Chiasma Chief Says Firm May Challenge FDA Over Rare-Disease Drug Rejection

Chiasma Chief Says Firm May Challenge FDA Over Rare-Disease Drug Rejection May 12, 2016
By Alex Keown, Breaking News Staff

WALTHAM, Mass. – Executives at Chiasma, a biopharmaceutical company, plan to challenge the U.S. Food and Drug Administration over its rejection of the company’s New Drug Application for Mycapssa, a maintenance treatment for adult patients with acromegaly, the Boston Business Journal reported.

Last month, the FDA sent Massachusetts-based Chiasma a Complete Response Letter regarding its oral drug candidate Mycapssa (octreotide). Regulators did not believe the company’s application had provided substantial 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 III clinical trial. Regulators requested Chiasma “conduct a randomized, double-blind and controlled trial that enrolls patients from the United States and be of sufficiently long duration to ensure that control of disease activity is stable at the time point selected for the primary efficacy assessment.”

Mark Leuchtenberger, chief executive officer of Chiasma, said in a conference call that company leadership disagrees with the regulatory board, saying the information included in the NDA “demonstrates the efficacy and safety of Mycapssa,” the Boston Business Journal reported. As a result, Chiasma’s leadership plans to meet with representatives from the FDA by the end of June to press their case.

“If the outcome is not acceptable to us, we may pursue dispute resolution with the agency,” he said in the call, according to the Boston Business Journal. “For the moment, however, we believe that further dialogue with the agency could prove productive.”

Shares of Chiasma plummeted after the FDA rejected Mycapssa. Share prices fell from $10.17 to $3.75 in April. Since then, the stock has continued to drop. It is currently trading at $2.69 per share.

If Mycapssa is ultimately approved, it could be the first oral treatment for acromegaly. Octreotide capsules have been granted orphan designation in 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 III trial comparing the safety and efficacy of Mycapssa to monthly somatostatin analog injections to support a potential Marketing Authorization Application with the European Medicines Agency.

There are an estimated 69,000 individuals with acromegaly worldwide. Common features of acromegaly are 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.

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