EMA as well as FDA recently designated PureIMS’s colistin dry powder inhaler ‘Colistin Cyclops®’ orphan drug product for the treatment of cystic fibrosis.
Colistin Cyclops® is an orally inhaled antibiotic used for treating (chronic) Pseudomonas aeruginosa infections of the lungs. Despite the transformative potential of CFTR modulators in addressing the root causes of cystic fibrosis, traditional treatments like colistin remain crucial. Colistin is the antibiotic least sensitive to antimicrobial resistance of Pseudomonas aeruginosa.
FDA and EMA grant Orphan Drug Designation to products intended to diagnose, prevent, or treat rare diseases with a low prevalence such as cystic fibrosis, to incentivize and support the development of treatments for these underserved patient populations. EMA further acknowledges that, although satisfactory methods of treatment of cystic fibrosis have been authorized, PureIMS has established that Colistin Cyclops® will be of significant benefit to cystic fibrosis patients. Preliminary clinical data were provided that demonstrate a significant reduction in cough versus other dry powder formulations of approved medicines, as well as patient reported outcomes which support better convenience than nebulized delivery of authorized medicines. This is considered a clinically relevant advantage.
“This orphan drug designation by both regulatory agencies is an important recognition of an unmet need for inhaled antibiotic treatments that are less burdensome to cystic fibrosis patients. It allows PureIMS to intensify discussions with EMA and FDA on the best development approach for Colistin Cyclops®, potentially leading to accelerated regulatory pathways, such as conditional marketing authorization (EMA) or expedited review (FDA)”, says Jaap Wieling, CEO at PureIMS.
About PureIMS:
PureIMS is a clinical stage pharmaceutical and medication systems company focused on developing and commercializing innovative inhaled therapies, based on its proprietary dry powder inhaler Cyclops®. The focus is on the treatment of systemic and respiratory diseases with significant unmet medical need.
About Cyclops®:
Cyclops® is a credit card sized, easy-to-use, pre-filled DPI that is developed for inhalation powders. Upon inhalation it uses the patient’s breath to disperse the dry powder formulation into small particles appropriately sized for deep lung deposition. Cleaning and disinfection of Cyclops®, nor handling of capsules are necessary.
For questions, please contact Frank Lambert at PureIMS at flambert@pureims.com or Cristiano Musso at LifeSci Consulting at cmusso@lifesciconsulting.com
