Secondary hyperparathyroidism (SHPT) is a common complication of advanced chronic kidney disease (CKD), caused by phosphate retention and vitamin D deficiency, leading to elevated parathyroid hormone (PTH) levels and increased bone and cardiovascular complications. Active vitamin D analogs such as calcitriol and doxercalciferol are used to suppress PTH levels, though they differ in pharmacologic properties and effects on mineral metabolism. Intravenous preparations may offer improved bioavailability and adherence in hemodialysis patients.This randomized controlled trial will be conducted in the Department of Nephrology at Chittagong Medical College Hospital among 98 adult hemodialysis patients with SHPT. Participants will be randomly assigned to receive either intravenous doxercalciferol or oral calcitriol. The study aims to compare their effectiveness in lowering PTH levels and evaluate safety outcomes.
Continuous variables will be expressed as mean ± standard deviation, while categorical variables will be presented as frequencies and percentages. Independent t-tests, chi-square tests, or Fisher's exact tests will be used for statistical comparisons. Data analysis will be performed using SPSS version 27.0, with a p-value <0.05 considered statistically significant.

Start Date05 Jul 2026

Sponsor / Collaborator

Chittagong Medical College

CTR20230975

/ CompletedNot Applicable

度骨化醇注射液治疗慢性肾脏病血液透析合并继发性甲状旁腺功能亢进症患者的有效性和安全性的开放、单臂、多中心临床研究

[Translation] An open, single-arm, multicenter clinical study on the efficacy and safety of doxorubicin injection in the treatment of chronic kidney disease patients with hemodialysis and secondary hyperparathyroidism

评价度骨化醇注射液治疗慢性肾脏病血液透析合并继发性甲状旁腺功能亢进症的患者的有效性和安全性。

[Translation]

To evaluate the efficacy and safety of doxorubicin injection in the treatment of chronic kidney disease patients undergoing hemodialysis and secondary hyperparathyroidism.

Start Date05 May 2023

Sponsor / Collaborator

Nanjing Heron Pharmaceutical Science & Technology Co., Ltd.

[+1]

ChiCTR2300073196

/ Not yet recruitingNot ApplicableIIT

An Open-Label, Randomized, Single-arm, Multicenter Study to Assess the Safety and Efficacy of Doxercalciferol Injection in Patients With Chronic Kidney Disease With Secondary Hyperparathyroidism on Dialysis

Start Date01 Mar 2023

Sponsor / Collaborator

Jiangsu Provincial People's Hospital

[+1]

100 Clinical Results associated with Doxercalciferol

100 Translational Medicine associated with Doxercalciferol

100 Patents (Medical) associated with Doxercalciferol

216

Literatures (Medical) associated with Doxercalciferol

01 Dec 2025·CLINICAL PHARMACOLOGY & THERAPEUTICS

Repurposing Acebutolol for Osteoporosis Treatment: Insights From Multi‐Omics and Multi‐Modal Data Analysis

Article

Author: Qing Tian ; Zhe Luo ; Hong‐Mei Xiao ; Dragana Lovre ; Hui Shen ; Kuan‐Jui Su ; Jonathan Greenbaum ; Dan‐Yang Liu ; Yue Zhang ; Yun Deng ; Vivian Fonseca ; Li Wu ; Xiang‐He Meng ; Shuang Liang ; Chuan Qiu ; Fernando L. Sanchez ; Yang Li ; Jia‐Chen Liu ; Qiao‐Rong Yi ; Lan‐Juan Zhao ; Li‐Jun Tan ; Hong‐Wen Deng

