Ascendis Pharma Announces Submission of Supplemental Biologics License Application to FDA for TransCon™ hGH for the Treatment of Adults with Growth Hormone Deficiency
Ascendis Pharma A/S (Nasdaq: ASND) has submitted a supplemental Biologics License Application (sBLA) to the FDA for TransCon hGH to treat adults with growth hormone deficiency (GHD). This submission aims to expand the label of SKYTROFA®, currently approved for pediatric GHD in the U.S. The sBLA is based on results from the foresiGHt Phase 3 trial, which compared weekly TransCon hGH with weekly placebo and daily hGH in 259 adults with GHD aged 23-80.
The trial demonstrated that TransCon hGH was superior to placebo in reducing trunk fat and increasing total body lean mass at Week 38. It was generally safe and well-tolerated, with no discontinuations related to the study drug and comparable safety to daily hGH. This submission supports Ascendis Pharma's strategy to develop SKYTROFA into a blockbuster product and address the unmet medical need in adult GHD treatment.
Ascendis Pharma A/S (Nasdaq: ASND) ha presentato una domanda supplementare di licenza biologica (sBLA) alla FDA per TransCon hGH per il trattamento di adulti con carenza di ormone della crescita (GHD). Questo invio mira ad ampliare l'etichetta di SKYTROFA®, attualmente approvato per il GHD pediatrico negli Stati Uniti. La sBLA si basa sui risultati del trial fase 3 foresiGHt, che ha confrontato il TransCon hGH settimanale con placebo settimanale e hGH giornaliero in 259 adulti con GHD di età compresa tra 23 e 80 anni.
Lo studio ha dimostrato che TransCon hGH era superiore al placebo nella riduzione del grasso addominale e nell'aumento della massa magra corporea totale alla settimana 38. È stato generalmente sicuro e ben tollerato, senza interruzioni correlate al farmaco dello studio e con una sicurezza comparabile a quella dell'hGH giornaliero. Questo invio supporta la strategia di Ascendis Pharma di sviluppare SKYTROFA in un prodotto di grande successo e rispondere al bisogno medico non soddisfatto nel trattamento del GHD negli adulti.
Ascendis Pharma A/S (Nasdaq: ASND) ha presentado una solicitud suplementaria de licencia biológica (sBLA) a la FDA para TransCon hGH para tratar a adultos con deficiencia de hormona de crecimiento (GHD). Esta presentación tiene como objetivo ampliar la etiqueta de SKYTROFA®, que actualmente está aprobado para GHD pediátrico en EE. UU. La sBLA se basa en los resultados del ensayo fase 3 foresiGHt, que comparó TransCon hGH semanal con placebo semanal y hGH diario en 259 adultos con GHD de entre 23 y 80 años.
El ensayo demostró que TransCon hGH era superior al placebo en la reducción de grasa del tronco y en el aumento de la masa magra corporal total a la semana 38. Fue generalmente seguro y bien tolerado, sin interrupciones relacionadas con el medicamento del estudio y con una seguridad comparable a la del hGH diario. Esta presentación respalda la estrategia de Ascendis Pharma para desarrollar SKYTROFA en un producto exitoso y abordar la necesidad médica no satisfecha en el tratamiento del GHD en adultos.
Ascendis Pharma A/S (Nasdaq: ASND)는 TransCon hGH의 FDA에 대한 보충 생물학적 허가 신청(sBLA)을 제출하였습니다. 이 신청은 현재 미국에서 소아 성장 호르몬 결핍증(GHD)에 대해 승인된 SKYTROFA®의 라벨 확대를 목표로 하고 있습니다. sBLA는 23세에서 80세 사이의 GHD를 가진 259명의 성인에 대한 주간 TransCon hGH, 주간 위약 및 매일 hGH를 비교한 foresiGHt 3상 시험의 결과를 기반으로 하고 있습니다.
시험 결과, TransCon hGH는 제38주에 주위험군에서 지방 감소 및 총체적 체지방 증가에서 위약보다 우수한 성능을 보였습니다. 일반적으로 안전하고 잘 견뎌졌으며, 연구 약물과 관련된 중단이 없었고, 매일 hGH와 유사한 안전성을 보여주었습니다. 이 제출은 SKYTROFA를 블록버스터 제품으로 발전시키고 성인 GHD 치료의 충족되지 않은 의료 수요를 해결하려는 Ascendis Pharma의 전략을 지원합니다.
Ascendis Pharma A/S (Nasdaq: ASND) a soumis une demande de licence biologique complémentaire (sBLA) à la FDA pour TransCon hGH afin de traiter les adultes souffrant d'une carence en hormone de croissance (GHD). Cette soumission vise à élargir l'étiquette de SKYTROFA®, actuellement approuvé pour GHD pédiatrique aux États-Unis. Le sBLA est basé sur les résultats de l'
L'essai a démontré que TransCon hGH était supérieur au placebo pour réduire la graisse du tronc et augmenter la masse corporelle maigre totale à la semaine 38. Il était généralement sûr et bien toléré, sans interruptions liées au médicament de l'étude et avec une sécurité comparable à celle de l'hGH quotidien. Cette soumission soutient la stratégie d'Ascendis Pharma de développer SKYTROFA en un produit phare et de répondre aux besoins médicaux non satisfaits dans le traitement de la GHD chez les adultes.
