Nearly four years ago, the FDA left for dead Chiasma's pill for abnormal limb and organ growth based on concerns over the design of a late-stage pivotal trial. But now that Chiasma has picked itself up off the floor and scored an FDA nod for its once-scorned drug, that nightmare scenario is a thing of the past.
Chiasma's Mycapssa (octreotide) has become the first FDA-approved oral therapy to treat acromegaly, a rare disorder in which overproduction of human growth hormone leads to abnormal enlargement of patients' extremities and internal organs, the drugmaker said.
Acromegaly, made famous by the late professional wrestler Andre the Giant, is believed to affect around 24,000 U.S. patients at any time.
The FDA approved Mycapssa as an oral version of somatostatin analogs, a group of injectable drugs that slow the overproduction of growth hormones that lead to serious complications in patients with acromegaly. There are around 8,000 U.S. patients each year treated with a somatostatin analog, Chiasma said, usually after undergoing an unsuccessful surgery to remove the pituitary gland tumor that causes growth hormone overproduction.
Mycapssa will launch at a list price before rebates of $5,152 for a 28-day treatment course at a 40-milligram starting dose, Chiasma said during a Friday call with analysts.
Chiasma CEO Raj Kannan applauded Mycapssa's approval as a major step for the small but visible acromegaly community—and a big win for Chiasma's commercial hopes.
"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," Kannan said.
Mycapssa's long-gestating green light comes nearly four years after the FDA knocked down the drug's application for approval based on design concerns around a pivotal phase 3 trial.
That setback caused Chiasma to lay off 33% of its workforce in August 2016 after building up a team of 65 full-time employees to handle the expected commercial launch of the drug. After working through the issues in the FDA's complete response letter, Chiasma launched the successful phase 3 trial that eventually secured the FDA's go ahead.
Mycapssa will help replace monthly injection regimens for patients currently treated with somatostatin analogs and has a similar safety profile to those drugs with the injection site reaction issues. The drug's labeled side effects include gallstone risks, high and low blood sugar levels, abnormal thyroid and heart function, and decreased vitamin B12 levels.
Chiasma is aiming to launch Mycapssa in the fourth quarter pending a "planned manufacturing supplement to the approved NDA," the drugmaker said in a release. Chiasma is also seeking orphan drug designation for Mycapssa.
The company will eventually build out a team of 45 sales professionals to help with the rollout and "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," Chiasma said.
That team will be fleshed out for the planned fourth-quarter full launch, but Chiasma in the meantime will operate with a smaller commercial squad to reach out to to targeted endocrinologists and patient groups in a remote setting as it awaits the FDA's ruling on its manufacturing supplement, the drugmaker said.
Mycapssa' remote launch will aim to move established somatostatin analog patients onto the pill through telehealth consultations with physicians and mobile phlebotomy teams to help with blood testing.
On its call with analysts, Chiasma said Mycapssa's launch would benefit from the small number endocrinologists actively treating acromegaly in the U.S.—roughly 90% of patients are handled by just 1,000 accounts, Chiasma said—and solid prescribing information for patients already on somatostatin analogs.
On the payer side, Chiasma plans to hype Mycapssa's "significant innovation" in the acromegaly market as well as the fact that it will be prescribed as a replacement for existing standard-of-care rather than an addition to payer budgets.
Editor's note: This story has been updated.