Antibiotic Iclaprim Granted FDA Orphan Drug Status for CF Lung Infections

Ana Belo van Wijk, PhD avatar

by Ana Belo van Wijk, PhD |

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iclaprim granted orphan drug status

The U.S. Food and Drug Administration (FDA) granted orphan drug status to a broad spectrum antibiotic called iclaprim, developed by Motif Bio, for the treatment of one of the most common lung infections in patients with cystic fibrosis (CF), Staphylococcus aureus.

The FDA’s designation could accelerate the availability of iclaprim for CF patients.

Iclaprim is a new antibiotic formulation that has been proven in in vitro laboratory studies to have potent killing capacity against diverse bacteria. In particular, iclaprim rapidly killed practically all Gram-positive methicillin-resistant Staphylococcus aureus (MRSA) bacteria within six hours in vitro, compared to the antibiotic vancomycin typically used in patients infected with MSRA.

Due to the increase in serious and life-threatening infections caused by multi-drug resistant bacteria in recent years, iclaprim could increase the options available for CF patients with resistant lung infections.

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,” Graham Lumsden, CEO of Motif Bio, said in a press release.

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

Motif Bio acquired the iclaprim assets in 2015 from Nuprim following the merger of Motif with Nuprim. The same year, the FDA granted iclaprim a Qualified Infectious Disease Product (QIDP) designation and fast track status for bacterial skin and skin structure infections.

In clinical studies, iclaprim was administered intravenously at a fixed dose, and compared to other standard-of-care antibiotics, no dosage adjustment was required for patients with renal impairment or patients at risk of diabetes. No toxicity to the kidneys was detected in previous trials.

The antibiotic iclaprim was also studied in a mouse model of MRSA infection. The mouse model used in the study mimics the conditions observed in the lungs of human patients with CF. The results of this study will be presented at IDWeek 2017, Oct. 4-8 in San Diego.

According to Motif Bio, “Clinical and microbiology data indicate iclaprim has a targeted Gram-positive spectrum of activity, low propensity for resistance development, fixed dose administration and favorable tolerability profile.”

If iclaprim is approved, the fixed dose will “help reduce the resources required in hospitals since dosage adjustment by health care professionals is avoided and overall hospital treatment costs may be lower,” the company concluded.