Orphan Drug Designation for the use of Teicoplanin in Cystic Fibrosis

RP announcement

Milan, September 2017 Neupharma and Rare Partners are glad to announce their strategic partnership for a development project in the area of lung infections caused by Methicillin-resistant Staphylococcus aureus (MRSA).
This partnership had initially focused on cystic fibrosis, a therapeutic area where Neupharma has been deeply involved for many years.
On August 23rd, 2017 the European Commission granted to Neupharma the orphan drug designation for the use of Teicoplanin in the treatment of cystic fibrosis. The designation is for a new inhalable formulation for the treatment of MRSA lung infections in cystic fibrosis patients, a life-threatening condition.

Fabio Borella, President and co- founder of Neupharma, said: “ receiving orphan drug designation is a very important regulatory milestone, and we are pleased that our formulation of Teicoplanin for cystic fibrosis has been granted this status. This project will allow Neupharma to have its own product marketed in all the UE Countries and strengthens its know-how in the nebulization therapy”.

Marco Prosdocimi, Managing Director of Rare Partners, said that "this important result has been made possible thanks to the collaboration we have established with an important player such as Neupharma, one of the companies in our country that is a reference point for doctors and at the forefront in the treatment of respiratory infections, cystic fibrosis in particular. We are confident that this result may increase the attractiveness of a collaboration with our company, as a way to advance experimental research results to clinical application".

About Cystic Fibrosis

Cystic fibrosis is caused by changes (mutations) in a gene that encodes for a protein called ‘cystic-fibrosis transmembrane conductance regulator’ (CFTR), which is involved in regulating the production of mucus and digestive juices.

Cystic fibrosis is a long-term debilitating and life-threatening disease because it severely damages the lung tissue, leading to problems with breathing and to recurrent chest infections.

About MRSA infection in Cystic Fibrosis

Median survival of children born and diagnosed with CF in 2010 is projected to be 37 years for females and 40 years for males, however median survival time is 6.2 years shorter in the group who had respiratory tract cultures positive for MRSA in comparison with the group without MRSA. Teicoplanin is probably the most effective antibiotic available against MRSA but its use is limited by a scarce lung penetration after i.v. administration. The nebulization of teicoplanin directly into the bronchial tree can solve this problem and maximise efficacy with low systemic toxicity.

About Neupharma

Neupharma is a young and modern company focused on innovation aiming at rare diseases, particularly cystic fibrosis. Neupharma is market leader in Italy for the inhaled treatment of Chronic Pseudomonas aeruginosa lung infection, moreover Neupharma distributes in Italy  the most advanced medical devices for the inhalation therapy in cooperation with the world leader Pari GmbH.