Executives at Chiasma, a Massachusetts-based biopharmaceutical company, plan to challenge the FDA over its rejection of the company’s new drug application (NDA) for octreotide (Mycapssa), an oral maintenance treatment for adults with acromegaly, according to an article posted on the BioSpace website.
Last month, the FDA sent Chiasma a complete response letter stating that the company’s application had not provided sufficient evidence of efficacy to warrant approval. The FDA advised the company to conduct another clinical trial to overcome this deficiency.
In its letter, the FDA expressed concerns regarding certain aspects of the company’s single-arm, open-label, phase 3 clinical studies. The agency requested that Chiasma conduct a randomized, double-blind, controlled trial that enrolls patients in the United States and that is of sufficient duration to ensure that control of disease activity is stable at the time point selected for the primary efficacy assessment.
In a recent conference call, an executive at Chiasma stated that the company disagrees with the FDA’s decision, asserting that information included in the NDA “demonstrates the efficacy and safety of Mycapssa.” As a result, Chiasma’s leadership plans to meet with representatives from the agency by the end of June to present their case.
If octreotide is ultimately approved, it would be the first oral treatment for acromegaly. Octreotide capsules were granted orphan designations in both the United States and the European Union for the potential treatment of acromegaly. While Chiasma plans to appeal the FDA ruling, the company is conducting another phase 3 study to compare the safety and efficacy of octreotide with that of monthly somatostatin analog injections to support a potential marketing authorization application with the European Medicines Agency.
An estimated 69,000 individuals worldwide have acromegaly, which is caused by a pituitary tumor. Common features of the disease include facial changes, intense headaches, joint pain, impaired vision, and enlargement of the hands, feet, tongue, and internal organs. Serious health conditions associated with the progression of acromegaly include type-2 diabetes, hypertension, respiratory disorders, and cardiac and cerebrovascular disease. Current treatment options include surgery to remove the pituitary tumor or radiation therapy, which destroys the tumor cells.