FDA Approves of Chiasma’s Oral Somatostatin Analog for the Treatment of Acromegaly

Rare Daily Staff

The U.S. Food and Drug Administration approved Mycapssa capsules for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide, both injectable somatostatin analogs.

Acromegaly is a rare chronic disease often caused by a benign pituitary tumor and characterized by excess production of growth hormone and insulin-like growth factor-1 hormone that is frequently treated with chronic burdensome injections. If left untreated, acromegaly can lead to serious, and sometimes life-threatening medical conditions such as type 2 diabetes, hypertension, respiratory disorders, and cardiac and cerebrovascular disease. Chiasma estimates that approximately 8,000 patients are on injectable somatostatin analogs (SSA) in the United States.

Mycapssa is the first and only oral SSA approved by the FDA and the first product approved by the FDA developing using Chiasma’s proprietary TPE technology, which is designed to convert selected peptide-based injectables into oral formulations. Although most peptides are degraded in the stomach, Mycappssa is protected by an enteric coating so that it can safety reach the small intestine for absorption into the bloodstream.

“The FDA approval of Mycapssa represents a major therapeutic advancement for people with acromegaly and validation of our TPE delivery platform,” said Raj Kannan, CEO of Chiasma.

Mycapssa is indicated as a long-term treatment for people with acromegaly, who will now be able to take a pill instead of enduring chronic injections.

“People living with acromegaly experience many challenges associated with injectable therapies and are in need of new treatment options,” said Jill Sisco, president of the Acromegaly Community. “The entire acromegaly community has long awaited oral therapeutic options and it is gratifying to see that the FDA has now approved the first oral SSA therapy with the potential to make a significant impact in the lives of people with acromegaly and their caregivers.”

The FDA approval of Mycapssa was based on the positive results of a randomized, double-blind, placebo-controlled, nine-month phase 3 clinical trial of octreotide capsules, which met the primary endpoint and all four secondary endpoints, as well as safety data from all of Chiasma’s phase 3 clinical trials of Mycapssa. The results showed that 58 percent of patients on Mycapssa maintained IGF-1 response compared to 19 percent of patients on placebo after nine months. Adverse reactions included cholelithiasis and complications of cholelithiasis; hyperglycemia and hypoglycemia; thyroid function abnormalities; cardiac function abnormalities; and decreased vitamin B12 levels and abnormal Schilling’s tests.

Chiasma expects Mycapssa to be commercially available in the fourth quarter of 2020 subject to FDA’s timely approval of a planned manufacturing supplement to the approved NDA. Chiasma plans to scale-up its customer facing team in sales, patient services and market access to approximately 45 employees. To help patients switch to Mycappssa, Chiasma plans to offer an array of patient support services. The company also says it will price Mycapssa competitively with the fastest growing SSA in the U.S. acromegaly market.