FDA Clears Antibacterial Arikayce for MAC Lung Disease

Megan Brooks


October 01, 2018

The US Food and Drug Administration (FDA) has approved amikacin liposome inhalation suspension (Arikayce, Insmed) for the treatment of Mycobacterium avium complex (MAC) lung disease in adults with limited or no alternative treatment options.

Arikayce is the first drug approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) established by Congress under the 21st Century Cures Act to speed up the development and approval of antibacterial medications to treat serious or potentially lethal infections in a limited population of patients with unmet need.

"We're seeing a lot of early interest among sponsors in using this new pathway, and it's our hope that it’ll spur more development and approval of antibacterial drugs for treating serious or life-threatening infections in limited populations of patients with unmet medical needs," FDA Commissioner Scott Gottlieb, MD, said in a news release announcing approval, which he called "an important policy milestone."

The approval of Arikayce was based on results of the phase 3 CONVERT randomized controlled trial involving patients with refractory nontuberculous mycobacterial lung disease caused by MAC. The trial, which is ongoing, has shown that Arikayce improves sputum culture conversion rates when combined with guideline-based therapy.

By the sixth month of treatment, 29% of patients treated with Arikayce plus a background multidrug antibacterial regimen had no growth of mycobacteria in their sputum cultures for 3 consecutive months compared with 9% of patients on guideline-based therapy without Arikayce.

Arikayce is administered once daily using the Lamira Nebulizer System (PARI Pharma GmbH).

Arikayce has a boxed warning about the increased risk of respiratory conditions including hypersensitivity pneumonitis, bronchospasm, exacerbation of underlying lung disease, and hemoptysis that have led to hospitalizations in some cases. Other common side effects in patients taking Arikayce were dysphonia, cough, ototoxicity, upper airway irritation, musculoskeletal pain, fatigue, diarrhea, and nausea.

The approval of Arikayce is a "momentous occasion for all of us living with and advocating for people with MAC lung disease,” Philip Leitman, president of Nontuberculous Mycobacteria Info & Research (NTMir), said in a news release from Insmed.

“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," said Leitman.

The company expects Arikayce to be available in select specialty pharmacies in the coming weeks. Mandy Fahey, director of corporate communications at Insmed, told Medscape Medical News, "The wholesale acquisition cost of Arikayce is $363 per vial. The annual cost of therapy when Arikayce is used daily, as directed, is $132,495."

The company's Arikares Support Program provides coordinators to help patients navigate the reimbursement process and trainers to help patients use Arikayce properly. More information on the program is available by calling 1-833-ARIKARE.

The FDA granted Arikayce orphan drug, fast track, breakthrough therapy priority review and qualified infectious disease product (QIDP) designations. QIDP designation is given to antibacterial products that treat serious or life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act.

Arikayce was approved under the FDA's accelerated approval pathway and the company is required to conduct an additional postmarket study to describe the clinical benefits of Arikayce.

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