currently face an unmet medical need, one that this treatment can hopefully address.

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