FDA grants GP-101 orphan status

Source: biovale 2014-8-8 Genoa pharmaceutical announced on August 6 that FDA has granted GP-101 (aerosol pirfenidone) as an orphan drug in the treatment of idiopathic pulmonary fibrosis (IPF) Oral administration of pirfenidone (brand name esbrit) has been shown to slow the progression of IPF Unfortunately, a very large oral dose is needed to achieve effective lung drug concentration Although the upper safety threshold (801 mg tid, i.e., 801 mg, 3 times a day) of pirfenidone has been established, the lung dose delivered by oral administration is too low to achieve the best effect In addition, the tolerance of gastrointestinal exposure and blood level after oral administration of pirafenidone was poor For these reasons, it is impossible to achieve the best therapeutic effect of IPF by increasing the oral dose In addition, there are other complex problems, such as food absorption of drugs, first pass metabolism, dose reduction caused by safety, drug withdrawal program, etc., which further reduce the lung dose of drugs and the interruption of maintenance treatment In order to solve the shortcomings of oral administration and maximize the efficacy of IPF, Genoa company has modified the formulation of pirfenidone, developed a novel inhaled pirfenidone (GP-101), which can produce pirfenidone aerosol and deliver it directly to the lungs In this way, a very small dose of inhaled pirfenidone can achieve the same IPF effect (5mg vs 801mg, 160 times lower dose) as that of oral pirfenidone (esbrite) In view of such a small inhaled dose, the safety and tolerability problems existing in the oral administration of pirafenidone will be solved, so as to improve the compliance of patients, and it is expected to improve the inhaled dose to achieve superior IPF efficacy As a safe and well tolerated inhaled product, GP-101 is expected to be used in combination with other drugs (such as nintedanib, a new IPF drug of Boehringer Ingelheim), in addition to being an esbrite alternative for improving efficacy Idiopathic pulmonary fibrosis (IPF) is a common idiopathic diffuse pulmonary fibrosis disease, which has the worst prognosis in chronic non tumor respiratory diseases The main symptoms were progressive dyspnea and irreversible impairment of lung function Many patients are characterized by progressive shortness of breath, physical disability and acute deterioration Within a few years of diagnosis, patients may die, and the mortality rate is even higher than many cancers It is obvious that improving respiratory quality and survival period are the main treatment targets of this disease.