Non-cystic fibrosis bronchiectasis (NCFB) is a chronic respiratory disease, of which 50 to 80 percent of the cases develop from unknown origins. Initial onset of the disease tends to generate a vicious cycle of recurrent lung infections and chronic inflammation that can lead to increased damage to airways. The most common bacterial infections include Haemophilus influenza and Pseudomonas aeruginosa, and are found in more than 60 percent of adult patients with stable bronchiectatic disease.
Bayer HealthCare has announced that the FDA Office of Orphan Products Development has granted orphan drug designation for its investigational Ciprofloxacin Dry Powder for Inhalation for therapeutic use of NCFB. Individuals who suffer from NCFB experience frequent severe acute pulmonary bacterial exacerbations, which promotes further inflammation along with airway and lung tissue damage.
Orphan drugs are pharmaceutical agents developed specifically to treat rare medical conditions. The condition itself is referred to as an “orphan disease,” and once orphan diseases are designated, orphan drugs can be designated as well. In the U.S., it is easier to gain marketing approval from the FDA for an orphan drug, and there are often other fiscal incentives. This refers to extended exclusivity periods, all intended to boost the drugs development which otherwise may lack sufficient profit motive. Orphan status to a disease and drug often times results in medical breakthroughs that may not otherwise been achieved due to the financial constraints of drug research and development.
In the U.S., in order for orphan drug standards to be met, there must be fewer than 200,000 individuals that are affected by an orphan disease, and the company is not expected to be able to recover the costs of developing and marketing the drug.
Bayer HealthCare is developing the orphan drug Ciprofloxacin Dry Powder for Inhalation (Ciprofloxacin DPI) for long-term intermittent treatment for decreasing the frequency of acute exacerbations in NCFB individuals with respiratory bacterial infections. The orphan drug is made of ciprofloxacin (a fluoroquinolone antibiotic) that has been formulated into a dry powder for inhalation using Novartis’ PulmoSphere™ technology which will use the T-326 powder inhaler. The therapy is currently being tested in a global Phase III clinical trial (RESPIRE; NCT01764841).