The first drug specifically to treat patients with Mycobacterium avium complex, a rare and chronic lung disease, has been approved by the FDA. Arikayce is also the first drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, established under the 21st Century Cures Act.
“As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections. That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors,” FDA Commissioner Scott Gottlieb, MD, said in a statement.
Arikayce represents the first drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway), which was established by Congress under the 21st Century Act in order to advance development and approval of antibacterial and antifungal drugs for treating potentially life-threatening infections in a small population with unmet need.
Furthermore, approvals under the LPAD pathway may be supported by streamlined clinical development programs and may incorporate smaller or fewer clinical trials. The labeling of Arikayce features certain statements that establish the drug as safe and effective but only for use in a limited population—which is required for drugs approved under the LPAD pathway.
The drug was also approved under the Accelerated Approval pathway, which the FDA uses to approve drugs for serious or life-threatening disease or conditions where the drug demonstrated an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients.
“Arikayce provides a much-needed treatment for patients with this chronic and life-threatening disease who have not responded to the current standard of care,” said Philip Leitman, president of Nontuberculous Mycobacteria Info & Research. “Many of these patients have been suffering for years and face significant challenges in their day-to-day lives, and we are excited to finally have an approved treatment for them.”
The FDA approved Arikayce based on results from the ongoing phase 3 CONVERT study. The study met its primary endpoint of sputum culture conversion by the sixth month of treatment. The addition of Arikayce to guideline-based therapy eliminated evidence of nontuberculous mycobacterial, which is caused by MAC, in sputum by the sixth month in 29% of patients compared with 9% of patients on guideline-based therapy alone.
The FDA also noted that the Boxed Warning of Arikayce includes increased risk of respiratory conditions, while common side effects include dysphonia, cough, ototoxicity, upper airway irritation, musculoskeletal pain, fatigue, diarrhea, and nausea.