FIRST AND ONLY TREATMENT FOR CHRONIC HYPOPARATHYROIDISM APPROVED IN AUSTRALIA
YORVIPATH® (palopegteriparatide) has been registered by Australia's Therapeutic Goods Administration (TGA) as the first and only treatment for chronic hypoparathyroidism in adults. The drug, which received Orphan Drug Designation and Priority Review, represents a significant advancement in treating this rare endocrine disease that affects 6.4-37 per 100,000 people globally.
The approval was supported by the Phase 3 PaTHway trial results, where 93% of YORVIPATH-treated patients achieved independence from both active vitamin D and therapeutic doses of elemental calcium. The drug demonstrated favorable safety profiles, with injection site reactions (21.6%), headache (18.7%), and paraesthesia (13.7%) being the most common adverse reactions.
YORVIPATH will be distributed in Australia by Specialised Therapeutics through an exclusive agreement with Ascendis Pharma, following approvals from the US FDA and EMA. The drug will be reviewed by the Pharmaceutical Benefits Advisory Committee in March 2025 for PBS listing.
YORVIPATH® (palopegteriparatide) è stato registrato dall'Australian Therapeutic Goods Administration (TGA) come il primo e unico trattamento per l'ipoparatiroidismo cronico negli adulti. Questo farmaco, che ha ricevuto la designazione di Farmaco Orfano e una Revisione Prioritaria, rappresenta un avanzamento significativo nel trattamento di questa rara malattia endocrina che colpisce da 6,4 a 37 persone ogni 100.000 nel mondo.
L'approvazione è stata supportata dai risultati dello studio di fase 3 PaTHway, dove il 93% dei pazienti trattati con YORVIPATH ha raggiunto l'indipendenza sia dalla vitamina D attiva che dalle dosi terapeutiche di calcio elementare. Il farmaco ha dimostrato profili di sicurezza favorevoli, con reazioni nel sito di iniezione (21,6%), mal di testa (18,7%) e parestesia (13,7%) come le reazioni avverse più comuni.
YORVIPATH sarà distribuito in Australia da Specialised Therapeutics tramite un accordo esclusivo con Ascendis Pharma, dopo le approvazioni della FDA statunitense e dell'EMA. Il farmaco sarà esaminato dal Pharmaceutical Benefits Advisory Committee a marzo 2025 per l'inserimento nella PBS.
YORVIPATH® (palopegteriparatide) ha sido registrado por la Administración de Productos Terapéuticos de Australia (TGA) como el primer y único tratamiento para el hipoparatiroidismo crónico en adultos. El medicamento, que recibió la designación de Medicamento Huérfano y Revisión Prioritaria, representa un avance significativo en el tratamiento de esta rara enfermedad endocrina que afecta a entre 6.4 y 37 de cada 100,000 personas a nivel mundial.
La aprobación se apoyó en los resultados del ensayo de fase 3 PaTHway, donde el 93% de los pacientes tratados con YORVIPATH lograron independencia tanto de la vitamina D activa como de las dosis terapéuticas de calcio elemental. El fármaco demostró perfiles de seguridad favorables, con reacciones en el sitio de inyección (21.6%), cefalea (18.7%) y parestesias (13.7%) siendo las reacciones adversas más comunes.
YORVIPATH será distribuido en Australia por Specialised Therapeutics a través de un acuerdo exclusivo con Ascendis Pharma, tras las aprobaciones de la FDA de EE. UU. y la EMA. El medicamento será revisado por el Pharmaceutical Benefits Advisory Committee en marzo de 2025 para su inclusión en la PBS.
YORVIPATH® (팔로페게티리파타이드)는 호주 치료재품청(TGA)에 의해 성인 만성 저갑상선증에 대한 최초이자 유일한 치료제로 등록되었습니다. 이 약물은 희귀약물 지정 및 우선 검토를 받았으며, 전 세계적으로 10만 명당 6.4~37명이 영향을 받는 이 희귀 내분비 질환 치료에서 중요한 발전을 나타냅니다.
승인은 3상 PaTHway 시험 결과에 의해 뒷받침되었으며, YORVIPATH로 치료받은 환자의 93%가 활성 비타민 D와 치료용 칼슘을 모두 독립적으로 사용할 수 있게 되었습니다. 이 약물은 주사 부위 반응(21.6%), 두통(18.7%), 감각 이상(13.7%)과 같은 일반적인 부작용을 보이며 안전성 프로필이 우수함을 입증했습니다.
