The Food and Drug Administration (FDA) has approved Arikayce (amikacin liposome inhalation suspension for oral inhalation; Insmed) for adults who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy.
Arikayce is the first drug approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs. “This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies,” said FDA Commissioner Scott Gottlieb, MD. “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.”
The approval was based on data from the Phase 3 CONVERT study in patients with refractory MAC lung disease (N=336) as confirmed by at least 2 sputum culture results. Patients were randomized to receive Arikayce plus a background multi-drug antibacterial regimen or background regimen alone.
Results showed that the proportion of patients achieving culture conversion (3 consecutive monthly negative sputum cultures) by the sixth month of treatment was significantly (P<.0001) greater for Arikayce plus background regimen (29%) compared with background regimen alone (8.9%).
Arikayce carries a Boxed Warning describing the risk of increased respiratory reactions including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbations of underlying pulmonary disease. Other side effects in Arikayce-treated patients were dysphonia, cough, ototoxicity, upper airway irritation, musculoskeletal pain, fatigue/asthenia, diarrhea, and nausea.
Arikayce is intended for oral inhalation use and is administered via the Lamira Nebulizer System only. It is available in a 590mg/8.4mL dosage strength in unit-dose vials and is dispensed as a 28-vial kit; each carton contains a 28-day supply of medication. Commercial availability of the product is expected sometime in the fourth quarter of 2018, according to Insmed.
For more information visit Insmed.com.