Why Arikayce Phase2 Study Will Be Successful? PART 3
Currently, there is no drug approved in Europe or the US for treating NTM lung infections, and, as a result, all current drug treatments for NTM are used off-label. Those treatments usually consist of lengthy multi-drug antibiotic regimens, which are usually not efficacious and have poor compliance.
Roden noted that Amikacin has been used to treat NTM, but it is mainly administrated by intravenous infusion, where systemic toxicities limit the effective concentration in the lung and long enough durations of therapy to achieve culture clearance.
Due to the desperate situation in treating pulmonary NTM infections, Arikayce has been granted QIDP status under the GAIN act. This entitles Insmed to regular and ongoing dialogue with the FDA to clarify the adequacy of the clinical dataset and the path forward to registration.
Moreover, physicians are embracing new drugs for NTM as the management of patients with NTM remains challenging, and no new antibiotics have been studied for the treatment of NTM lung infections in multi-center, randomized clinical trials since the late 1990s.
Thus, Arikayce should likely move to phase3 in NTM and if approved, would face fewer competition and be a topline driver for Insmed.