YORVIPATH는 미국 FDA와 EMA의 승인을 받은 후, Ascendis Pharma와의 독점 계약을 통해 Specialised Therapeutics에 의해 호주에서 배포될 것입니다. 이 약물은 2025년 3월 PBS 등재를 위해 제약 혜택 자문 위원회에서 검토될 예정입니다.
YORVIPATH® (palopegteriparatide) a été enregistré par l'Administration des produits thérapeutiques d'Australie (TGA) comme le premier et unique traitement pour l'hypoparathyroïdie chronique chez les adultes. Ce médicament, qui a obtenu la désignation de médicament orphelin et une révision prioritaire, représente une avancée significative dans le traitement de cette maladie endocrinienne rare qui touche entre 6,4 et 37 personnes pour 100 000 dans le monde.
L'approbation a été soutenue par les résultats de l'essai de phase 3 PaTHway, où 93 % des patients traités par YORVIPATH ont atteint l'indépendance vis-à-vis de la vitamine D active et des doses thérapeutiques de calcium élémentaire. Le médicament a montré des profils de sécurité favorables, avec des réactions au site d'injection (21,6 %), des maux de tête (18,7 %) et des paresthésies (13,7 %) étant les réactions indésirables les plus courantes.
YORVIPATH sera distribué en Australie par Specialised Therapeutics grâce à un accord exclusif avec Ascendis Pharma, après les approbations de la FDA américaine et de l'EMA. Le médicament sera examiné par le Pharmaceutical Benefits Advisory Committee en mars 2025 pour une inscription au PBS.
YORVIPATH® (palopegteriparatide) wurde von der Therapeutic Goods Administration (TGA) Australiens als die erste und einzige Behandlung für chronische Hypoparathyreoidismus bei Erwachsenen registriert. Das Medikament, das die Orphan Drug Designation und eine Prioritätsprüfung erhielt, stellt einen bedeutenden Fortschritt in der Behandlung dieser seltenen endokrinen Erkrankung dar, die weltweit 6,4 bis 37 von 100.000 Menschen betrifft.
Die Genehmigung wurde durch die Ergebnisse der Phase-3-Studie PaTHway unterstützt, bei der 93% der mit YORVIPATH behandelten Patienten unabhängig von aktivem Vitamin D und therapeutischen Dosen von elementarem Calcium wurden. Das Medikament zeigte günstige Sicherheitsprofile, wobei Injektionsstellenreaktionen (21,6%), Kopfschmerzen (18,7%) und Parästhesien (13,7%) die häufigsten unerwünschten Reaktionen waren.
YORVIPATH wird in Australien von Specialised Therapeutics über eine exklusive Vereinbarung mit Ascendis Pharma vertrieben, nachdem die Genehmigungen von der US FDA und der EMA vorliegen. Das Medikament wird im März 2025 vom Pharmaceutical Benefits Advisory Committee zur PBS-Listung überprüft.
- First and only TGA-approved treatment for chronic hypoparathyroidism in adults
- 93% success rate in Phase 3 trial for reducing dependency on vitamin D and calcium supplements
- Secured distribution rights across multiple Asia-Pacific markets
- Already approved by FDA and EMA, indicating strong regulatory acceptance
- Product not yet available for prescribing pending PBS listing
- Reported adverse reactions in clinical trials affecting up to 21.6% of patients
Insights
The Australian TGA approval of YORVIPATH represents a strategic victory for Ascendis Pharma, establishing a first-mover advantage in the Asia-Pacific region for chronic hypoparathyroidism treatment. The approval's significance is amplified by its Orphan Drug Designation and Priority Review pathway status, which typically indicates both market exclusivity benefits and expedited review processes.
The addressable market, while relatively small with an estimated prevalence of 6.4-37 per 100,000 people globally, presents a compelling opportunity due to several factors:
- High unmet medical need with no other approved treatments
- Chronic nature of the condition requiring long-term therapy
- Potential for premium pricing as an orphan drug
- Distribution agreement covering seven key Asia-Pacific markets
The Phase 3 trial results showcase remarkable efficacy, with 93% of patients achieving independence from conventional therapy. This high success rate, combined with a favorable safety profile, strengthens the drug's position for PBS listing and potential inclusion in treatment guidelines. The reduction in pill burden represents a significant quality-of-life improvement that could drive strong patient adoption.
