Yorvipath (palopegteriparatide) / Ascendis, Teijin, Specialised Therap, Royalty - LARVOL DELTA

Palopegteriparatide (Yorvipath) for hypoparathyroidism. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Endocrine Disorders • Hypoparathyroidism

The calcium-sensing receptor: a comprehensive review on its role in calcium homeostasis and therapeutic implications. (PubMed, Am J Transl Res) - "Current pharmacological agents, including calcimimetics such as cinacalcet and etelcalcetide, have proven effective in managing secondary hyperparathyroidism (SHPT); however, they are associated with side effects such as hypocalcemia. Emerging investigational drugs, including palopegteriparatide and other small molecules, show promise in addressing various calcium-related conditions. Despite challenges that have led to the discontinuation of some drug developments, ongoing research is focused on refining CaSR-targeted therapies to improve efficacy, reduce adverse effects, and enhance patient outcomes."

Journal • Review • Endocrine Disorders • Hypoparathyroidism • Secondary Hyperparathyroidism

Palopegteriparatide use for severe postoperative hypocalcemia (ESPE-ESE 2025) - No abstract available

Endocrine Disorders

Long-Term Efficacy and Safety of Palopegteriparatide Treatment in Adults With Chronic Hypoparathyroidism: 4-Year Results From the Phase 2 PaTH Forward Trial (ESPE-ESE 2025) - No abstract available

Clinical • P2 data • Endocrine Disorders • Hypoparathyroidism

Switch from rhPTH1-84 to TransCon PTH with individual Dose Adjustment in Adult Hypoparathyroidism – results for 40 patients one month after treatment transition. (ESPE-ESE 2025) - No abstract available

Clinical • Endocrine Disorders • Hypoparathyroidism

Switch from rhPTH1-84 to TransCon PTH in patients with hypoparathyroidism normalizes phosphate, magnesium metabolism, and bone turnover. (ESPE-ESE 2025) - No abstract available

Clinical • Endocrine Disorders • Hypoparathyroidism

Palopegteriparatide for the treatment of Autosomal Dominant Hypocalcemia type 1 (ADH1) (ESPE-ESE 2025) - No abstract available

Endocrine Disorders

2024 FDA TIDES (Peptides and Oligonucleotides) Harvest. (PubMed, Pharmaceuticals (Basel)) - "Interestingly, among the strategies employed in recent approvals to enhance stability and/or delivery, the prodrug approach, exemplified by palopegteriparatide and pegulicianine, is emerging as a more targeted and precise therapeutic strategy. Additionally, the Enhanced Stabilization Chemistry (ESC)-GalNAc platform has been expanded for hepatic delivery of a new oligonucleotide drug, olezarsen. Furthermore, novel modifications to the ribose moiety in oligonucleotides, such as the 3'-amino substitution in imetelstat, enhance their stability. This review examines the TIDES approved in 2024 based on their chemical structure, medical targets, modes of action, administration routes, and common adverse effects. In addition, it highlights how the prodrug strategy has improved targeting efficiency and extended the half-lives of the active drugs."

Journal • Review • Developmental Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders

PaTHway: A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Daily in Adults With Hypoparathyroidism (clinicaltrials.gov) - P3 | N=84 | Completed | Sponsor: Ascendis Pharma Bone Diseases A/S | Active, not recruiting ➔ Completed

Trial completion • Endocrine Disorders • Hypoparathyroidism

PTH Substitution Therapy for Chronic Hypoparathyroidism: PTH 1-84 and Palopegteriparatide. (PubMed, Curr Osteoporos Rep) - "According to phase II and phase III studies, palopegteriparatide seems to fill the gaps identified in existing therapies for hypoPT. Palopegteriparatide is the first real replacement therapy for the management of hypoparathyroidism and seems to fill the gaps identified in existing therapies for hypoPT."

Journal • Review • Endocrine Disorders • Hypoparathyroidism

FIRST AND ONLY TREATMENT FOR CHRONIC HYPOPARATHYROIDISM APPROVED IN AUSTRALIA (PRNewswire) - "Independent biopharmaceutical company Specialised Therapeutics (ST) welcomes the registration of YORVIPATH (palopegteriparatide) by the Therapeutic Goods Administration (TGA),''for the treatment of chronic hypoparathyroidism in adults'....It is the first and only medicine to be listed on the Australian Register of Therapeutic Goods (ARTG) for the treatment of chronic hypoparathyroidism....YORVIPATH's approval in Australia was supported by the results of Ascendis Pharma's Phase 3 PaTHway trial..."

Approval • Hypoparathyroidism

Hypoparathyroidism: diagnosis, management and emerging therapies. (PubMed, Nat Rev Endocrinol) - "Palopegteriparatide has now been approved as PTH replacement therapy for hypoparathyroidism. Emerging therapies will also be presented in this Review."

Journal • Review • Endocrine Disorders • Hypoparathyroidism • Immunology • Metabolic Disorders • Nephrology • Renal Calculi • Renal Disease

Endocrinology : what's new in 2024 (PubMed, Rev Med Suisse) - "In this article, we look at a selection of recent developments in various areas of endocrinology. We focus on advances in endocrine pharmacotherapy and endocrine surgery, addressing several areas: a) the thyroid safety of Glucagon-Like Peptide-1 (GLP1) analogues; b) the efficacy of adrenal surgery for mild autonomous cortisol secretion; c) crinecerfont in the management of congenital adrenal hyperplasia in adults and children; d) paltusotin as a novel oral therapy for acromegaly and e) TransCon PTH (palopegteriparatide) as a novel therapy for chronic hypoparathyroidism."

