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Insmed claims first FDA okay for limited population antibiotic

Arikayce is cleared to treat lung disease caused by MAC

Insmed’s Arikayce has become the first antibiotic to be approved under a new FDA pathway that aims to encourage the develop of treatments for rare, serious infections.

Arikayce is an inhaled liposomal formulation of generic aminoglycoside antibiotic amikacin, and has been cleared to treat lung disease caused by Mycobacterium avium complex (MAC) in patients who don’t respond to conventional treatment.

The Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) was set up in 2016 to encourage and hasten the development of new antibiotics or antifungals for that address a limited market, and otherwise may not offer developers a return on their R&D investment.

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.

Before now, patients with nontuberculous mycobacterial (NTM) lung disease who have failed previous therapy have no approved treatment options, leaving them exposed to symptoms such as persistent cough, fatigue, weight loss, night sweats, and occasionally shortness of breath and coughing up of blood. In some cases, particularly older patients, the infection can be life-threatening.

“We need to encourage the development of drugs that can treat resistant infections [and] that means utilising novel tools intended to streamline development and encourage investment into these important endeavours,” commented FDA Commissioner Scott Gottlieb.

“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.

For Insmed, the approval marks the end of a 10-year effort to develop Arikayce, and comes on the strength of interim data from an ongoing phase 3 trial which compared the inhaled antibiotic given alongside guideline-based therapy (GBT) to GBT alone. After six months, MAC infections were eliminated in 29% of the Arikayce plus GBT group, compared to 9% of patients on GBT alone.

“We estimate that as many as 10,000 to 15,000 of NTM MAC patients in the US fail treatment, when using the off-label antibiotic regimen that is the current standard of care,” said William Lewis, Insmed’s chief executive.

Despite the limited patient population, analysts have predicted that Arikayce could become a big product, particularly if it ends up being used off-label for first-line use. For example, Cowen has previously suggested that based on a net price of around $48,000 the drug could make almost $900m in sales by 2026 in the US, with another $700m or so in Europe if approved.

Article by
Phil Taylor

1st October 2018

From: Regulatory

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