Osteoporosis is a common metabolic bone disease with aging, characterized by low bone mineral density (BMD) and higher fragility fracture risk. Although current pharmacological interventions provide therapeutic benefits, long‐term use is limited by side effects and comorbidities. In this study, we employed driver signaling network identification (DSNI) and drug functional networks (DFN) to identify repurposable drugs from the Library of Integrated Network‐Based Cellular Signatures. We constructed osteoporosis driver signaling networks (ODSN) using multi‐omics data and developed DFN based on drug similarity. By integrating ODSN and DFN with drug‐induced transcriptional responses, we screened 10,158 compounds and identified several drugs with strong targeting effects on ODSN. Mendelian randomization assessed potential causal links between cis‐eQTLs of drug targets and BMD using genome‐wide association study data. Our findings indicate four drugs, including Ruxolitinib, Alfacalcidol, and Doxercalciferol, may exert anti‐osteoporosis effects. Notably, Acebutolol, a β‐blocker for hypertension, has not previously been implicated in osteoporosis therapy. For validation, zebrafish osteoporosis models were established using Dexamethasone‐induced bone loss, followed by treatment with Acebutolol hydrochloride and Alfacalcidol. Both compounds demonstrated significant protective effects against osteoporosis‐related bone deterioration. Furthermore, a population‐based data set, utilizing propensity score matching and analyzed via a generalized linear model, revealed that individuals taking β‐blocker drugs exhibited significantly higher BMD than users of other cardiovascular medications. In summary, this study integrates multi‐omics approaches, experimental validation, and real‐world population data to propose acebutolol as a novel candidate for osteoporosis treatment. These findings warrant further mechanistic studies and clinical trials to evaluate its efficacy in osteoporosis management.

01 Sep 2025·CELL PROLIFERATION

Utilising Human Myometrial and Uterine Fibroid Stem Cell‐Derived Three Dimentional Organoids as a Robust Model System for Understanding the Pathophysiology of Uterine Fibroids

Article

Author: Omran, Mervat M. ; Bariani, Maria Victoria ; Thompson, Winston E. ; Bai, Tao ; Vafaei, Somayeh ; Ali, Farzana Liakath ; Ali, Mohamed ; Alkhrait, Samar ; Yang, Qiwei ; Al‐Hendy, Ayman

ABSTRACT:

Uterine fibroids (UFs) are the most common benign gynecologic tumours affecting women of reproductive age. This study aims to deepen the understanding of UFs complex aetiology through harnessing the power of 3D organoid models derived from human myometrial stem cells to emulate the in vivo behaviour of these tumours. Isolated SCs were cultured over 7 days under a defined culture system. Immunohistochemistry, Immunofluorescence, organoid stiffness, RNA Sequencing was conducted, and differential gene expression was assessed using RT‐PCR. The derived organoids exhibited diverse populations of cells, including stem cells, smooth muscle, and fibroblasts. Excessive ECM deposition was shown via Collagen and Fibronectin expression. We confirmed that our organoids expressed oestrogen receptor in a pattern similar to that in their corresponding tissue, as well as responded to steroid hormone. Interestingly, we revealed significant racial disparities in ECM accumulation within organoids derived from different racial groups. This augmented ECM deposition is theorised to enhance tissue stiffness, as assessed using Young's modulus. Additionally, our research demonstrated significant decreases in fibrotic markers upon treatment with Vitamin D3 and Doxercalciferol. Furthermore, the pro‐fibroid effects of environmental phthalates further elucidate the potential factors contributing to UF pathology. The 3D organoid model can serve as a robust platform to study the underlying molecular mechanisms of UFs, besides offering invaluable insights for potential therapeutic interventions.

