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Despite advancements in rare disease research over the past decade, more than 6,600 rare diseases still lack approved treatments, leaving patients with little or no options for managing their disease.[1] One example is Nontuberculous Mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC), a chronic, debilitating condition that can cause severe, permanent damage to the lungs. Currently, physicians in the U.S. and EU are forced to rely on a combination of drugs that are not approved specifically for the treatment of NTM lung disease and are inadequate for many patients.

Insmed Incorporated, a biopharmaceutical company focused on rare diseases, saw the significant need among the NTM community and developed an approach that integrates a potent antibiotic, amikacin, with a proprietary chemical matrix that potentially alters and significantly enhances the delivery and antibacterial performance of the compound in this specific disease. Amikacin is an established antibiotic that historically has only been available in an intravenous formulation and is associated with severe toxicity that significantly limits its use. Insmed applied its proprietary liposome technology to develop amikacin liposome inhalation suspension, or ALIS, in which amikacin is encapsulated in liposomes and delivered via proprietary inhalation technology directly to the lungs where the NTM infection resides. This novel approach offers the potential for improved efficacy with a manageable side effect profile.

ALIS is currently under review by the U.S. Food and Drug Administration (FDA) and, if approved, will be the first and only inhaled therapy specifically for the treatment of NTM lung disease — as well as Insmed’s first commercial product. While the road from small, research-focused startup to commercial-stage company is never easy, Insmed has embraced its entrepreneurial mentality and kept its true mission front and center — transforming patients’ lives.

While many smaller companies pursue partnerships with the promise of additional resources, market-building power, and established infrastructure, Insmed has developed global capabilities in-house, hiring the right talent in the right places without having to partner with a larger company for support. The company’s hiring process emphasizes cultural fit above all else, carefully screening for employees who are willing to roll up their sleeves and work hard on behalf of patients in need.

Throughout the decade-long development of ALIS, Insmed maintained an eye toward commercialization — and the future — with the goal of bringing ALIS to market independently on a global basis. With the flexibility to make swift and efficient decisions, Insmed has built awareness of NTM lung disease among the medical community and begun forging a path toward faster patient identification and diagnosis in a previously undeveloped market — a significant hurdle for any company in the healthcare space.

Importantly, the robust clinical trial program for ALIS included trial sites in 18 countries, with significant numbers of patients in Europe and Japan to potentially support regulatory pathways in those regions. The company is also exploring collaborations with industry and the scientific and academic communities to further expand its pipeline. With a startup spirit, strong science, and a growing infrastructure, Insmed is well-equipped to continue to develop novel therapies that make a real difference in the lives of patients with rare diseases for years to come.

[1] RARE Diseases: Facts and Statistics. (2017, March 29). Retrieved July 7, 2018, from