The upcoming PBAC review in March 2025 is important for commercial success in Australia. PBS listing would not only ensure broader market access but could also set favorable pricing precedents for other markets in the region. The partnership with Specialised Therapeutics provides established distribution channels and local market expertise, potentially accelerating market penetration across Southeast Asia.
- YORVIPATH® (palopegteriparatide) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of chronic hypoparathyroidism in adults1
- First-in-class novel PTH replacement therapy
Australia becomes the first country to obtain Marketing Authorisation for YORVIPATH since FDA approval
Hypoparathyroidism is a rare, complex endocrine disease, affecting an estimated 6.4-37 per 100,000 people globally.5,6 It is an endocrine disorder in which the production of parathyroid hormone (PTH) by the parathyroid glands is abnormally low or absent, causing low levels of calcium (hypocalcaemia) and high levels of phosphorous (hyperphosphataemia) in the blood.7,8 This impacts proper functioning of nerves and muscles, leading to weakness, muscle spasms or cramps, headaches, hair loss, numbness or tingling, and memory problems.8,9 Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extraskeletal calcifications, and cognitive impairment.10
Leading Australian Endocrinologist and Head of the School of Clinical Sciences at Monash Health, Professor Peter Ebeling AO, applauded the TGA approval of YORVIPATH in addressing an unmet medical need for Australian adults diagnosed with chronic hypoparathyroidism.
"Until now, the only treatment option for adults with chronic hypoparathyroidism has been conventional therapy with calcium and active vitamin D supplementation to keep blood calcium levels normal," said Professor Ebeling. "While these treatments help to manage the symptoms of hypoparathyroidism, they do not address the underlying deficiency of PTH and contribute to the significant pill burden for patients."
"The lack of effective therapeutic options has been an urgent medical need in this area, and the local approval of YORVIPATH represents an important advance in the treatment of chronic hypoparathyroidism in
YORVIPATH is a first-in-class PTH replacement therapy. A prodrug of parathyroid hormone (PTH [1-34]), YORVIPATH is administered subcutaneously once daily, with sustained release of active PTH designed to provide PTH levels in the physiological range for 24 hours/day.11
YORVIPATH's approval in
Data from the pivotal trial showed
The safety analysis demonstrated YORVIPATH was generally well-tolerated, with the most frequently reported adverse reactions being injection site reactions (
In addition to the significant short-term and long-term medical complications of chronic hypoparathyroidism, individuals diagnosed with the condition also experience reduced health-related quality of life, including the negative impact of symptoms on their daily life and work productivity.12
Chief Executive Officer of the Australian Thyroid Foundation (ATF), Beverley Garside, acknowledged the significance of the TGA approval in offering adults living with chronic hypoparathyroidism the opportunity to improve their health and wellbeing. However, she reinforced that this is not the time for complacency, as it is just the first step towards securing subsidised access to an important new treatment option for this community.
"As the leading national voice for good thyroid health in
YORVIPATH is being made available in
"The Australian approval of YORVIPATH reflects our values and dedication to applying science to help address the significant unmet medical needs expressed by the hypoparathyroidism community," said Roy Khoury, Vice President, Head of International Markets at Ascendis Pharma. "We are pleased to partner with Specialised Therapeutics to broaden access to this innovative therapy, based on our TransCon technology, which offers a new approach to the treatment of this often-debilitating rare disease."
ST Chief Executive Officer, Mr Carlo Montagner, said the approval of YORVIPATH was a meaningful milestone, but the company would not stop until it achieved reimbursement for eligible Australians with hypoparathyroidism through the Pharmaceutical Benefits Scheme (PBS).
"We are proud to have brought the first and only TGA-registered treatment for chronic hypoparathyroidism to Australians living with the condition so rapidly following the recent FDA and EMA approvals," said Mr Montagner. "Chronic hypoparathyroidism has considerable and life-long wide-ranging impacts on these individuals and we are committed to working with the local hypoparathyroidism community and the Australian Government to ensure YORVIPATH is accessible to eligible patients through the PBS at the earliest opportunity."