01 Sep 2025·Comparative Biochemistry and Physiology D-Genomics & Proteomics

Analysis of intestinal metabolites in Litopenaeus vannamei infected with white faeces syndrome based on untargeted metabolomics

Article

Author: Jiang, Yongjie ; Zhang, Wei ; Zhang, Haitao ; Pang, Aobo ; Zhang, Xin ; Wang, Tingting ; Tan, Beiping ; Xv, Kangze ; Luthfiah, Adinda

White faeces syndrome (WFS) has always been one of the main intestinal diseases of Litopenaeus vannamei, which has caused huge economic losses to shrimp farming. WFS infection is known to result from a combination of pathogens, but the changes in intestinal metabolites following WFS infection are unknown. In this study, morphological sections of the hepatopancreas and intestine of WFS infected shrimp were analyzed, and lesions were found in the hepatopancreas and intestine of infected shrimp. The hepatopancreatic bodies of shrimp infected with WFS were arranged and scattered, and the stellate cavity was deformed. The connective tissue within the intestinal fold's atrophies to the point where the muscular layer almost disappeared. The enzyme linked kit results showed that lysozyme activity in hepatopancreas was significantly increased, total antioxidant capacity was significantly decreased, intestinal total antioxidant capacity and superoxide dismutase activities were significantly decreased in WFS infected shrimp, indicating that the antioxidant capacity of hepatopancreas and intestine were impaired. The intestinal metabolites of WFS shrimp and healthy shrimp were analyzed by non-targeted metabolic technology, and 10 differential metabolites and 7 differential metabolic pathways were screened. Among them, arginine, a significant differential metabolite, may positively activate the mTOR pathway, leading to the high expression of mTOR pathway, which is closely related to intestinal health. This indicates that when L. vannamei. is infected with WFS, the arginine content in the intestine is up-regulated, which positively activates the mTOR signaling pathway leading to pathway disorder, thereby destroying the intestinal health of L. vannamei. Through this study, we can not only understand the intestinal metabolic characteristics of WFS, but also provide a theoretical reference for the prevention and treatment of WFS in L. vannamei.

News (Medical) associated with Doxercalciferol

14 May 2026

·www.biospace.com

FDA Approves New Dosing Option for CRYSVITA® (burosumab-twza) in Adults With XLH

Expanded dosing options support individualized treatment for adults with XLH PRINCETON, N.J., May 14, 2026 (GLOBE NEWSWIRE) -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co. Ltd., today announced the U.S. Food and Drug Administration (FDA) has approved a dosing update to the CRYSVITA ® (burosumab-twza) Prescribing Information for adults living with X-linked hypophosphatemia (XLH), a rare, progressive, genetic disease that impacts the bones and muscles in both children and adults. Healthcare providers now have the option to increase the dose and frequency of CRYSVITA for patients whose serum phosphorus is below the normal range after the initial treatment period. These patients may be transitioned to a dosage of 0.5 mg/kg, not to exceed 90 mg, once every 2 weeks. If further dosage increase is needed after 4 weeks of treatment with 0.5 mg/kg every 2 weeks, the dosage can be increased to 1 mg/kg, not to exceed 90 mg, administered every 2 weeks. “Kyowa Kirin is committed to delivering solutions that make a meaningful difference for people living with XLH. As the only FDA-approved treatment for XLH, CRYSVITA plays a critical role in care, and it’s essential that clinicians have the flexibility to support optimal patient outcomes,” said Richard Wilson, Senior Vice President, Rare Disease Franchise Head. “While most adults with XLH maintain normal serum phosphorus levels with CRYSVITA administered every four weeks, this update enables a more personalized approach for those who need additional support to reach and maintain serum phosphorus levels within normal range.” To review the new dosing guidance, please see the full Prescribing Information for CRYSVITA, Section 2.3 (Recommended Dosage for Adult Patients with X-linked Hypophosphatemia, 18 years of age and older). About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in children and adults (osteomalacia). About CRYSVITA ® (burosumab-twza) Injection CRYSVITA is a recombinant, fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore renal phosphorus reabsorption and increase the serum concentration of active vitamin D. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient’s serum phosphorus levels. Hypercalcemia Increases in serum calcium have been reported in patients treated with CRYSVITA. Patients with risk factors such as pre-existing hyperparathyroidism, prolonged immobilization, dehydration, hypervitaminosis D, or renal impairment, are at higher risk of hypercalcemia. Monitor these patients for serum calcium and parathyroid hormone levels before and during CRYSVITA treatment for moderate to severe hypercalcemia. In patients with moderate to severe hypercalcemia, CRYSVITA should not be administered until hypercalcemia is adequately managed. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin’s values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at . CONTACT: Contact: Susan Thiele Head of Product and Therapeutic Communications, North America susan.thiele.38@kyowakirin.com

