Ascendis Pharma Announces Submission of Supplemental Biologics License Application to FDA for TransCon™ hGH for the Treatment of Adults with Growth Hormone Deficiency
September 30 2024 - 8:30AM
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/SAscendis 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 DeficiencyGrowth
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 |
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Patti Bank |
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ICR Westwicke |
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+1 (415) 513-1284 |
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patti.bank@westwicke.com |
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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
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