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We have three endocrinology rare disease product candidates in clinical trials

We have three endocrinology rare disease product candidates in clinical trials

We built our first pipeline in endocrinology rare diseases because it is an area where we see the potential of TransCon® technology to address unmet needs – replacing missing hormones in a predictable manner while extending their duration of action to potentially improve therapeutic effects.

All three of our endocrinology product candidates in clinical development have best-in-class potential, and each one leverages established biology to help make a difference for patients. But we’re not stopping here.

Committed to endocrinology rare diseases

Consistent with our strategic roadmap for growth, Vision 3x3, we plan to further grow these candidates through label expansion and by establishing global clinical reach, either directly or through partnerships. One example of this commitment is our pursuit of adult growth hormone deficiency as a new opportunity for TransCon hGH (lonapegsomatropin). Another is our strategic investment in VISEN Pharmaceuticals in China.

Our science aims to make a difference for patients, caregivers and healthcare providers as we build a potentially best-in-class endocrinology rare disease pipeline, including TransCon hGH, TransCon PTH and TransCon CNP.

Endocrinology rare disease candidates under investigation

TransCon hGH (lonapegsomatropin)

Growth Hormone Deficiency (GHD)

TransCon hGH (lonapegsomatropin)

GHD is a rare growth disorder that affects not only height but overall health and development. Children with GHD are characterized primarily by growth failure, as well as metabolic abnormalities, psychosocial challenges and poor quality of life.

GHD in adults is associated with abnormal metabolism and body composition, as well as psychological difficulties. If untreated in patients with these symptoms, GHD in adults may lead to decreased bone mineral density, increased risk of cardiovascular mortality, and poor quality of life.

Adult Hypoparathyroidism

Hypoparathyroidism is defined as a decrease or lack of parathyroid hormone, leading to low levels of calcium in the blood and an increase in serum phosphorus.

Calcium and/or vitamin D supplementation can help relieve symptoms, but these treatments can have undesirable side effects including an increased risk of kidney stones and chronic kidney disease.


Achondroplasia is a skeletal disease and the most common form of dwarfism. Those living with the condition may experience severe skeletal complications and comorbidities.

Abnormal skeletal development can lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression. Chronic ear infections due to abnormal eustachian tubes can lead to hearing loss and speech delay.

CNP = C-type natriuretic peptide; hGH = human growth hormone; PTH = parathyroid hormone.


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