Cystic Fibrosis

Mul-1867 new hope to Cystic fibrosis patients.

Cystic fibrosis is a chronic disease that affects the lung and digestive system and impacts 70,000 patients worldwide (including 35,000 in the US).

It is the most common genetic disorder among Caucasian children. The exact prevalence in Europe is unknown, but estimates range between 1/8,000 and 1/10,000 individuals.
The disease is chronic and generally progressive leading to the patient death. Cystic fibrosis patients due to unusually thick and sticky mucous in the lungs suffer from life threatening pulmonary infections that lead to their death. Treatment of lung infections is, therefore, a key to cystic fibrosis patient longevity.

Existing medications have significantly improved the prognosis for cystic fibrosis patients: in the 1960’s the majority of patients died before 5 years of age, whereas the current average life-span exceeds 35 years and life-expectancy is 40 years but their effectiveness is not enough.

Mul-1867 therapy with the potential to significantly improve the health and quality of life for those affected by cystic fibrosis. Importantly, and unlike other treatments that are specific to particular cystic fibrosis mutations, Mul-1867 is intended for use by the entire cystic fibrosis patient population.

The Orphan Drug Act provides for granting special status to a drug to treat a rare disease or condition. This status is referred to as orphan designation (or sometimes “orphan status”).