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: -

Clinical Trials

US FDA accepts Ascendis' sBLA of TransCon for review to treat adults with growth hormone deficiency

Ascendis Pharma's sBLA for TransCon hGH to treat adult GHD accepted by FDA, with a PDUFA goal date of July 27, 2025. Based on the foresiGHt trial, TransCon hGH showed efficacy and safety, aiming to address the high unmet need in adult GHD treatment.

finanznachrichten.de

a month ago

FDA Accepts Ascendis Pharma's Supplemental Biologics License Application for TransCon hGH for the Treatment of Adults with Growth Hormone Deficiency

Ascendis Pharma announced FDA's acceptance of its sBLA for TransCon hGH in adult GHD, with a PDUFA goal date of July 27, 2025. The submission is based on the foresiGHt trial, showing TransCon hGH's efficacy and safety in adults. Adult GHD affects body composition and health, with Ascendis aiming to expand SKYTROFA's treatment reach.

stocktitan.net

a month ago

Ascendis Pharma's TransCon hGH Scores FDA Filing Acceptance After Superior

Ascendis Pharma announced FDA acceptance of its sBLA for TransCon hGH to treat adult growth hormone deficiency (GHD), with a PDUFA date set for July 27, 2025. The application is supported by the foresiGHt Phase 3 trial, which demonstrated TransCon hGH's superiority in reducing trunk fat and increasing lean body mass, with a safe and well-tolerated profile.

investing.com

2 months ago

Ascendis Pharma's SWOT analysis: stock faces growth hurdles amid pipeline promise

Ascendis Pharma faces challenges with Skytrofa sales but prepares to launch Yorvipath and TransCon CNP, aiming to offset setbacks and drive growth.

m.uk.investing.com

2 months ago

TD Cowen cuts Ascendis Pharma target to $153, keeps buy rating

TD Cowen lowers Ascendis Pharma's price target to $153 from $160, maintaining a Buy rating. Skytrofa's revenue of €47 million missed estimates due to increased sales deductions, prompting a FY24 guidance revision. Ascendis Pharma anticipates presenting Phase 2 data for Turner syndrome in Q4 and launching Yorvipath in the U.S. in mid-January. Regulatory submissions for CNP product are on track for 2025, and a deal with Novo Nordisk offers long-term upside. Ascendis Pharma's Q3 net loss of €99.2 million was lower than expected, but revenue and EPS missed predictions. Citi and Oppenheimer adjust price targets, highlighting potential catalysts and the strategic collaboration with Novo Nordisk.

finance.yahoo.com

2 months ago

Ascendis Pharma Reports Third Quarter 2024 Financial Results

Ascendis Pharma reports Q3 2024 financials: YORVIPATH U.S. launch in mid-January 2025, Q3 revenue €8.5 million ex-U.S.; TransCon CNP NDA submission planned for Q1 2025, MAA for Q3 2025; SKYTROFA Q3 revenue €47.2 million; full-year 2024 SKYTROFA revenue expected €200-€220 million; operating expenses ~€600 million; Novo Nordisk collaboration for metabolic and cardiovascular diseases; Q3 total revenue €57.8 million; R&D costs €73.5 million; SG&A expenses €69.8 million; net loss €99.2 million.

Drug Development Updates

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