Ascendis Pharma A/S Receives European Approval for TransCon™ hGH for Pediatric Growth Hormone Deficiency
14 January 2022 - 12:00AM
Ascendis Pharma A/S (Nasdaq: ASND) today announced that the
European Commission (EC) has granted marketing authorization for
Lonapegsomatropin Ascendis Pharma (developed under the name
TransCon hGH) as a once-weekly subcutaneous injection for the
treatment of children and adolescents ages 3 to 18 years with
growth failure due to insufficient secretion of endogenous growth
hormone (also known as growth hormone deficiency, or GHD). TransCon
hGH is a prodrug of somatropin that provides sustained release of
unmodified somatropin (hGH) at predictable therapeutic levels in
the body.
“We aim to build a leading global brand for TransCon hGH and are
proud to have the first once-weekly growth hormone replacement for
pediatric GHD approved in both the European Union and the United
States,” said Jan Mikkelsen, Ascendis Pharma President and CEO.
“With this approval as a starting point, we look forward to
bringing a broad portfolio of TransCon products to physicians and
patients in Europe.”
The EC approval is based on clinical results submitted in the
Marketing Authorisation Application (MAA), including data from the
Company’s Phase 3 heiGHt, fliGHt and enliGHten Trials, which
collectively treated more than 300 pediatric patients diagnosed
with GHD, as well as data from a non-clinical safety program.
In August 2021, the U.S. Food & Drug Administration approved
TransCon hGH for the treatment of pediatric patients one year and
older who weigh at least 11.5 kg and have growth failure due to
inadequate secretion of endogenous growth hormone. TransCon hGH is
also in development for pediatric GHD in Japan and China.
About Pediatric Growth Hormone Deficiency
Pediatric GHD is a serious orphan disease caused when the pituitary
gland does not produce enough growth hormone. Physiological levels
of growth hormone are required for overall endocrine health and
development of healthy bone, muscle, and adipose tissue. Children
with GHD are characterized by short stature and may also experience
metabolic abnormalities, psychosocial challenges, and an overall
poor quality of life. For decades, the standard of care for GHD has
been a daily subcutaneous injection of somatropin to improve growth
and overall endocrine health.
About Ascendis Pharma A/SAscendis Pharma is
applying its innovative platform technology 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, the company uses its TransCon
technologies to create new and potentially best-in-class therapies.
Ascendis is headquartered in Copenhagen, Denmark, and has
additional facilities in Heidelberg and Berlin, Germany; Palo Alto
and Redwood City, California; and Princeton, New Jersey. Please
visit www.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) Ascendis’ goal
to build a leading global brand for TransCon hGH, (ii) Ascendis’
plans to build commercial capabilities and initiate sales of
TransCon hGH in select EU countries and to deliver a broad
portfolio of TransCon products to physicians and patients in
Europe, (iii) Ascendis’ receipt of additional EC approvals for
TransCon hGH, (iv) Ascendis’ development of TransCon hGH in Japan
and China, (v) Ascendis’ ability to apply its platform technology
to build a leading, fully integrated biopharma company, and (vi)
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 and
distributors to supply TransCon hGH, the SKYTROFA® Auto-Injector
and other study drug for commercial sales in the U.S. and clinical
studies; unforeseen safety or efficacy results in its oncology
programs, TransCon hGH, TransCon PTH and TransCon CNP or other
development programs; unforeseen expenses related to
commercialization of lonapegsomatropin-tcgd in the U.S., the co-pay
program, and the further development of TransCon hGH, expenses
related to the development and potential commercialization of its
oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or
other development programs, selling, general and administrative
expenses, other research and development expenses and Ascendis’
business generally; delays in the development of its oncology
programs, TransCon hGH, TransCon PTH and TransCon CNP or other
development programs related to manufacturing, regulatory
requirements, speed of patient recruitment or other unforeseen
delays; dependence on third party manufacturers to supply study
drug for planned clinical studies; Ascendis’ ability to obtain
additional funding, if needed, to support its business activities
and the effects on its business from the worldwide COVID-19
pandemic. 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’ Annual Report on Form
20-F filed with the U.S. Securities and Exchange Commission (SEC)
on March 10, 2021 and Ascendis’ other future reports filed with, or
submitted to, the SEC. 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. © January 2022 Ascendis Pharma A/S.
Investor Contacts: |
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Media
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Tim Lee |
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Melinda Baker |
Ascendis Pharma |
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Ascendis Pharma |
+1 (650) 374-6343 |
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+1 (650) 709-8875 |
tle@ascendispharma.com |
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media@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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ir@ascendispharma.com |
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