MedPath

Lonapegsomatropin

Generic Name
Lonapegsomatropin
Brand Names
Skytrofa, Skytrofa (previously Lonapegsomatropin Ascendis Pharma)
Drug Type
Biotech
Chemical Formula
-
CAS Number
1934255-39-6
Unique Ingredient Identifier
OP35X9610Y
Background

Lonapegsomatropin, also known as TransCon hGH or ACP 001, is a methoxypegylated prodrug of human growth hormone (somatropin) indicated for the treatment of children 1 year and older, weighing at least 11.5 kg, with growth failure due to insufficient growth hormone secretion. Unlike somatropin, which requires daily dosing, lonapegsomatropin can be administered as a single weekly dose.

Lonapegsomatropin was granted FDA approval on 25 August 2021. It was later approved by the European Commission on 13 January 2022.

Indication

In the US, lonepegsomatropin is indicated in the treatment of growth failure due to inadequate growth hormone secretion in patients one year and older who weigh at least 11.5 kg. In Europe, lonepegsomatropin is indicated to treat growth failure in children and adolescents aged from three years up to 18 years due to insufficient endogenous growth hormone secretion or growth hormone deficiency.

Associated Conditions
Growth Failure
Associated Therapies
-
biopharmadive.com
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Novo inks Ascendis deal to develop long-acting GLP-1, other metabolic drugs

Novo Nordisk invests in obesity and metabolic disease, partnering with Ascendis Pharma to use its delivery tech for long-acting GLP-1 drug and other diabetes/cardiovascular products. Novo could pay up to $285M for the GLP-1 drug and $77.5M per additional drug. Ascendis' tech aims to reduce dosing frequency, with Novo focusing on longer-acting agents to improve patient adherence.

Ascendis Pharma submits sBLA for growth hormone deficiency therapy

Ascendis Pharma submitted an sBLA to the FDA for TransCon hGH to treat adult GHD, supported by the foresiGHt Phase III trial results. The trial showed TransCon hGH's efficacy and safety in 259 adults with GHD, demonstrating significant reductions in trunk fat and increases in lean mass.
medwatch.com
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Ascendis Pharma submits FDA application for expanded use of Skytrofa

Ascendis Pharma submits supplemental application to FDA for Skytrofa approval in adults with growth hormone deficiency.
biospace.com
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Ascendis Pharma Announces Submission of Supplemental Biologics License Application to ...

Ascendis Pharma submits sBLA to FDA for TransCon hGH for adult GHD, based on Phase 3 foresiGHt trial results showing efficacy and safety. TransCon hGH aims to address unmet medical needs in GHD treatment.
stocktitan.net
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Ascendis Pharma A/S American Depositary Shares

Ascendis Pharma A/S (ASND) is a biotech company using TransCon technology to develop prodrug therapies, focusing on North America, China, and Europe. Key products include SKYTROFA and YORVIPATH. In 2023, revenue grew to €266.7M, driven by SKYTROFA and an upfront payment. Ascendis aims to launch TransCon PTH in the U.S. in 2024 and achieve operating cash flow breakeven. Vision 2030 includes blockbuster status for three Endocrinology Rare Disease products.
seekingalpha.com
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Ascendis raised to outperform by Oppenheimer ahead of drug launch

Oppenheimer upgrades Ascendis Pharma to outperform, citing valuation and the upcoming U.S. launch of Yorvipath. The firm highlights strong demand for Yorvipath and potential growth in sales, setting a price target at $180.
investing.com
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Oppenheimer upgrades Ascendis Pharma shares on potential drug launches

Oppenheimer upgrades Ascendis Pharma to Outperform with a $180 price target, anticipating the U.S. launch of Yorvipath in Q1 2025 and Phase 3 results for TransCon CNP. The firm sees potential for €1.7 billion in global revenue by 2029 and undervaluation in TransCon CNP. Ascendis Pharma faces challenges with Skytrofa sales but aims for approvals of all three product candidates by end-2025. The company ended Q2 2024 with EUR259 million in cash and forecasts EUR220-240 million in Skytrofa revenue for 2024.
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