FDA CRL - Camargo Pharmaceutical Services

Chiasma, Inc., through its authorized agent Camargo Pharmaceutical Services, received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA 208232) for Mycapssa (octreotide) capsules. The FDA, operating under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act, outlined two main reasons for its decision. Firstly, unresolved deficiencies were identified during a recent inspection of a manufacturing facility, requiring satisfactory resolution. Secondly, the application failed to provide substantial evidence of Mycapssa's effectiveness. Specifically, the agency noted a lack of bioequivalence between Mycapssa and Sandostatin IR, alongside missing comparative pharmacokinetic and pharmacodynamic data. The pivotal study, CH-ACM-01, was deemed insufficient. Its single-arm, open-label design could not definitively attribute observed effects to Mycapssa, largely due to potential confounding from patients' prior acromegaly treatments and residual therapeutic effects. The FDA also highlighted an overall worsening of disease control, evidenced by rising IGF-1 levels, and criticized the primary efficacy endpoint criterion for being insensitive and selected late in the study. For approval, Chiasma must address the manufacturing facility issues and generate robust data demonstrating Mycapssa's efficacy, likely requiring further clinical studies to characterize its pharmacological profile and confirm sustained clinical benefit.