– Compassionate use enables treating physicians in Germany to request access to investigational TransCon PTH (palopegteriparatide) for eligible adult patients with hypoparathyroidism
COPENHAGEN, Denmark, June 05, 2023 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has started enrollment for a Compassionate Use Program (CUP) in Germany for TransCon PTH (palopegteriparatide), an investigational prodrug designed to provide sustained release of active parathyroid hormone (PTH 1-34) within the physiological range for 24 hours per day in adult patients with hypoparathyroidism with once-daily administration. The CUP was approved by Germany’s Federal Institute for Drugs & Medical Devices (Bundesinstitut für Arzneimittel & Medizinprodukte, or BfArM).
“We are pleased to offer this pre-approval Compassionate Use Program for TransCon PTH for eligible patients in Germany to address the unmet medical needs of this patient community,” said Moritz Meissner, M.D., Ascendis Pharma’s Head of Medical Affairs Germany. “Because therapy with oral calcium and active vitamin D aims to control and alleviate hypocalcemic symptoms in patients but does not address the underlying cause of hypoparathyroidism – and importantly, does not address PTH physiology and may itself be associated with secondary complications – we remain committed to advancing potential new treatment options for this disease.”
Through the CUP, treating physicians can request TransCon PTH (palopegteriparatide) for eligible adult patients with hypoparathyroidism whose clinical condition, in the opinion of the treating physician, requires PTH treatment with palopegteriparatide, and who cannot be adequately treated with currently approved products or participate in a palopegteriparatide clinical trial.
Physicians in Germany who are interested in the TransCon PTH (palopegteriparatide) CUP can request full information and initiate enrollment by contacting ascendis.medical@mytomorrows.com.
About Compassionate Use in Germany
In Germany, access to medicinal products that have not yet received marketing authorization may be allowed if sufficient indications of the efficacy and safety of the medicinal product exist and if a clinical trial is being conducted on it, or if an application for a marketing authorization has been submitted to the European Medicines Agency (EMA). Information on current compassionate use programs in Germany can be found on the website of the Federal Institute for Drugs & Medical Devices (Bundesinstitut für Arzneimittel & Medizinprodukte, or BfArM) here.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated, global 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, Berlin and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). 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) TransCon PTH’s ability to address the unmet medical needs of eligible patients in Germany, (ii) Ascendis’ ability to advance potential new treatment options for hypoparathyroidism, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated, global 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 its 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, and 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, and TransCon are trademarks owned by the Ascendis Pharma group. © June 2023 Ascendis Pharma A/S.
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