19 Sep 2024

·www.prnewswire.com

Kyowa Kirin to Present New Research Spotlighting Global Efforts to Advance Science and Patient Care in X-linked Hypophosphatemia at ASBMR Annual Meeting

One oral presentation and nine posters will shed light on real-world experiences of people living with XLH and the impact of burosumab treatment TOKYO, Sept. 19, 2024 /PRNewswire/ -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151) will share new research that enhances understanding about the use of burosumab in real-world clinical practice as well as the burden of those living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease. The findings will be presented at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting, which takes place September 27-30 in Toronto. The oral presentation of "Patient reported outcomes (PRO) from a real-world study of burosumab treatment in adults with X-linked hypophosphatemia in the UK" will be made by Judith Bubbear, MD, Royal National Orthopaedic Hospital (UK) on Saturday, September 28, 11:30 am EDT (Presentation #1022). "As we learn more about XLH and how it impacts people's lives, real-world evidence can provide much-needed insight into potential strategies for managing this debilitating disease," said Angela Williams, PhD, Vice President and Global Head of Health Economics and Outcomes Research at Kyowa Kirin. "The breadth of our ASBMR data reflects our continued focus on advancing the science and understanding of XLH and improving the lives of people living with XLH." ASBMR XLH poster presentations: Real-world impact of burosumab treatment in people living with XLH: "Effectiveness of burosumab versus conventional therapy in adults with X-linked hypophosphatemia in a real-world setting from the XLH Disease Monitoring Program" (Poster#SAT-430) Lead author: Pablo Florenzano, MD, Pontificia Universidad Católica de Chile Poster Session I: Saturday, September 28, 2:15 – 3:45 pm EDT "Biochemical measurements according to age group in burosumab-treated patients with X-linked hypophosphatemia (XLH) in a real-world setting: an analysis of the XLH Disease Monitoring Program" (Poster #Fri-424, #Sun-424) Lead author: Leanne M. Ward, MD, Children's Hospital of Eastern Ontario Welcome Reception/Plenary Poster Session: Friday, Sept. 27, 5:30 – 7:30 pm EDT Poster Session II; Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Symptoms experienced by adolescents living with X-linked hypophosphatemia at the end of skeletal growth (EOSG) treated with burosumab—a mixed methods analysis" (Poster #SAT-445) Lead author: Vrinda Saraff, MD, Birmingham Women's and Children's Hospital (UK) Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in adult Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SUN-119) Lead author: Wei Liu, MD, Peking Union Medical College Hospital Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in pediatric Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SAT-121) Lead author: Xiaoping Luo, MD, Tongji Medical College, Huazhong University of Science and Technology Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT XLH burden on multiple aspects of patients' lives: "Association between pain medication use and patient-reported outcomes in adults with X-linked hypophosphatemia: an exploratory analysis of a phase 3 study" (Poster #Sat-025) Lead author: Angela Williams, PhD, Kyowa Kirin International Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Individuals with hereditary hypophosphatemia are not prone to early death despite significant earlier development of co-morbidities: a retrospective Danish register study" (Poster #Sun-024) Lead author: Signe Sparre Beck-Nielsen, MD, PhD, Aarhus University Hospital (Denmark) Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "X-linked Hypophosphatemia Community Impact Survey: psychosocial health, symptoms, and self-care" (Poster #SAT-LB 543) Lead author: Jill Simmons, MD, Vanderbilt University Medical Center Late Breaking Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT Bridging evidence gaps to improve clinical decision-making in XLH: "Advancing Patient Evidence in XLH (APEX): rational and design of real-world XLH global data unification program" (Poster #SAT-427) Lead author: Maria Luisa Brandi, MD, PhD, FIRMO Foundation, Florence, Italy and the University Vita-Salute San Raffaele, Milan, Italy Poster Session I: Saturday, Sep 28, 2:15-3:45 pm EDT About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia). In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Postmarketing experience reported in CRYSVITA-treated pediatric XLH patients: blood phosphorus increased. Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. Learn more about Kyowa Kirin at: kyowakirin.com. COR-US-CRY-0049 September 2024 SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical Result

