Pendopharm, Inc.

Hypoparathyroidism is a rare endocrine disease characterised by insufficient levels of the parathyroid hormone. Credit: Jo Panuwat D/Shutterstock. Ascendis Pharma has obtained US Food and Drug Administration (FDA) approval for its YORVIPATH (palopegteriparatide) to treat hypoparathyroidism in adults. Hypoparathyroidism is a rare endocrine disease characterised by insufficient levels of the parathyroid hormone, affecting multiple organs. A prodrug of parathyroid hormone (PTH[1-34]), YORVIPATH is administered once a day. It aims to provide a continuous exposure to released PTH over a dosing period of 24 hours. Upon the launch of YORVIPATH, Ascendis intends to introduce a comprehensive suite of patient services through its US Ascendis Signature Access Program. These services assist patients in navigating their treatment journey and offer financial assistance to those eligible. See Also: Bavarian Nordic wins positive CHMP opinion to amend Imvanex approval EAN 2024: anti-CGRPs more effective than onabotulinumtoxinA in chronic migraine prevention in real world The latest approval for YORVIPATH was based on a thorough review of the clinical package for TransCon PTH (palopegteriparatide), submitted with the new drug application. The package included findings from the global Phase II PaTH Forward and Phase III PaTHway trials. Ascendis is currently finalising the manufacturing of the commercial product for the US market. The initial supply will become available in the first quarter of 2025. The company also plans to request FDA approval to commercialise the existing manufactured product. If approved, this could allow for an earlier introduction in the US market by the fourth quarter of 2024. Ascendis Pharma president and CEO Jan Mikkelsen said: “FDA approval of our second TransCon product, YORVIPATH, reflects our values and dedication to following the science to help patients, as well as our unwavering commitment these past years to addressing the significant unmet medical needs of the hypoparathyroidism community in the US. “We are deeply grateful to patients, clinicians and advocates for their many contributions to this important milestone.” In July 2024, the company entered an exclusive distribution agreement with Pendopharm to offer the hypoparathyroidism treatment TransCon PTH in the Canadian market.

Chronic hypoparathyroidism is a rare endocrine disorder characterised by low or inadequate levels of PTH. Credit: Pixel-Shot / Shutterstock. Pharmascience unit Pendopharm has entered an exclusive distribution agreement with Ascendis Pharma to bring the hypoparathyroidism treatment TransCon PTH (palopegteriparatide) to the Canadian market. Pendopharm will be responsible for securing regulatory approval and overseeing the commercialisation of the product in the country. Neither companies has disclosed the financial terms of the deal. Ascendis Pharma executive vice-president and chief commercial officer Camilla Harder Hartvig stated: “We are delighted to partner with Pendopharm to bring TransCon PTH to Canada, contributing to our shared goal of making a meaningful difference in the lives of patients living with chronic hypoparathyroidism.” A parathyroid hormone (PTH) replacement therapy, TransCon PTH is aimed at treating chronic hypoparathyroidism, a rare endocrine disorder characterised by low or inadequate levels of PTH. See Also: HF-158K1 by HighField Biopharmaceuticals for Bladder Cancer: Likelihood of Approval Innovent sells its share in CAR T-cell therapy to IASO Bio for 18% equity The therapy has already secured approval in the European Union, the European Economic Area and Great Britain under the brand name YORVIPATH, specifically for adult patients. TransCon PTH is undergoing development for the same indication in the US, Japan and other international markets. Pendopharm vice-president and GM Jad Isber stated: “This partnership represents Pendopharm’s commitment to bringing innovative medicines that address unmet medical needs to Canadian patients. “We look forward to working with Canadian endocrinologists to make TransCon PTH available to all patients who may benefit from it.” In May 2024, Pendopharm announced the renewal of a distribution agreement with Anika Therapeutics for Cingal, Monovisc and Orthovisc up to 2030.

