Patients

About Hypoparathyroidism

Hypoparathyroidism is defined by a deficiency of parathyroid hormone (PTH). PTH is produced by four small parathyroid glands that are located on the thyroid. A deficiency or absence of PTH results in decreased calcium and elevated phosphorus levels in the blood4, which are the primary laboratory findings of hypoparathyroidism. Loss of PTH most commonly occurs due to damage or removal of the parathyroid glands during thyroid or neck surgery.

Who is affected?

Hypoparathyroidism predominantly affects women but can also affect men. There are approximately 80,000 and 110,000 people with hypoparathyroidism in the U.S.1,2,3 and E.U., respectively, of which about 80% are women. More than two-thirds of women with hypoparathyroidism are peri- and menopausal women who are at an increased risk of developing osteoporosis, a condition characterized by weak and brittle bones that increases the risk of bone fractures. It is estimated that about 25% of people with hypoparathyroidism have chronic kidney disease or kidney failure.

What are the symptoms?

Clinical manifestations of hypoparathyroidism vary and impact a large number of tissues and organ systems, including the muscles, brain, heart, and kidneys. Despite available treatments, patients frequently experience persistent, life-altering symptoms and reduced quality of life. In addition, they often develop kidney disease and have abnormal bone architecture.

Limitations of current treatments

Conventional therapies for patients with hypoparathyroidism include calcium supplements and activated vitamin D. These therapies do not adequately control symptoms, and long-term treatment with these therapies may result in dangerously high levels of calcium in the urine that can cause kidney stones, chronic kidney disease and impaired kidney function.2

Recombinant human PTH (1-84) is a once daily injection approved for adult patients with hypoparathyroidism who do not respond to conventional therapies. Replacing the missing or deficient PTH hormone is a more physiologic approach to treatment than conventional therapies, but because of its short half-life, rhPTH (1-84) doesn’t adequately regulate levels of calcium in the body for a full 24 hours, resulting in poor symptom control, and continued long-term risk to the kidneys. Consequently, patients continue to experience symptoms with no improvement in their reduced quality of life. Ongoing studies are further evaluating the safety of long-term use of PTH (1-84).2,5,6

Unmet need

More effective therapies that adequately control symptoms for a full 24 hours and that avoid unwanted side effects are urgently needed to restore balance for patients with hypoparathyroidism and enable them to live a normal life.

About Lipodystrophy

Lipodystrophy is an ultra-rare, life-threatening metabolic syndrome, characterized by either a complete or partial loss of fat tissue, that results in the absence of or the inability to respond to the hormone, leptin.

Lipodystrophy may be either inherited or acquired as a result of medications, radiation treatment, immune dysfunction or for unknown reasons. reasons. No cure currently exists for lipodystrophy.7,8

Patients with partial lipodystrophy lose subcutaneous fat (fat just under the skin), especially in the arms and legs, but accumulate fat around the neck, face and abdominal organs. This results in metabolic complications, including the development of diabetes, decreased levels of high density lipoprotein (HDL) cholesterol that protects against heart disease, and elevated triglycerides that can cause pancreatitis and hardening of blood vessels.9, 10 Partial lipodystrophy affects roughly 1,800 patients in the U.S. and E.U.11, and has no approved therapy in the U.S. due to poor efficacy and safety issues. There is one treatment approved for use in the E.U., but it is poorly effective.

Patients with general lipodystrophy have a complete absence of fat, resulting in severe metabolic complications including the development of diabetes, liver failure and elevated triglycerides.11 Although rare, general lipodystrophy affects roughly 700 patients in the U.S. and E.U., and it carries an extreme healthcare cost burden.12

Some available therapies treat specific symptoms of the disease, but fail to target the underlying cause. These same treatments are inefficient in severe forms, and are often associated with unwanted side effects. There is only one FDA-approved treatment for generalized lipodystrophy, but its use carries warnings for the risk of developing certain cancers (lymphomas) or antibodies that may prevent the treatment from working.

Given that no cure exists and conventional treatments fail to re-establish metabolic regulation, safe and effective therapies are greatly needed to treat metabolic complications in patients suffering from this severe disease.

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