Ascendis Pharma A/S (Nasdaq: ASND) hat einen ergänzenden Antrag für eine Biologics-Lizenz (sBLA) bei der FDA für TransCon hGH zur Behandlung von Erwachsenen mit Wachstumshormonmangel (GHD) eingereicht. Ziel dieser Einreichung ist es, die Kennzeichnung von SKYTROFA®, das derzeit in den USA für pädiatrisches GHD zugelassen ist, zu erweitern. Das sBLA basiert auf den Ergebnissen der foresiGHt Phase 3-Studie, die wöchentliches TransCon hGH mit wöchentlichem Placebo und täglichem hGH bei 259 Erwachsenen mit GHD im Alter von 23 bis 80 Jahren verglichen hat.
Die Studie zeigte, dass TransCon hGH überlegen war gegenüber Placebo bei der Reduzierung des Rumpf-Fetts und der Erhöhung der gesamten mageren Körpermasse in Woche 38. Es war allgemein sicher und gut verträglich, ohne Studienabbruch aufgrund des Studienmedikaments und mit vergleichbarer Sicherheit wie tägliches hGH. Diese Einreichung unterstützt die Strategie von Ascendis Pharma, SKYTROFA zu einem Blockbuster-Produkt zu entwickeln und den ungedeckten medizinischen Bedarf in der Behandlung von GHD bei Erwachsenen zu adressieren.
- Submission of sBLA for TransCon hGH to treat adult GHD, potentially expanding market reach
- Positive Phase 3 trial results showing superiority over placebo in key efficacy endpoints
- No discontinuations related to the study drug in the Phase 3 trial
- Comparable safety and tolerability profile to daily hGH
- None.
Insights
The submission of an sBLA for TransCon hGH to treat adult growth hormone deficiency (GHD) is a significant development for Ascendis Pharma. This expansion from pediatric to adult GHD could substantially increase the market potential for SKYTROFA. The Phase 3 foresiGHt trial results are particularly noteworthy:
- Demonstrated superiority in primary and key secondary efficacy endpoints
- Statistically significant reduction in trunk fat and increase in total body lean mass compared to placebo
- Comparable safety and tolerability to daily hGH
The unmet medical need in adult GHD is substantial, with the majority of affected adults currently untreated. If approved, weekly TransCon hGH could offer a more convenient option, potentially improving treatment adherence and outcomes. This move aligns with Ascendis Pharma's strategy to develop SKYTROFA into a blockbuster product, which could significantly impact the company's future revenue and market position in the growth hormone space.
The sBLA submission for TransCon hGH in adult GHD represents a potentially significant revenue driver for Ascendis Pharma. Key financial implications include:
- Expanded market: The adult GHD market is largely untapped, offering substantial growth potential.
- Blockbuster potential: Label expansion could help SKYTROFA achieve blockbuster status (
$1 billion + annual sales). - Competitive advantage: Once-weekly dosing may differentiate TransCon hGH from daily alternatives, potentially capturing market share.
- Value leadership: Strengthens Ascendis' position in the U.S. growth hormone market, which could lead to pricing power and improved margins.
Investors should monitor the FDA review process, as approval could significantly impact Ascendis' revenue projections and market valuation. The company's
COPENHAGEN, Denmark, Sept. 30, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration (FDA) for TransCon hGH (marketed as SKYTROFA® (lonapegsomatropin-tcgd) in the U.S. for pediatric growth hormone deficiency) for the treatment of adults with growth hormone deficiency (GHD).
“The sBLA submission for TransCon hGH for adult GHD supports our strategy to develop SKYTROFA into a blockbuster product through label expansion and build upon our value leadership position in the U.S. growth hormone market,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Growth hormone plays a vital role in human health at all ages, and, with research showing that the majority of adults living with growth hormone deficiency are not currently treated for this condition, we believe once weekly TransCon hGH could provide a new potential treatment option to address this unmet medical need.”
The 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 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 patients showing a statistically significant reduction from baseline in trunk fat and increase in total body lean mass at Week 38 compared to placebo.
TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily hGH.
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.
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
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) Ascendis’ intent to develop SKYTROFA into a blockbuster product through label expansion and build upon its value leadership position in the U.S. growth hormone market, (ii) TransCon hGH’s potential to provide a new treatment option to address the unmet medical needs of adults living with growth hormone deficiency, (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.
© September 2024 Ascendis Pharma A/S.
| Investor Contacts: | Media Contact: |
| Tim Lee | Melinda Baker |
| Ascendis Pharma | Ascendis Pharma |
| +1 (650) 374-6343 | +1 (650) 709-8875 |
| tle@ascendispharma.com | media@ascendispharma.com |
| ir@ascendispharma.com | |
| Patti Bank | |
| ICR Westwicke | |
| +1 (415) 513-1284 | |
| patti.bank@westwicke.com | |
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
What is the purpose of Ascendis Pharma's recent sBLA submission for TransCon hGH (ASND)?
What were the key findings from the foresiGHt Phase 3 trial for TransCon hGH (ASND)?
How many participants were involved in the Phase 3 trial for TransCon hGH (ASND)?
What is Ascendis Pharma's strategy for SKYTROFA (TransCon hGH) in the growth hormone market (ASND)?
