FDA Approves First Oral Somatostatin Analog for Acromegaly

Nancy A. Melville


June 29, 2020

The US Food and Drug Administration (FDA) has approved oral octreotide (Mycapssa, Chiasma) delayed-release capsules for the long-term maintenance treatment of patients with acromegaly who previously responded to and tolerated octreotide or lanreotide 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 Acromegaly Community, a patient support group, in a Chiasma press release.

"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 somatostatin analog (SSA) therapy with the potential to make a significant impact in the lives of people with acromegaly and their caregivers," she added.

Acromegaly, a rare, chronic disease usually caused by a benign pituitary tumor that leads to excess production of growth hormone and insulin-like growth factor-1 (IGF-1) hormone, can be cured through the successful surgical removal of the pituitary tumor. However, management of the disease remains a lifelong challenge for many who must rely on chronic injections.

The new oral formulation of octreotide is the first and only oral somatostatin analog approved by the FDA.

The approval was based on the results of the 9-month, phase 3 pivotal CHIASMA OPTIMAL clinical trial, involving 56 adults with acromegaly controlled by injectable SSAs.

The patients, who were randomized 1:1 to octreotide capsules or placebo, were dose-titrated from 40 mg/day up to a maximum of 80 mg/day, equaling two capsules in the morning and two in the evening.

The study met its primary endpoint. Overall, 58% of patients taking octreotide maintained IGF-1 response compared with 19% of those on placebo at the end of 9 months (P = .008), according to the average of the last two IGF-1 levels that were 1 times or less the upper limit of normal, assessed at weeks 34 and 36.  

The trial also met its secondary endpoints, which included the proportion of patients who maintain growth hormone response at week 36 compared with screening; time to loss of response; and proportion of patients requiring reversion to prior treatment.

Safety data were favorable. Adverse reactions to the drug, detailed in the prescribing information, include cholelithiasis and associated complications; hyperglycemia and hypoglycemia; thyroid function abnormalities; cardiac function abnormalities; decreased vitamin B12 levels, and abnormal Schilling's test results.

Results from the clinical trial "are encouraging for patients with acromegaly," said the study's principal investigator, Susan Samson, MD, PhD, of Baylor College of Medicine, in Houston, Texas, in the Chiasma statement.

"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," she added.

Chiasma reports that it expects Mycapssa to be available in the fourth quarter of 2020, pending FDA approval of a planned manufacturing supplement to the approved new drug application.

The company further plans to provide patient support services including assistance with insurance providers and specialty pharmacies and support in incorporating treatment into patients' daily routines.

Despite effective biochemical control of growth hormone, many patients with acromegaly continue to suffer symptoms, mainly due to comorbidities, so it is important that these are also adequately treated, a consensus group concluded earlier this year.

The CHIASMA OPTIMAL trial was funded by Chiasma.

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