Ascendis Pharma Receives Orphan Drug Exclusivity in the U.S. for YORVIPATH® (Palopegteriparatide) for the Treatment of Hypoparathyroidism in Adults
Ascendis Pharma A/S (Nasdaq: ASND) has received Orphan Drug exclusivity from the U.S. FDA for YORVIPATH® (palopegteriparatide) in treating hypoparathyroidism in adults. This grants seven years of market exclusivity in the United States. YORVIPATH, a once-daily prodrug of parathyroid hormone, is designed to provide continuous PTH exposure over 24 hours.
Hypoparathyroidism, affecting an estimated 70,000 to 90,000 people in the U.S., is a rare endocrine disease caused by insufficient parathyroid hormone levels. YORVIPATH is the first and only FDA-approved treatment for adult hypoparathyroidism, addressing the underlying disease.
Ascendis Pharma A/S (Nasdaq: ASND) ha ricevuto l'esclusiva per farmaci orfani dalla FDA statunitense per YORVIPATH® (palopegteriparatide) nel trattamento dell'ipoparatiroidismo negli adulti. Questo conferisce sette anni di esclusiva di mercato negli Stati Uniti. YORVIPATH, un pro-farmaco da assumere una volta al giorno, è progettato per fornire un'esposizione continua all'ormone paratiroideo per 24 ore.
L'ipoparatiroidismo, che colpisce un numero stimato di 70.000 a 90.000 persone negli Stati Uniti, è una malattia endocrina rara causata da livelli insufficienti dell'ormone paratiroideo. YORVIPATH è il primo e unico trattamento approvato dalla FDA per l'ipoparatiroidismo negli adulti, affrontando la malattia sottostante.
Ascendis Pharma A/S (Nasdaq: ASND) ha recibido la exclusividad para medicamentos huérfanos de la FDA de EE. UU. para YORVIPATH® (palopegteriparatide) en el tratamiento de hipoparatiroidismo en adultos. Esto otorga siete años de exclusividad en el mercado en Estados Unidos. YORVIPATH, un profármaco de hormona paratiroidea que se toma una vez al día, está diseñado para proporcionar una exposición continua a la PTH durante 24 horas.
El hipoparatiroidismo, que afecta a un estimado de 70,000 a 90,000 personas en EE. UU., es una enfermedad endocrina rara causada por niveles insuficientes de hormona paratiroidea. YORVIPATH es el primer y único tratamiento aprobado por la FDA para el hipoparatiroidismo en adultos, abordando la enfermedad subyacente.
Ascendis Pharma A/S (NASDAQ: ASND)는 성인 저갑상선증 치료를 위한 YORVIPATH® (팔로펙테리파타이드)에 대해 미국 FDA로부터 희귀약품 독점권을 부여 받았습니다. 이는 미국에서 7년의 시장 독점권을 부여하는 것입니다. YORVIPATH는 매일 한 번 복용하는 부형제로, 24시간 동안 지속적인 PTH 노출을 제공하도록 설계되었습니다.
저갑상선증은 미국에서 약 70,000명에서 90,000명 사이의 사람들에게 영향을 미치는 드문 내분비 질환으로, 갑상선 호르몬 수치가 부족하여 발생합니다. YORVIPATH는 성인 저갑상선증을 위한 FDA 승인 첫 번째이자 유일한 치료제로, 기본 질환을 다루고 있습니다.
Ascendis Pharma A/S (Nasdaq: ASND) a reçu l'exclusivité des médicaments orphelins de la FDA américaine pour YORVIPATH® (palopegteriparatide) dans le traitement de l'hypoparathyroïdie chez les adultes. Cela accorde sept ans d'exclusivité sur le marché aux États-Unis. YORVIPATH, un pro-médicament à prendre une fois par jour, est conçu pour fournir une exposition continue à la PTH pendant 24 heures.
L'hypoparathyroïdie, qui touche environ 70 000 à 90 000 personnes aux États-Unis, est une maladie endocrine rare causée par des niveaux insuffisants d'hormone parathyroïdienne. YORVIPATH est le premier et unique traitement approuvé par la FDA pour l'hypoparathyroïdie chez les adultes, traitant la maladie sous-jacente.