MONTREAL, July 4, 2024 /CNW/ - Pendopharm, a division of Pharmascience Inc., announced today that it has signed an exclusive distribution agreement with Ascendis Pharma A/S (NASDAQ: ASND) ("Ascendis"), a global biopharmaceutical company headquartered in Copenhagen, Denmark. Under the terms of the agreement, Pendopharm is responsible for regulatory approval and commercialization of Ascendis' hypoparathyroidism treatment TransConTM PTH (palopegteriparatide) in Canada. Financial terms of the agreement are not disclosed. TransConTM PTH is a parathyroid hormone (PTH) replacement therapy designed to treat chronic hypoparathyroidism, a rare condition caused by absent or insufficient levels of PTH. It is approved under the brand name YORVIPATH® in the European Union, European Economic Area, and Great Britain as a PTH replacement therapy for the treatment of adults with chronic hypoparathyroidism1. TransConTM PTH is also in development for the treatment of adults with chronic hypoparathyroidism in the United States, Japan, and other countries. A leading Canadian Parathyroidologist Dr Aliya Khan noted that TransConTM PTH will be the first approved treatment option available to Canadian patients living with chronic hypoparathyroidism. "Hypoparathyroidism can seriously impact quality of life and has potentially life-threatening consequences," Dr Khan said. "To have an option that is able to treat the underlying cause of the disease, moving beyond standard oral calcium and active vitamin D, is a great step forward." "This partnership represents Pendopharm's commitment to bringing innovative medicines that address unmet medical needs to Canadian patients," said Jad Isber, VP and GM of Pendopharm. "We look forward to working with Canadian endocrinologists to make TransConTM PTH available to all patients who may benefit from it." Ascendis Pharma Executive Vice President and Chief Commercial Officer Camilla Harder Hartvig said "We are delighted to partner with Pendopharm to bring TransConTM PTH to Canada, contributing to our shared goal of making a meaningful difference in the lives of patients living with chronic hypoparathyroidism." This new partnership aligns with Pharmascience's ambition to establish itself as a leading organization in the life sciences ecosystem. "We are excited by the addition of TransConTM PTH to our portfolio and the life-changing impact it will have on patients. Our wide range of generic and specialty products demonstrates Pharmascience's continued commitment to improve access to medicines and bring new innovation for patients in Canada and globally" stated Martin Arès, Chief Executive Officer of Pharmascience. © 2024 Pendopharm, division of Pharmascience Inc. All rights reserved. About Pendopharm Pendopharm, a division of Pharmascience Inc., is a leading Canadian specialty pharmaceutical company providing patients with innovative medicines that address unmet medical needs. Its areas of focus are gastroenterology, sports medicine and orthopedics, neurology and cardiology. Pendopharm has extensive experience and knowledge to successfully manage its growing product portfolio. Additional information: About Pharmascience Inc. Pharmascience Inc. is one of the largest pharmaceutical manufacturers in Canada and is headquartered in Montreal, Canada. Pharmascience delivers high-quality medicines to over 50 countries worldwide. Pharmascience's global presence and agile business development model provide healthcare communities around the world with the high-quality Canadian medicines to respond to patients' needs. 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, 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 to learn more. About TransConTM PTH (palopegteriparatide) TransConTM PTH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily, designed to provide parathyroid hormone levels within the normal physiological range across the 24-hour dosing period. YORVIPATH was granted marketing authorization by the European Commission (EC) and European Economic Area (EEA) in November 2023 and by the United Kingdom's Medicines & Healthcare products Regulatory Agency (MHRA) in April 2024 as a PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. In the United States, the U.S. Food & Drug Administration (FDA) has set a PDUFA date of August 14, 2024 to complete their review of Ascendis Pharma's New Drug Application for TransCon PTH for adults with chronic hypoparathyroidism. TransCon PTH is also in development in Japan through Teijin Ltd. and in China through VISEN Pharmaceuticals. About Hypoparathyroidism Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidneys and indirectly on the intestine. Hypoparathyroidism is considered chronic if it persists >12 months following surgery per the 2022 Guidelines from the Second International Workshop.2 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 (70-80%), while other etiologies include autoimmune, genetic, and idiopathic causes. References YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics. Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25. SOURCE Pendopharm Company Codes: NASDAQ-NMS:ASND