The FDA granted accelerated approval for Insmed’s drug Arikayce for the treatment of a lung disease caused by the bacteria group Mycobacterium avium complex, or MAC, in certain patients who have not responded to conventional treatment. And regulators used the moment to flag a new pathway opened at the FDA that is intended to speed up the arrival of a new generation of badly needed antibacterials.
Arikayce is the first drug to be approved under the new Limited Pathway for Antibacterial and Antifungal Drugs, or LPAD, which allows for more streamlined clinical trials involving smaller patient populations.
“As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections. That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors,” said FDA commissioner Scott Gottlieb in a statement issued Friday.
“This approval is the first time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, and it marks an important policy milestone. This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies. 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 in limited populations of patients with unmet medical needs,” Gottlieb added.
Arikayce was approved under the agency’s accelerated approval pathway and has received orphan drug status. The agency said approval was granted based on clinical data that showed 29% of patients on a multi-drug antibacterial regimen who took Arikayce for six months showed no growth of the bacteria in their sputum for at least three consecutive months compared with only 9% of patients who did not add Arikayce to their regimens.
Positive data from that ongoing Phase III study sent shares soaring in early September on hopes that it would lead to a swift FDA approval.
As a condition of Arikayce’s accelerated approval, Insmed will be required to run an additional clinical trial to receive full approval. The company said Friday that it is collaborating with the FDA on the design of the trial, which will likely be a randomized, double-blind, placebo-controlled study evaluating the benefit of Arikayce in patients with Nontuberculous Mycobacterial lung disease caused by MAC.
Arikayce, also known as amikacin liposome inhalation suspension, is delivered through a nebulizer. The product will carry a black box warning advising it may trigger bronchospasms, hypersensitivity pneumonitis, the exacerbation of lung disease, and spitting up of blood. Other side effects include difficulty speaking, cough, damaged hearing, upper airway irritation, musculoskeletal pain, fatigue, diarrhea and nausea, according to the FDA.
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