Journal • Acromegaly • Congenital Adrenal Hyperplasia • Endocrine Disorders • Hypoparathyroidism

Management of autosomal dominant hypocalcemia type 1: Literature review and clinical practice recommendations. (PubMed, J Endocrinol Invest) - "We provide an overview of conventional and new treatments for ADH1 patients. Based on these data, we propose practical recommendations to assist clinicians in the management of ADH1 patients."

Journal • Review • Endocrine Disorders • Hypoparathyroidism • Nephrology • Renal Calculi

Palopegteriparatide. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Efficacy and Safety of TransCon PTH in Adults with Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial. (PubMed, J Clin Endocrinol Metab) - "At week 52 of the PaTHway trial, TransCon PTH showed sustained efficacy, safety, and tolerability in adults with hypoparathyroidism."

Journal • P3 data • Endocrine Disorders • Hypoparathyroidism

Yorvipath® - Ersatz für Parathormon. (PubMed, MMW Fortschr Med) - No abstract available

Journal

Measuring treatment impacts on symptoms in adults with hypoparathyroidism: findings from the PaTHway trial. (PubMed, J Patient Rep Outcomes) - P3 | "Findings indicate that TransCon PTH treatment improved participants' physical and cognitive hypoparathyroidism symptoms in meaningful ways, while reducing the daily burden associated with conventional therapy."

Clinical • Journal • Endocrine Disorders • Hypoparathyroidism • Pain • Renal Disease

FDA Approves YORVIPATH (Palopegteriparatide) as the First and Only Treatment for Hypoparathyroidism in Adults (GlobeNewswire) - "Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has approved YORVIPATH (palopegteriparatide; developed as TransCon PTH) for the treatment of hypoparathyroidism in adults....Ascendis is completing manufacturing of commercial product for the U.S. market and anticipates initial supply will be available in the first quarter of 2025. In addition, Ascendis plans to request FDA approval to commercialize existing manufactured product, which, if approved, could be introduced in the U.S. in the fourth quarter of 2024."

FDA approval • Launch US • Endocrine Disorders • Hypoparathyroidism

Palopegteriparatide Improves Skeletal Dynamics in Adults With Chronic Hypoparathyroidism: 3-Year Results From the Phase 2 PaTH Forward Trial (ASBMR 2024) - No abstract available

Clinical • P2 data • Endocrine Disorders • Hypoparathyroidism • Renal Disease

Palopegteriparatide Treatment Improves Renal Function in Adults with Chronic Hypoparathyroidism: 1-Year Results from the Phase 3 PaTHway Trial. (PubMed, Adv Ther) - P3 | "In this post hoc analysis of the PaTHway trial, palopegteriparatide treatment was associated with significantly improved eGFR at week 52 in addition to previously reported maintenance and normalization of serum and urine biochemistries. Further investigation of palopegteriparatide for the preservation of renal function in hypoparathyroidism is warranted."

Clinical • Journal • P3 data • Endocrine Disorders • Hypoparathyroidism • Nephrology • Renal Calculi • Renal Disease

United Kingdom’s MHRA Approves YORVIPATH (palopegteriparatide) in Great Britain for the Treatment of Adults with Chronic Hypoparathyroidism (GlobeNewswire) - "Ascendis Pharma A/S...announced that the United Kingdom’s Medicines & Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for YORVIPATH (palopegteriparatide; developed as TransCon™ PTH) in Great Britain as a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism, and has also granted YORVIPATH orphan drug status. YORVIPATH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily."

European regulatory • Endocrine Disorders • Hypoparathyroidism

Treatment of Hypoparathyroidism by Re-Establishing the Effects of Parathyroid Hormone. (PubMed, Endocrinol Metab (Seoul)) - "Recently, palopegteriparatide (also known as TransCon PTH) has been marketed in Europe and is expected also to be approved in other countries. Furthermore, the treatment of autosomal dominant hypocalcemia type 1 with a calcilytic (encaleret) is also being tested. All in all, improved treatment options are on the way that will likely take the treatment of HypoPT to the next level."

Journal • Endocrine Disorders • Hypoparathyroidism • Renal Disease

FDA Accepts for Review Resubmitted NDA for TransCon PTH (Palopegteriparatide) in Adult Patients with Hypoparathyroidism (GlobeNewswire) - "Ascendis Pharma A/S...announced that the U.S. Food & Drug Administration (FDA) has accepted for review the Company’s resubmitted New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for the treatment of adult patients with hypoparathyroidism. The agency considered the resubmission a complete, class 2 response and set a Prescription Drug User Fee Act (PDUFA) goal date of May 14, 2024."

NDA • PDUFA date • Endocrine Disorders • Hypoparathyroidism

Ascendis Pharma Resubmits NDA for TransCon PTH to the U.S. FDA (GlobeNewswire) - "Ascendis Pharma A/S...announced that it has resubmitted its New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for the treatment of adults with hypoparathyroidism to the U.S. Food & Drug Administration (FDA). The resubmission follows the Type A meeting held with the FDA in late August."

NDA • Endocrine Disorders • Hypoparathyroidism