FDA Accepts Ascendis Pharma’s Supplemental Biologics License Application for TransCon™ hGH for the Treatment of Adults with Growth Hormone Deficiency
Ascendis Pharma (NASDAQ: ASND) announced FDA acceptance of its supplemental Biologics License Application (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 evaluated 259 adults with GHD aged 23-80 years.
The trial demonstrated TransCon hGH's superiority in primary and key secondary efficacy endpoints at Week 38, showing significant reduction in trunk fat and increased total body lean mass compared to placebo. The treatment was generally safe and well-tolerated, with no discontinuations related to study drug and comparable safety to daily hGH treatment.
Ascendis Pharma (NASDAQ: ASND) ha annunciato l'accettazione da parte della FDA della sua domanda supplementare di licenza biologica (sBLA) per TransCon hGH per il trattamento della carenza di ormone della crescita negli adulti (GHD), con una data PDUFA fissata per il 27 luglio 2025. La domanda è supportata dallo studio fase 3 foresiGHt, che ha valutato 259 adulti con GHD di età compresa tra 23 e 80 anni.
Lo studio ha dimostrato la superiorità di TransCon hGH nei principali endpoint di efficacia primaria e secondaria alla settimana 38, mostrando una riduzione significativa del grasso del tronco e un aumento della massa magra corporea totale rispetto al placebo. Il trattamento è stato generalmente sicuro e ben tollerato, senza interruzioni correlate al farmaco dello studio e con una sicurezza comparabile al trattamento quotidiano con hGH.
Ascendis Pharma (NASDAQ: ASND) anunció la aceptación por parte de la FDA de su Solicitud de Licencia Biológica Suplementaria (sBLA) para TransCon hGH para el tratamiento de la deficiencia de hormona del crecimiento en adultos (GHD), con una fecha PDUFA establecida para el 27 de julio de 2025. La solicitud se apoya en el ensayo fase 3 foresiGHt, que evaluó a 259 adultos con GHD de entre 23 y 80 años.
El ensayo demostró la superioridad de TransCon hGH en los principales objetivos de eficacia primaria y secundaria a la semana 38, mostrando una reducción significativa en la grasa troncal y un aumento de la masa corporal magra total en comparación con el placebo. El tratamiento fue generalmente seguro y bien tolerado, sin interrupciones relacionadas con el fármaco del estudio y con una seguridad comparable al tratamiento diario con hGH.
Ascendis Pharma (NASDAQ: ASND)는 성인 성장 호르몬 결핍증(GHD) 치료를 위한 TransCon hGH의 보완 생물학적 허가 신청서(sBLA)가 FDA의 승인을 받았다고 발표했으며, PDUFA 날짜는 2025년 7월 27일로 설정되었습니다. 이 신청서는 23세에서 80세 사이의 GHD를 가진 259명의 성인을 평가한 foresiGHt 3상 시험에 의해 뒷받침됩니다.
시험은 38주차에서 TransCon hGH의 우수성을 입증하며, 지방 감소와 총 체중 증가를 보여주었습니다. 치료는 일반적으로 안전하고 잘 견딜 수 있었으며, 연구 약물과 관련된 중단이 없었고, 매일 hGH 치료와 비슷한 안전성을 보였습니다.
Ascendis Pharma (NASDAQ: ASND) a annoncé l'acceptation par la FDA de sa demande de licence biologique supplémentaire (sBLA) pour TransCon hGH afin de traiter la carence en hormone de croissance chez les adultes (GHD), avec une date PDUFA fixée au 27 juillet 2025. La demande est soutenue par l'essai de phase 3 foresiGHt, qui a évalué 259 adultes atteints de GHD âgés de 23 à 80 ans.
L'essai a démontré la supériorité de TransCon hGH sur les critères d'efficacité primaire et secondaire à la semaine 38, montrant une réduction significative de la graisse du tronc et une augmentation de la masse corporelle maigre totale par rapport au placebo. Le traitement a été généralement sûr et bien toléré, sans interruptions liées au médicament de l'étude et une sécurité comparable à celle du traitement quotidien par hGH.
