Regulatory Affairs

FDA grants orphan drug status for Motif Bio’s Staphylococcus Aureus lung treatment

Published 19 September 2017

The US Food and Drug Administration (FDA) has granted Motif Bio's investigational drug candidate, iclaprim, orphan drug designation to treat of Staphylococcus aureus lung infections in patients with cystic fibrosis.

Orphan designation grants special status to a drug or biologic under development to treat a rare disease or condition and qualifies the sponsor of the product for various development incentives, including tax credits for qualified clinical testing, waiver of user fees and potentially up to seven years of market exclusivity for the given indication, if approved.

“Staphylococcus aureus, including MRSA, is one of the common causes of lung infections in patients with cystic fibrosis and we do not believe that any antibiotic has been approved for this indication. 

Some 80% or more of patients with cystic fibrosis die as a result of respiratory infections caused by a variety of bacteria, and MRSA infections have been growing in recent years,” said Graham Lumsden, Chief Executive Officer of Motif Bio. 

“Formulation development work is underway at Motif Bio to explore potential intravenous and inhaled formulations designed specifically for cystic fibrosis patients.”

Iclaprim has been studied in an animal model of chronic pulmonary methicillin resistant Staphylococcus aureus (“MRSA”) infection, which mimics the pathophysiology observed in the lungs of patients with cystic fibrosis. These data will be presented at IDWeek on October 6, 2017 in San Diego, CA.

Iclaprim is a novel investigational antibiotic that has a different and underutilised mechanism of action compared to other antibiotics. Iclaprim exhibits potent in vitro activity against Gram-positive clinical isolates of many genera of staphylococci, including methicillin-resistant Staphylococcus aureus (MRSA).

Iclaprim is rapidly bactericidal, achieving 99.9% in vitro kill against MRSA within 4 to 6 hours of drug exposure versus 8 to 10 hours for vancomycin. To date, iclaprim has been studied in over 1,300 patients and healthy volunteers. In clinical studies iclaprim has been administered intravenously at a fixed dose with no dosage adjustment required in patients with renal impairment or in obese patients.

The iclaprim fixed dose may, if approved, help reduce the resources required in hospitals since dosage adjustment by health care professionals is avoided and overall hospital treatment costs may be lower, especially in patients with renal impairment.

Source: Company Press Release