FDA approves first and only oral treatment of Acromegaly

The US Food and drug administration has approved first and only oral somatostatin analog octreotide as a long-term maintenance therapy for people with acromegaly.

Octreotide (Mycapssa, Chiasma) is the first product approved by the FDA using Chiasma's Transient Permeability Enhancer (TPE) technology for people who have responded to and tolerated treatment with octreotide injection or lanreotide.

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.

The FDA approval of MYCAPSSA was based on the positive results of the randomized, double-blind, placebo-controlled, nine-month Phase 3 CHIASMA OPTIMAL 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 following important adverse reactions are described in the MYCAPSSA prescribing information: cholelithiasis and complications of cholelithiasis; hyperglycemia and hypoglycemia; thyroid function abnormalities; cardiac function abnormalities; and decreased vitamin B12 levels and abnormal Schilling's tests.

"The FDA approval of Mycapssa represents a major therapeutic advancement for people with acromegaly and validation of our TPE delivery platform," Raj Kannan, CEO of Chiasma, said in the release. "We are grateful to patients, healthcare providers, advocates and clinical trial investigators, as well as our employees who have worked diligently to bring Mycapssa to people with acromegaly. As we move into this next exciting phase as a commercial company, we are prepared to execute on a successful U.S. launch by working with healthcare providers to bring Mycapssa to as many patients who could benefit from it."

Adverse events with octreotide could include cholelithiasis and complications of cholelithiasis; hyperglycemia and hypoglycemia; thyroid function abnormalities; cardiac function abnormalities; and decreased vitamin B12 levels and abnormal Schilling's tests, according to the release.

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, Inc. "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."

"For patients living with acromegaly and for their physicians and nurses, the FDA approval of oral octreotide capsules ushers in a new era of treatment," said Shlomo Melmed, MB, ChB, MACP, executive vice president of Academic Affairs and dean of the Medical Faculty at Cedars-Sinai. "Over the last 30 years treating physicians have come to trust octreotide in the treatment of acromegaly, and an oral alternative allows patients to avoid many of the documented treatment burdens associated with injections."

"Results from the pivotal Phase 3 CHIASMA OPTIMAL clinical trial are encouraging for patients with acromegaly," said Susan Samson, M.D., Ph.D., FRCPC, FACE, principal investigator of the CHIASMA OPTIMAL clinical trial. "Based on data from the CHIASMA OPTIMAL trial showing patients on therapy being able to maintain mean IGF-1 levels within the normal range at the end of treatment, I believe oral octreotide capsules hold meaningful promise for patients with this disease and will address a long-standing unmet treatment need."

The company 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 MYCAPSSA, Chiasma plans to offer an array of patient support services ranging from assistance with insurance providers and specialty pharmacies to giving patients support to help incorporate MYCAPSSA seamlessly into their daily living. The company believes that patients, physicians, nurses, and payers will appreciate the significant benefits provided by MYCAPSSA. To ensure patients have broad access to this innovation, Chiasma plans to price MYCAPSSA competitively with the fastest growing SSA in the U.S. acromegaly market reflecting the value provided by MYCAPSSA to the health care system.

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