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US FDA Grants Bronchitol Orphan Drug Status for Treatment of Cystic Fibrosis
20 / 07 / 2005
Pharmaxis (ASX:PXS) announced today that the United States Food and Drug
Administration (FDA) has expanded the orphan drug designation for the company's product BronchitolT. Bronchitol has been granted the additional indication of facilitating mucus clearance in patients with cystic fibrosis.
The orphan drug status entitles Pharmaxis to a range of incentives; these include a seven year period of market exclusivity, assistance from the FDA with clinical trial design and an exemption from FDA User fees. Many orphan products receive expedited review or accelerated approval because they are for serious or life-threatening diseases. Cystic fibrosis is a life threatening condition.
In February 2005, the FDA granted orphan drug designation for BronchitolT as a treatment for patients with bronchiectasis. Alan Robertson, Pharmaxis chief executive officer said, "Pharmaxis now has a terrific opportunity to make a real difference to people with cystic fibrosis. There have been no therapeutic advances to help clear congested lungs for patients with cystic fibrosis in the past ten years. We are looking forward to working with the FDA on getting this important product to patients not just in the USA, but worldwide."
Pharmaxis has closed enrolment for its Phase II clinical trial for Bronchitol in cystic fibrosis in Australia. The company is conducting two further Phase II studies in cystic fibrosis patients to help determine the optimal dose of Bronchitol and examine the effects of using Bronchitol compared with existing treatments.
Orphan drug status is granted by the FDA to those products intended for the diagnosis, prevention and treatment of rare diseases, or conditions where no current therapy exists or would be improved. There are 33,000 diagnosed cystic fibrosis patients in the United States. In Australia, 2,500 people suffer from the disease, a fifth of whom are children under five years of age.
Administration (FDA) has expanded the orphan drug designation for the company's product BronchitolT. Bronchitol has been granted the additional indication of facilitating mucus clearance in patients with cystic fibrosis.
The orphan drug status entitles Pharmaxis to a range of incentives; these include a seven year period of market exclusivity, assistance from the FDA with clinical trial design and an exemption from FDA User fees. Many orphan products receive expedited review or accelerated approval because they are for serious or life-threatening diseases. Cystic fibrosis is a life threatening condition.
In February 2005, the FDA granted orphan drug designation for BronchitolT as a treatment for patients with bronchiectasis. Alan Robertson, Pharmaxis chief executive officer said, "Pharmaxis now has a terrific opportunity to make a real difference to people with cystic fibrosis. There have been no therapeutic advances to help clear congested lungs for patients with cystic fibrosis in the past ten years. We are looking forward to working with the FDA on getting this important product to patients not just in the USA, but worldwide."
Pharmaxis has closed enrolment for its Phase II clinical trial for Bronchitol in cystic fibrosis in Australia. The company is conducting two further Phase II studies in cystic fibrosis patients to help determine the optimal dose of Bronchitol and examine the effects of using Bronchitol compared with existing treatments.
Orphan drug status is granted by the FDA to those products intended for the diagnosis, prevention and treatment of rare diseases, or conditions where no current therapy exists or would be improved. There are 33,000 diagnosed cystic fibrosis patients in the United States. In Australia, 2,500 people suffer from the disease, a fifth of whom are children under five years of age.