Ascendis Pharma A/S (Nasdaq: ASND) hat von der U.S. FDA die Orphan Drug Exklusivität für YORVIPATH® (Palopegteriparatid) zur Behandlung von Hypoparathyreoidismus bei Erwachsenen erhalten. Dies gewährt sieben Jahre Marktexklusivität in den Vereinigten Staaten. YORVIPATH, ein einmal täglich einzunehmendes Prodrug des Parathormons, ist darauf ausgelegt, über 24 Stunden eine kontinuierliche PTH-Exposition zu gewährleisten.
Hypoparathyreoidismus, der schätzungsweise 70.000 bis 90.000 Menschen in den USA betrifft, ist eine seltene endokrine Erkrankung, die durch unzureichende Parathormonspiegel verursacht wird. YORVIPATH ist die erste und einzige von der FDA zugelassene Behandlung für erwachsene Hypoparathyreoidismus und behandelt die zugrunde liegende Erkrankung.
- Received Orphan Drug exclusivity in the U.S. for YORVIPATH
- Granted seven years of market exclusivity in the United States
- First and only FDA-approved treatment for adult hypoparathyroidism
- Potential to address the underlying disease in 70,000 to 90,000 U.S. patients
- Orphan exclusivity also granted in European Union and other countries
- None.
Insights
The FDA's granting of Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide) is a significant milestone for Ascendis Pharma. This seven-year market exclusivity provides a substantial competitive advantage in the treatment of hypoparathyroidism. The prodrug's unique design, offering continuous 24-hour PTH exposure, addresses a critical need in managing this rare endocrine disorder affecting 70,000 to 90,000 people in the U.S.
As the first FDA-approved treatment for adult hypoparathyroidism, YORVIPATH has the potential to revolutionize patient care. The orphan designation not only reflects the drug's uniqueness but also provides Ascendis with financial incentives, including tax credits and fee waivers, which could accelerate further research and development in this area.
This regulatory achievement significantly strengthens Ascendis Pharma's market position. The seven-year exclusivity period provides a clear runway for revenue generation without direct competition, potentially boosting the company's financial performance. With orphan drug status in multiple regions, including the U.S. and EU, Ascendis has secured a global competitive advantage.
Investors should note that while this news is positive, the true financial impact will depend on YORVIPATH's market penetration and pricing strategy. The addressable market of 70,000-90,000 patients in the U.S. alone presents a significant opportunity. However, factors such as reimbursement policies and potential off-label competition could influence the drug's commercial success.
– U.S. FDA Orphan Drug exclusivity provides seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults
COPENHAGEN, Denmark, Sept. 11, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the United States Food & Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide, developed as TransCon PTH), providing seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults. YORVIPATH is a prodrug of parathyroid hormone (PTH [1-34]), administered once daily, designed to provide continuous exposure to released PTH over the 24-hour dosing period. Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone that impact multiple organs and affects an estimated 70,000 to 90,000 people in the United States.
“YORVIPATH has now been granted orphan exclusivity in the U.S., European Union, and other countries, reflecting the uniqueness of YORVIPATH to address this rare disease,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “As the first and only FDA-approved treatment of hypoparathyroidism in adults, we believe that YORVIPATH has great potential to address the underlying disease and look forward to making it available to patients in the U.S. as quickly as possible.”
TransCon PTH (palopegteriparatide) originally received Orphan Drug Designation from the U.S. FDA in June 2018. The FDA grants orphan designation to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States, and that potentially may be safer or more effective than already approved products. Subject to certain exceptions, orphan designation provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity upon approval of the product in the orphan-designated indication, waiver of FDA user fees, and tax credits for clinical research. The granting of orphan designation does not alter the FDA’s regulatory requirements to establish safety and effectiveness of a drug through adequate and well-controlled studies to support approval and commercialization, nor does it provide any advantage during the regulatory review and approval processes.
About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidneys and indirectly on the intestine. 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. Post-surgical hypoparathyroidism accounts for the majority of cases (70
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.
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) YORVIPATH’s potential to address the underlying disease of hypoparathyroidism, (ii) Ascendis’ intent to make YORVIPATH available to patients in the U.S. as quickly as possible, (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’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 7, 2024 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. © September 2024 Ascendis Pharma A/S.
| Investor Contacts: Tim Lee Ascendis Pharma +1 (650) 374-6343 tle@ascendispharma.com ir@ascendispharma.com | Media Contact: Melinda Baker Ascendis Pharma +1 (650) 709-8875 media@ascendispharma.com |
| Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com |
FAQ
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