The U.S. Food and Drug Administration ( FDA ) has approved Mycapssa ( Octreotide ) capsules for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with Octreotide or Lanreotide.
Mycapssa is the first and only oral somatostatin analog ( SSA ) approved by the FDA.
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 phase 3 clinical trials of Mycapssa.
Important adverse reactions were: 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 CHIASMA OPTIMAL trial was a randomized, double-blind, placebo-controlled, nine-month clinical trial of octreotide capsules. The trial enrolled 56 adult acromegaly patients whose disease was biochemically controlled by injectable Somatostatin analogs ( Octreotide or Lanreotide ) based upon levels of IGF-1, a byproduct of increased growth hormone ( GH ), levels caused by acromegaly ( average IGF-1 less than or equal to 1.0 × upper limit of normal [ ULN ] ). The patients also had confirmed active acromegaly following their last surgical intervention based upon an elevated IGF-1 at that time of greater than or equal to 1.3 × ULN.
Patients were randomized on a 1:1 basis, to Octreotide capsules or placebo. Patients were dose titrated from 40 mg per day ( equaling one capsule in the morning and one capsule in the evening ) to up to a maximum of 80 mg per day ( equaling two capsules in the morning and two capsules in the evening ).
The primary endpoint of the trial was the proportion of patients who maintained their biochemical response at the end of the nine-month, double-blind, placebo-controlled period as measured using the average of the last two IGF-1 levels less than or equal to 1.0 × ULN ( assessed at weeks 34 and 36 ).
Hierarchical secondary endpoints have included: proportion of patients who maintain GH response at week 36 compared to screening; time to loss of response: IGF-1 of 2 consecutive visits is more than 1.0 × ULN; time to loss of response: IGF-1 of 2 consecutive visits is greater than or equal to 1.3 × ULN; and proportion of patients requiring reversion to prior treatment. ( Xagena )
Source: Chiasma, 2020