If you’re one of the small number of people living with acromegaly symptoms today (an estimated 60 out of every million people worldwide), we’ve got some good news for you.
The FDA recently accepted Chiasma, Inc.’s filing of a New Drug Application for octreotide capsules. This new oral drug is a maintenance therapy for adults with acromegaly; if approved, it would be the first oral form of a type of therapy called a somatostatin analog, which inhibits the release of GH associated with acromegaly.
Phase III clinical trials showed great promise; beyond just positive patient and doctor feedback, 65% of patients in the initial 7-month phase of the trial showed improvement in the severity and incidence of symptoms, while 62% of patients were responders in the 6-month extension phase.
As Chiasma awaits word of when the FDA will complete its review of the application, they’re moving forward on a possible 2016 launch under the brand name Mycapssa™ (which the FDA conditionally approved) and with the confidence of knowing octreotide capsules for the treatment of acromegaly were granted orphan designation in the U.S. and the EU.
Acromegaly is an extremely rare hormonal disorder that occurs when there’s a benign tumor growing on the pituitary gland; the pituitary gland is responsible for creating the body’s growth hormone (GH), and the tumor forces it to produce too much GH.
The excess GH triggers a chain reaction of events that leads to abnormal growth throughout the body.
Hands and feet are typically the first sign of growth, but over time acromegaly causes the bone structure of the face to change, resulting in a distinctive protruding brow and lower jaw. Left untreated, acromegaly can cause a whole host of serious problems, from arthritis to diabetes to heart disease. Fortunately, acromegaly is usually treatable—although its subtle symptoms make diagnosis tricky.
To date, the most reliable treatments have been surgery to remove the pituitary gland; radiation to target the tumor and lower GH production; and medications that stop or interfere with GH production.
No treatment is fool-proof, though, and success is never guaranteed. That’s why it’s always welcome news when another treatment option inches closer to approval…doubly so for rare diseases like acromegaly.
