Just recently, the FDA created the LPAD initiative to accelerate the approval of antibacterial and antifungal drugs used to treat rare infections, and this September, the first drug under this initiative was approved—Arikayce.
Arikayce is used to treat a rare condition called MAC lung disease, caused by Mycobacterium avium complex (MAC) bacteria commonly found in water and soil.
While this is a monumental approval for those suffering from MAC lung disease, it comes at a price and could cost as much as $132,495 annually.
What is Arikayce for?
Arikayce is the first and only medication used to specifically treat lung disease caused by the mycobacterium avium complex (MAC), and is for use in a limited population of patients who have not responded to conventional antibiotic treatment. Patients are to inhale Arikayce once daily through a nebulizer system.
MAC lung disease typically affects people with a compromised immune system, and can cause weight loss, cough, fever, tiredness, night sweats, and eventually irreversible lung damage.
Does Arikayce have any side effects?
Yes, common side effects include dysphonia (difficulty speaking), cough, hearing damage, upper airway irritation, musculoskeletal pain, tiredness, diarrhea and nausea.
Arikayce also has a black box warning, the strongest warning from the FDA, regarding its potential to cause dangerous respiratory conditions like inflamed lungs, tightening of the airways, exacerbation of underlying lung disease and spitting up blood that can lead to hospitalizations. Speak with your doctor if you are concerned about this warning.
What is the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD)?
Arikayce was approved under both the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) and the Accelerated Approval Pathway. Established by Congress in June of this year, LPAD is a special process used to ensure that treatments are available for rare bacterial infections. The Pathway involves a shorter approval process, possibly with fewer clinical trials; however, the FDA must still confirm that the drug is safe and effective.
Arikayce was also approved under the Accelerated Approval Pathway, which allows quick approval for drugs that treat serious conditions that fill an unmet need. Medications approved under this pathway are approved based on criteria such as a laboratory measurements, radiographic images, physical signs, or other measures that are thought to predict clinical benefit. Using these markers can save valuable time in the drug approval process.
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