Ascendis Pharma (NASDAQ: ASND) gab die Annahme ihres ergänzenden biologischen Antrags (sBLA) durch die FDA für TransCon hGH zur Behandlung des Erwachsenenwachstumshormonmangels (GHD) bekannt, wobei das PDUFA-Datum auf den 27. Juli 2025 festgelegt wurde. Der Antrag wird von der foresiGHt Phase 3-Studie unterstützt, in der 259 Erwachsene im Alter von 23 bis 80 Jahren mit GHD bewertet wurden.
Die Studie zeigte die Überlegenheit von TransCon hGH in Bezug auf primäre und wesentliche sekundäre Wirksamkeitsendpunkte in Woche 38, mit einer signifikanten Reduktion des Rumpfdepotfetts und einer Zunahme der gesamten fettfreien Körpermasse im Vergleich zu Placebo. Die Behandlung war im Allgemeinen sicher und gut verträglich, es gab keine Abbrüche im Zusammenhang mit dem Studienmedikament und die Sicherheit war vergleichbar mit der täglichen hGH-Behandlung.
- FDA acceptance of sBLA for TransCon hGH in adult GHD market expansion
- Successful Phase 3 trial results showing superiority in efficacy endpoints
- Strong safety profile with no treatment-related discontinuations
- Potential market expansion to address undertreated adult GHD population
- Long regulatory timeline with PDUFA date set for July 2025
- Current adult GHD market has low treatment rate (only 5-10% of patients)
Insights
PDUFA goal date is July 27, 2025
COPENHAGEN, Denmark, Dec. 12, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has accepted for review its supplemental Biologics License Application (sBLA) in adult growth hormone deficiency (GHD) for TransCon hGH (lonapegsomatropin-tcgd; marketed as SKYTROFA® for pediatric GHD). The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025.
“This marks another step towards achieving our objective to expand SKYTROFA’s label beyond pediatric GHD and expand its reach to address new groups of patients,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Adult GHD is an undertreated condition associated with significant comorbidities and higher annual healthcare costs compared to the 5
The sBLA submission is based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily human growth hormone (hGH) in adults with GHD. The trial evaluated 259 adults with GHD aged 23 to 80 years old, randomized 1:1:1, titrated to receive a target fixed dose of TransCon hGH, placebo, or daily hGH based on age and oral estrogen intake with approximately equivalent hGH mg/week for TransCon hGH and daily hGH. TransCon hGH demonstrated superiority on its primary efficacy and key secondary efficacy endpoints at Week 38, with TransCon hGH-treated participants showing a statistically significant reduction from baseline in trunk fat and increase in total body lean mass at Week 38 compared to placebo. In the trial, TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily hGH treatment.
About Adult Growth Hormone Deficiency
Growth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression.1
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform 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, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
Forward-Looking Statements
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) the PDUFA goal date for SKYTROFA, (ii) Ascendis’ objective to expand SKYTROFA’s label and reach to address new groups of patients, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations, and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA®, are trademarks owned by the Ascendis Pharma group. © December 2024 Ascendis Pharma A/S.
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1.Hoffman AR, Mathison T, Andrews D, Murray K, Kelepouris N, Fleseriu M. Adult Growth Hormone Deficiency: Diagnostic and Treatment Journeys From the Patients' Perspective. J Endocr Soc. 2022;6(7):bvac077. Published 2022 May 12. doi:10.1210/jendso/bvac077
FAQ
When is the PDUFA date for Ascendis Pharma's TransCon hGH adult GHD application?
What were the key results from TransCon hGH's Phase 3 foresiGHt trial?
How many patients were involved in the TransCon hGH Phase 3 trial for adult GHD?
What percentage of adult GHD patients currently receive treatment?
