Ascendis Pharma A/S Announces U.S. Food and Drug Administration (FDA) Accepts Biologics License Application (BLA) for TransCon™ hGH for Pediatric Growth Hormone Deficiency (GHD)
– Prescription Drug User Fee Act (PDUFA) target action date of
– FDA indicated that it is currently not planning to hold an Advisory Committee Meeting –
“We look forward to engaging with the FDA during its review of our BLA submission for TransCon hGH in pediatric GHD,” said
TransCon hGH is designed to release somatropin with the same mode of action and distribution as once-daily somatropin products, but with a once-weekly injection.1 The BLA for pediatric GHD is supported by the results a clinical development program that included eight clinical trials evaluating safety and efficacy in more than 400 subjects with GHD.
TransCon hGH is an investigational therapy that is not approved for use in any country. There is no long-acting growth hormone treatment approved by the FDA for the treatment of pediatric GHD. TransCon hGH has received orphan designation in both the
About Pediatric Growth Hormone Deficiency (GHD)2
Pediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Children with GHD are not only characterized by short stature, but they also may experience metabolic abnormalities, psychosocial challenges and poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH, which improves growth and overall endocrine health.
About TransCon™ Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action may be maintained. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies.
Ascendis Pharma currently has a pipeline of three independent endocrinology rare disease product candidates in clinical development and is advancing oncology as its second therapeutic area of focus. The company continues to expand into additional therapeutic areas to address unmet patient needs.
Ascendis is headquartered in Copenhagen,
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ PDUFA date of
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1 Sprogøe K, et al. Endocrine Connections. 2017(6): R171-181.
2 Backeljauw PF, et al. Endocrine Disorders in Adolescents. 2014: 292-403.
Source: Ascendis Pharma