25 Oct 2023

·www.globenewswire.com

OPKO Health to Present New Clinical Data on Rayaldee (ER Calcifediol) at Kidney Week 2023

MIAMI, Oct. 25, 2023 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) will present late-breaking clinical data on RAYALDEE® extended-release calcifediol (ERC) at the American Society of Nephrology (ASN) Kidney Week in Philadelphia on Thursday, November 2, 2023. These data will be presented in a poster, “Control of Secondary Hyperparathyroidism with Extended-release Calcifediol is Associated with Slower CKD Progression” (#TH-PO1152) at 10:00 a.m. Eastern time in Exhibit Halls B-D by authors Charles W. Bishop Ph.D., Stephen A. Strugnell Ph.D. and Akhtar Ashfaq, M.D., FACP, FASN. OPKO Health will present two other posters at ASN Kidney Week summarizing additional new clinical data on RAYALDEE. One poster, “Extended-release Calcifediol Overcomes Impact of Low eGFR on Vitamin D Metabolism” (#FR-PO319), will be presented in the “Bone and Mineral Metabolism: Basic” session at 10:00 a.m. Eastern time on Friday, November 3, 2023, in Exhibit Halls B-D. The data demonstrate that RAYALDEE effectively and reliably raises serum levels of 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D in non-dialysis patients with secondary hyperparathyroidism (SHPT), making it an attractive alternative to vitamin D hormone therapies (i.e., calcitriol, paricalcitol and doxercalciferol). The other poster, “Extended-Release Calcifediol: A Data Journey from Phase 3 Studies to Real-World Evidence Highlights the Importance of Early Treatment of Secondary Hyperparathyroidism” (#FR-PO972), will be presented in the “CKD Interventions: Trials and Quality Improvement” session at 10:00 a.m. Eastern time on Friday, November 3, 2023 in Exhibit Halls B-D. The data demonstrate that effective control of SHPT has been achieved with RAYALDEE treatment in both randomized clinical trials and in a real-world clinical experience trial. Data from these trials support early initiation of SHPT treatment with RAYALDEE in order to delay disease progression. About RAYALDEE® RAYALDEE is an extended-release (ER) oral formulation of calcifediol, a prohormone of calcitriol, the active form of vitamin D3. The product is the first and only medicine approved by the U.S. Food and Drug Administration for raising serum total 25D and lowering blood levels of intact parathyroid hormone (iPTH). RAYALDEE is approved to treat SHPT in adults with stage 3 or 4 CKD and vitamin D insufficiency in the U.S. and in 11 European countries. Slowing CKD progression with RAYALDEE treatment is not currently an approved indication. About OPKO Health, Inc. OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit www.opko.com. Cautionary Statement Regarding Forward-Looking Statements This press release contains "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," “could,” "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding the market for RAYALDEE, and our strategies or prospects and expectations about RAYALDEE, the therapeutic benefits, safety profile or effectiveness of RAYALDEE or whether early initiation of SHPT treatment with RAYALDEE would delay disease progression. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward- looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and in our other filings with the Securities and Exchange Commission, as well as the risks that the accuracy and effectiveness of the data may not be reproducible or indicative of future results and that currently available over-the-counter and prescription products, as well as products under development by others, may prove to be as or more effective than our products for the indications being studied. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward- looking statements. We intend that all forward-looking statements be subject to the safe- harbor provisions of the PSLRA. Contacts: LHA Investor RelationsYvonne Briggs, 310-691-7100ybriggs@lhai.com or Bruce Voss, 310-691-7100bvoss@lhai.com

