Case Study: USFDA The Limited Population Pathway and approval of Arikayce

The US Food and Drug Administration late Friday granted accelerated approval of Arikayce (amikacin liposome inhalation suspension), developed by US biotech Insmed, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options.

What is The Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD)?

LPAD allows drugs to be approved based on smaller, shorter clinical trials, and gives the FDA license to approve drugs that that might otherwise be problematic, such as those with side effects, on the grounds that they tackle an unmet medical need.

Arikayce is the first product approved via LAPD.
Arikayce is the first product approved via the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD).

Arikayce is the first and only therapy approved in the USA specifically for patients with MAC lung disease, a chronic and debilitating condition that can significantly increase patient morbidity and mortality.

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. 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.

As a condition of accelerated approval, Insmed is collaborating with the FDA on the design of an additional clinical study to support full approval. The study design is currently under discussion with the agency and is proposed to be a randomized, double-blind, placebo-controlled clinical trial to assess and describe the clinical benefit of Arikayce in patients with NTM lung disease caused by MAC.