Arrevus’s Oral Antibiotic ARV-1801 Receives FDA Orphan Drug Designation as CF Treatment

The U.S. Food and Drug Administration (FDA) has granted its orphan drug designation to ARV-1801 (sodium fusidate tablets), a treatment developed by Arrevus for patients with cystic fibrosis (CF).

CF patients frequently develop lung infections due to the accumulation of thick mucus in their lungs, creating an optimal environment for bacterial growth. Such bacterial infections commonly cause episodes of pulmonary exacerbations (or flares), during which patients experience a sudden worsening of lung function.

Frequent exacerbations can eventually cause a permanent decline in lung function.

Sodium fusidate is an effective, oral antibiotic against the bacteria Staphylococcus aureus, which, according to Arrevus, infects up to 70% of all CF patients. Additionally, the therapy is highly effective against methicillin-resistant Staphylococcus aureus (MRSA), which is an especially difficult-to-treat infection.

In fact, sodium fusidate is the first line agent for the treatment of MRSA in both the United Kingdom and Australia.

Arrevus announced in January that the FDA granted a form of priority review to ARV-1801 for the treatment of pulmonary exacerbations in CF patients. With its new orphan drug designation, ARV-1801 will be provided close guidance by the FDA, which will help accelerate the time to marketing approval.

“Orphan drug status is granted to clinical stage therapies targeting conditions that affect fewer than 200,000 patients in the United States,” Carl N. Kraus, MD, president and CEO of Arrevus, said in a press release.

“Cystic fibrosis patients have limited oral options to treat the frequent infections they encounter. We are thrilled to have FDA’s support, and we will continue our efforts to bring ARV-1801 to cystic fibrosis patients,” he added.

Additionally, orphan drug status opens up opportunities for the therapy to be eligible for grants, tax credits, and a seven-year market exclusivity agreement upon marketing approval.

“At the end of the day, what drives disease progression and kills people with cystic fibrosis are infections that become resistant to currently available anti-infective treatments. As our disease advances, we run out of antimicrobial tools to treat the infections ravaging our lungs,” said Emily Kramer-Golinkoff, co-founder of Emily’s Entourage and a CF patient.

“We are in dire need of new oral and inhaled anti-infective treatments, particularly for certain pathogens for which we have very limited oral and inhaled antibiotics to treat, including MRSA,” she added.

Currently, Arrevus is planning to initiate a Phase 2 clinical trial to evaluate ARV-1801 for treating pulmonary exacerbations in patients with CF.