Ascendis Pharma Announces European Commission Approval of YORVIPATH® (palopegteriparatide) for the Treatment of Adults with Chronic Hypoparathyroidism

- YORVIPATH (developed as TransCon PTH) is a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism

- Ascendis plans its first EU launch of YORVIPATH in Germany in January 2024

COPENHAGEN, Denmark, Nov. 20, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the European Commission (EC) has granted marketing authorization for YORVIPATH^® (palopegteriparatide) as replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. YORVIPATH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily. Ascendis plans its first European Union (EU) launch of YORVIPATH in January 2024 in Germany.

“Each patient living with chronic hypoparathyroidism faces serious health and quality of life concerns,” said Professor Lorenz C. Hofbauer, Professor of Medicine, Geriatrics, and Endocrinology, Technical University of Dresden. “To treat the underlying cause of disease, these patients need new treatment options that go beyond the limits and risks of conventional therapy, which today consists of oral calcium and active vitamin D.”

The EC approval follows the positive opinion adopted on September 14, 2023 by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending approval of YORVIPATH.

“By focusing on patient need and using science to drive our decisions, we have brought YORVIPATH – our second approved TransCon product – from concept through EU marketing authorization in only eight years and we are preparing to launch it in Germany this coming January,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “Knowing the urgent need that many patients and physicians have expressed for new treatment options, we will continue our work to make YORVIPATH widely available.”

TransCon PTH will be marketed in the EU as YORVIPATH, a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism. TransCon PTH is in development for adults with hypoparathyroidism in the United States, Japan, and other countries.

About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery per the 2016 Endocrine Society Guidelines, 2019 Canadian and International Consensus Statement, and 2022 European Society of Endocrinology Consensus Statement. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Postsurgical hypoparathyroidism accounts for the majority of cases (78%), with other etiologies that include autoimmune disorders, familial disorders, and idiopathic causes.

About Ascendis Pharma A/S
Ascendis 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 Germany (Heidelberg and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). 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) Ascendis’ plans regarding its launch of YORVIPATH in the EU, (ii) Ascendis’ ability to make YORVIPATH widely available, (iii) Ascendis’ ability to apply its innovative platform technology 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, including inflation, the effects on its business from the worldwide COVID-19 pandemic and ongoing conflicts such as that in the region surrounding Ukraine and Russia. 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 February 16, 2023 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 YORVIPATH^® are trademarks owned by the Ascendis Pharma group. © November 2023 Ascendis Pharma A/S.