Chiasma, Inc. (Nasdaq: CHMA), a U.S. late-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company's New Drug Application (NDA) for the marketing and sale of octreotide capsules, an oral drug proposed for the maintenance therapy of adult patients with acromegaly. The FDA is expected to inform the Company of the Prescription Drug User Fee Act (PDUFA) date by the end of August. The PDUFA date is the target date for the FDA to complete its review of the NDA.
"We are very pleased that the FDA has accepted this filing for our octreotide capsules product candidate, as well as conditionally accepted the proposed trade name of Mycapssa™," said Mark Leuchtenberger, chief executive officer of Chiasma. "Based on our Phase 3 results and feedback from patients and healthcare providers, we believe that octreotide capsules have the potential to be an important treatment option for adults living with acromegaly, whose current standard treatment consists of lifelong and painful injections with an array of undesirable side effects. We look forward to working closely with the FDA in the coming months."
The application, submitted on June 15, 2015, is supported by a multicenter Phase 3 study that evaluated patients for biochemical and symptomatic disease control over a period of up to 13 months following treatment with octreotide capsules, comprised of a 7-month core treatment phase and an optional 6-month extension phase. Sixty-five percent of patients in the modified intent to treat (mITT) population who received octreotide capsules twice-a-day were classified as responders, as measured by circulating concentrations of insulin-like growth factor-1 (IGF-1) and growth hormone (GH) at the end of the 7-month core treatment period, the primary endpoint of the trial. Sixty-two percent of patients in the mITT population were classified as responders at the end of the 6-month extension phase. In the study, the severity and incidence of acromegaly symptoms improved while patients were on octreotide capsules, compared to baseline. The Phase 3 results were published in the Journal of Clinical Endocrinology & Metabolism.
The NDA was submitted utilizing the FDA's 505(b)(2) regulatory pathway. The 505(b)(2) pathway enables a sponsor to rely, in part, on the FDA's prior findings of safety and efficacy for a previously approved product, or published literature, in support of the NDA. The FDA has also conditionally accepted the Company's proposed trade name of Mycapssa™, a decision that is expected to be finalized upon potential approval of the NDA.
If approved, octreotide capsules would be the first oral somatostatin analog approved for acromegaly. An oral therapy for acromegaly has the potential to provide an alternative to the chronic injections currently used for a majority of acromegaly patients. These injections have historically been associated with pain and injection-site reactions during or after injection, as well as breakthrough acromegaly symptoms near the end of dosing intervals. Octreotide capsules have been granted orphan designation in the U.S. and the EU for the treatment of acromegaly.
SOURCE Chiasma, Inc.