Meveol : orphan drug status granted by the FDA for the treatment of cystic fibrosis.

date_publication: 
09/12/2009

Alaxia is glad to announce that its medicinal product candidate Hypothiocyanite (OSCN) / Lactoferrin developed under the brand name Meveol, received Orphan drug status for the treatment of cystic fibrosis (CF) by the US FDA (Food and Drug Administration).



Philippe Bordeau, CEO of Alaxia said: “Three months after the recognition by EMeA (European Medicines Agency), the prompt validation of our file by the FDA consolidates us in the new therapeutic way which we open. The development of our medicinal product candidate for the treatment of the frequent respiratory infections and associated problems affecting cystic fibrosis patients continues at an intensive cadence. This recognition is an encouragement to follow the clinical trials preparation; especially taking into account recent scientific papers supporting our analysis on innate lung defense and thus enhancing Meveol interest. We would remind readers, at this time when pre-clinical studies are actively in progress, we consider only results of clinical trials will be able to confirm the therapeutic interest of the medicinal product candidate Meveol. We work in this direction with the members of our scientific committee.” The aim of the company is to make available for CF patients, as soon as possible, an innovating form of treatment that has been proven effective in vitro and in vivo, particularly on main bacterial strains involved in CF patients : Burkholderia cepacia, Pseudomonas aeruginosa mucoïd and MRSA.

About Orphan Drug designation:


  Orphan drug designation is intended to encourage research and the rapid availability of drug therapies for patients with rare diseases. After expertise by the healthcare authorities the most promising compounds have special provisions to promote and accelerate their development.  

About Meveol: OSCN / Lactoferrin


Scheduled for administration by inhalation the medicinal product candidate Hypothiocyanite / Lactoferrin (Meveol) is based on recent discoveries highlighting a failure of primary immune system in CF patients. Meveol aims to compensate this lack by giving directly in the lungs natural antimicrobial molecules not produced endogenously because of the genetic disease.   

About CF
Cystic fibrosis is the most common and life-threatening genetic diseases in Caucasian populations. It is characterized by impaired CFTR protein that regulates the transport of water and salts (chloride Cl-channel but also rhodanide SCN- channel) in epithelial cells. The inactivation of the CFTR channel causes multiple failures impairing the immune systems of human airways.


Although significant progress has been made in the management and care, this disease is not cured yet.   

About Alaxia :
Alaxia is an innovative company located in the heart of the pharmaceutical pool of excellence, Lyon France. It aims to develop new therapeutic solutions in the context of rare diseases, especially cystic fibrosis.