Drug ApprovalPhase 3

100 Deals associated with Doxercalciferol

External Link

KEGG	Wiki	ATC	Drug Bank
D01009	Doxercalciferol	H05BX03	DB06410

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperparathyroidism	Canada	Genzyme Corp.	26 Sep 2001
Hyperparathyroidism, Secondary	United States	Genzyme Corp.	09 Jun 1999

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Chronic Kidney Diseases	Phase 3	Germany	Sanofi BV	22 Oct 2007
Chronic large plaque psoriasis	Phase 2	United States	Genzyme Corp.	01 Apr 2008
Advanced Prostate Carcinoma	Phase 2	United States	Sanofi	01 Apr 2002

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02859896 (CTgov)	Phase 3	Hyperparathyroidism, Secondary \| Chronic Kidney Diseases	21	Hectorol (Doxercalciferol (Hectorol®))	rduomkrwih = zhapyciyqk pwhlebtnyq (qhfrstpjmg, agmewphwrr - hikmewhwgb) View more	-	02 Jul 2026
				Rocaltrol (Calcitriol (Rocaltrol®))	rduomkrwih = svuhymhmto pwhlebtnyq (qhfrstpjmg, mqagxwsnjz - pfwyrfkaom) View more
NCT00285467 (CTgov)	Not Applicable	Hyperparathyroidism, Secondary \| Renal Osteodystrophy \| Chronic Kidney Diseases	55	Vitamin D+Doxercalciferol (Doxercalciferol, Active Vitamin D)	benoaxtgwp(xppkclvzcb) = jtlivciqkn mxoefoatbt (rfzjxbcqdk, 34) View more	-	02 May 2016
				Cholecalciferol (Cholecalciferol, Inactive Vitamin D)	benoaxtgwp(xppkclvzcb) = iissuhnkvk mxoefoatbt (rfzjxbcqdk, 31) View more
NCT00601107 (CTgov)	Phase 2	Plaque psoriasis \| Chronic large plaque psoriasis	111	Placebo (Placebo)	xejzqwfgof = zfrcyfshne echbdjmwjs (qmtjosqmiy, zukqhpuehn - ebdciadthg) View more	-	05 May 2014
				Doxercalciferol (Doxercalciferol 2.5 mcg/Day)	xejzqwfgof = hpgvyxaaow echbdjmwjs (qmtjosqmiy, pcxzsrjjka - adyozkngyz) View more
NCT00528788 (CTgov)	Phase 4	Kidney Failure, Chronic \| Hyperparathyroidism, Secondary	24	doxercalciferol	mlzuoumiow(opxsrakife) = pdxpsbityj tlxaslblpq (tyyfgtxola, nwyickkiuv - kjnqhgrmgg) View more	-	14 Apr 2014
NCT00257920 (CTgov)	Phase 4	Chronic kidney disease, Stage V \| Hyperparathyroidism, Secondary	41	Zemplar (Zemplar)	bfrffwkkbo(dxufalxouf) = vfuxphcdbj gpuiontkdm (cbpzrqptut, 0.007) View more	-	20 May 2009
				Hectorol (Hectorol)	bfrffwkkbo(dxufalxouf) = duogdpvdep gpuiontkdm (cbpzrqptut, 0.007) View more
ASCO2007	Phase 2	Castration-Resistant Prostatic Cancer	-	Docetaxel	vfnnywqevj(udsmllqhtm) = xowqvfndnn cikkokwnch (upxugywvzk ) View more	-	20 Jun 2007
				Docetaxel + Doxercalciferol	vfnnywqevj(udsmllqhtm) = nrcksnfbkb cikkokwnch (upxugywvzk ) View more

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