Insmed’s drug, Arikayce, is also the first treatment approved specifically for mycobacterium avium complex (MAC) lung disease in adult patients with limited or no alternative treatment options.
The illness is a chronic condition and a course of standard treatment can take more than two years to be effective.
Arikayce is an inhaled formulation of amikacin – an antibiotic that until now, was delivered intravenously. The inhaled form of delivery sees the antibiotic taken up by lung macrophages where the infection resides.
The approval marks a success for the US Food and Drug Administration’s limited population pathway for antibacterial and antifungal drugs (LPAD), designed to help advance treatment for serious of life-threatening infections.
Working with the agency, Insmed is now conducting a confirmatory Phase III trial after its treatment also received accelerated approval.
The incidence of MAC lung disease is currently increasing in the US and Insmed expects that there are between 10,000 and 15,000 people with treatment-resistant nontuberculous mycobacterial lung disease in the US, the most common species of which is MAC.
A spokesperson for Insmed explained that the price of the drug per vial would be $363 (€315) and that with daily dosage this would lead to an annual cost of $132,495 per patient.
The spokesperson went on to say, “Our number one priority is to ensure that appropriate patients have access to Arikayce. We have been in discussion with payers and are confident that both private and public payers will recognize the potential benefits of Arikayce and will provide reimbursement for appropriate patients.”
In regards to expanding the geographical market for the treatment, the spokesperson confirmed that the company is going through the regulatory process in Japan and the EU.