Novel delivery of an old antibiotic nabs first-ever nod under new FDA pathway

Stamp with blue ink that says "FDA Approved"
Insmed's Arikayce has become the first drug approved under a novel FDA pathway designed to speed development of antibiotics. (Olivier Le Moal/Getty Images)

The FDA just doled out the first nod under a novel pathway aimed at accelerating development of antibacterial and antifungal drugs. Insmed's Arikayce is the recipient. 

The active component of antibiotic Arikayce is amikacin, a decade-old antibiotic that’s on the WHO’s essential drug list. But instead of administering it intravenously, Insmed created an inhaled formulation to treat Mycobacterium avium complex (MAC) lung disease as part of combo regimen for patients who don't respond to conventional treatment.

New Jersey-based Insmed is launching the drug—its first commercial product—in the coming weeks, and it's priced it at $363 per vial, or $132,495 annually if taken daily as directed. On a conference call Friday, CEO Will Lewis said the price “reflects the balance of value and access,” as the company has spent over $1 billion in the past 15 years to develop the drug.

The FDA has blessed several antibiotics lately, including Tetraphase’s novel tetracycline Xerava and Achaogen’s Zemdri. But Arikayce is the first one approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, which was established under the 21st Century Cures Act to advance novel drugs for relatively rare but life-threatening infections. The pathway allows smaller, shorter or fewer clinical trials to support a drug’s approval.

RELATED: Targeting a market Big Pharma's fleeing, Tetraphase wins FDA nod for antibiotic Xerava

Arikayce uses a liposomal technology dubbed Pulmovance and a nebulizer to deliver the drug directly to the lung’s infection area, and it therefore achieves prolonged release of amikacin while limiting systemic exposure, according to the company.

When paired with guideline-based therapy (GBT) in a phase 3 study conducted in 18 countries, the drug improved sputum culture conversion rates by month six. To be specific, the Arikayce-GBT arm eliminated lung disease caused by MAC in 29% of patients, compared with 9% in the GBT arm.

Per Insmed’s estimate, around 10,000 to 15,000 refractory MAC patients live in the U.S. A field sales team of 72 therapeutic specialists has been working to reach their physicians since April, the company’s chief commercial officer, Roger Adsett, said on a conference call Friday.

The drug’s label carries a Boxed Warning regarding the increased risk of certain respiratory conditions. Patients will be provided a delivery kit and a step-by-step guide to using the product, as well as the safety information, the company said.

Because it's approved under the accelerated approval pathway, Insmed needs to provide the FDA results from an additional clinical study to support a full nod. The company said it’s working with the agency on a trial design that’ll evaluate Arikayce’s efficacy in the previously untreated patients.