YORVIPATH will be reviewed by the Pharmaceutical Benefits Advisory Committee (PBAC) at its first meeting of the year, to be held from 12-14 March 2025. YORVIPATH will be made available for prescribing in
PBS Information: YORVIPATH is not listed on the Pharmaceutical Benefits Scheme (PBS). |
About Specialised Therapeutics
Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in
Additional information can be found at www.stbiopharma.com
About Ascendis Pharma
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, 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 Europe and the United States. Please visit: ascendispharma.com to learn more.
About Hypoparathyroidism10
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 intestines. 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 PaTHway12
The PaTHway trial was a double-blind, placebo-controlled, 26-week, Phase 3 study that assessed the efficacy and safety of PTH replacement therapy with palopegteriparatide for individuals with hypoparathyroidism. Participants (n=84) were randomised 3:1 to once-daily palopegteriparatide (initially 18 μg/d) or placebo, both co-administered with conventional therapy.
The study drug and conventional therapy were titrated according to a dosing algorithm guided by serum calcium. The composite primary efficacy endpoint was the proportion of participants at week 26 who achieved normal albumin-adjusted serum calcium levels (8.3–10.6 mg/dL), independence from conventional therapy (requiring no active vitamin D and ≤600 mg/d of calcium), and no increase in study drug over 4 weeks before week 26. At week 26,
About YORVIPATH® (palopegteriparatide)
YORVIPATH® (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug providing sustained release of active PTH. It was approved by the European Union as a parathyroid hormone (PTH) replacement therapy for the treatment of adults with chronic hypoparathyroidism15 and
Yorvipath® is a registered trademark of Ascendis Pharma A/S (NASDAQ: ASND).
References:
- Therapeutic Goods Administration. Australian Register of Therapeutic Goods (ARTG). Available at: https://www.tga.gov.au/australian-register-therapeutic-goods. [Accessed Dec 2024].
- YORVIPATH (palopegteriparatide) AU Product Information.
- Therapeutic Goods Administration. Prescription medicine determinations and designations: Notice for palopegteriparatide (Specialised Therapeutics Pharma Pty Ltd) – Orphan drug designation. Available at: https://www.tga.gov.au/resources/designations-determinations/notice-palopegteriparatide-specialised-therapeutics-pharma-pty-ltd. [Accessed Dec 2024].
- Therapeutic Goods Administration. Prescription medicine determinations and designations: Notice for palopegteriparatide (Specialised Therapeutics Pharma Pty Ltd) – Priority review. Available at: https://www.tga.gov.au/resources/designations-determinations/notice-palopegteriparatide-specialised-therapeutics-pharma-pty-ltd-0. [Accessed Dec 2024].
- Siggelkow H, Pacaud D, et al. Clin Endocrinol (Oxf). 2020;92(2).
- Bjornsdottir S, Sikjaer T, et al. J Bone Miner Res. 2022;37(12).
- Australian Thyroid Foundation. Thyroid Facts: Parathyroid Glands – Hypoparathyroidism. Available at: https://thyroidfoundation.org.au/Parathyroid-Glands [Accessed Dec 2024].
- National Organization for Rare Disorders. Disease Overview: Hypoparathyroidism - Symptoms, Causes, Treatment, 14 August 2024. Available at: https://rarediseases.org/rare-diseases/hypoparathyroidism/ [Accessed Dec 2024].
- Endocrine Society. Endocrine Library: Patient Resources; Hypoparathyroidism, 24 January 2022. Available at: https://www.endocrine.org/patient-engagement/endocrine-library/hypoparathyroidism [Accessed Dec 2024].
- Bilezikian JP. J Clin Endocrinol Metab. 2020;105(6):1722-1736. doi:10.1210/clinem/dgaa113.
- YORVIPATH (palopegteriparatide) US Prescribing Information.
- Khan AA, Bilezikian JP, Cusano NE, et al. J Bone Miner Res. 2023;38(1).
- US Food and Drug Administration. FDA approves new drug for hypoparathyroidism, a rare disorder, 9 August 2024. Available at: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-drug-hypoparathyroidism-rare-disorder [Accessed Dec 2024].
- European Medicines Agency. Yorvipath: Palopegteriparatide, 17 November 2023. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/yorvipath [Accessed Dec 2024].
- YORVIPATH (palopegteriparatide).EU Summary of Product Characteristics.
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SOURCE Specialised Therapeutics Asia
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