After Insmed ($INSM) revealed in March that its inhaled antibiotic failed to meet the primary endpoint in a Phase II trial, more than a few analysts and market watchers looked askance at the company's plans to ask the FDA to give it a breakthrough therapy designation. But, three months later, the New Jersey biotech has prevailed upon regulators, picking up the agency's coveted guarantee of a speedy review for its well-traveled treatment.
The agency granted Insmed's request on Arikayce, or liposomal amikacin for inhalation, giving the drug a breakthrough tag for treatment-refractory nontuberculous mycobacterial lung disease, a chronic ailment that affects about 50,000 people in the U.S., the company said.
In the Phase II study, the antibiotic missed its primary of reducing in bacterial density used as a measure of infection. However, in a secondary goal, 11 of the 44 patients taking Arikayce turned in negative cultures at the end of the study, a statistically significant improvement over the placebo arm.
That was enough to sway the FDA, which, in granting the breakthrough designation, agrees to give Insmed a front-row seat in the regulatory process as it considers Arikayce's potential benefits.
The news sent Insmed's shares up about 32% on Wednesday morning, not quite enough to regain their $19.64 high before Arikayce's Phase II slip in March.
Since its institution in 2012, the FDA's breakthrough tag has led to early approvals for companies including Roche ($RHHBY), Johnson & Johnson ($JNJ) and Gilead Sciences ($GILD). But, as Novartis ($NVS) found out with its now-rejected cardio drug serelaxin, a front-row seat with regulators doesn't guarantee they'll like what they see.
Arikayce, Insmed's only drug, has endured numerous trial failures and a clinical hold in its years in development, dragging the biotech's shares up and down as it has continuously pivoted to new potential indications for its inhaled antibiotic.
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