Calcitriol

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

Retrieved on: 
Wednesday, October 25, 2023
Real-World Evidence, FASN, Secondary hyperparathyroidism, Importance, ERC, Health, Poster, Society, American Society of Nephrology, ASN, Calcitriol, Serum, Quality, OPKO Health, CKD, FACP, Paricalcitol, Patient, Calcifediol, Pharmaceutical industry, SHPT

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.

Key Points: 
  • 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.
  • OPKO Health will present two other posters at ASN Kidney Week summarizing additional new clinical data on RAYALDEE.
  • 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.

Kyowa Kirin Presents Real-World Findings at ASBMR Annual Meeting Highlighting Progressive Disease Burden of X-linked Hypophosphatemia (XLH)

Retrieved on: 
Monday, October 16, 2023
Komodo, Diagnosis, Enthesopathy, Osteoarthritis, MBA, Health care, Physician, Hypertension, Obesity, Research, Calcitriol, Myalgia, Burosumab, Prevalence, Tendon, X-linked hypophosphatemia, XLH, Society, Kyowa Kirin, TSE, Spinal stenosis, Health, American Society for Bone and Mineral Research, Arthralgia, Fracture, Disease, Medicare, Kidney, Depression, Patient, DSM-IV codes, Kidney disease, Commercial, FH, Muscle weakness, Pharmaceutical industry

Most patients had received some form of conventional therapy for XLH prior to burosumab treatment, with 61% receiving calcitriol and 43% receiving phosphate supplements.

Key Points: 
  • Most patients had received some form of conventional therapy for XLH prior to burosumab treatment, with 61% receiving calcitriol and 43% receiving phosphate supplements.
  • Older patients were under-represented in the study population, limiting the generalizability of the findings to this group.
  • In this retrospective observational cohort study, researchers examined healthcare claims data from the Merative™ MarketScan® Commercial and Medicare Databases.
  • The differences between the study groups were apparent even among young children and persisted throughout a person's life.

Notable Labs Presents Data Demonstrating PPMP’s Potential to Identify Novel Drug Combinations in JMML at the 2023 EHA Hybrid Congress

Retrieved on: 
Friday, June 9, 2023
San Francisco, Tretinoin, University, European Hematology Association, CALC, University of California, San Francisco, Cancer, Juvenile myelomonocytic leukemia, Patient, Drug combination, Cytarabine, EHA, Calcitriol, Poster, Hospital, FLU, Pharmaceutical industry, JMML

FOSTER CITY, Calif., June 09, 2023 (GLOBE NEWSWIRE) -- Notable Labs, Inc. (“Notable”), a clinical stage therapeutic platform company developing predictive precision medicines for cancer patients, today presented data from an ex vivo study demonstrating the potential for its Predictive Precision Medicine Platform (PPMP) to identify more active investigational pre-hematopoietic stem cell therapy (pre-HCST) regimens for the treatment of juvenile myelomonocytic leukemia (JMML) at the European Hematology Association (EHA) Hybrid Congress being held in Frankfurt, Germany on June 8 – 15, 2023.

Key Points: 
  • Of 130 drug combinations, 27 were shown to be more active in killing leukemic cells ex vivo than compounds represented in these combinations on their own.
  • These results highlight PPMP as a potential tool for identifying superior drug combinations for patients with high medical needs, including with JMML.
  • Abstract title: Tretinoin and calcitriol enhance treatment regimens for juvenile myelomonocytic leukemia in ex vivo drug sensitivity assays.
  • Additional meeting information can be found on the 2023 EHA Congress website, https://ehaweb.org/congress/eha2023-hybrid-congress/eha2023/ .

End Drug Shortages Alliance Releases Report for Drugs Impacted by Pharmaceutical Plant Closure

Retrieved on: 
Wednesday, March 15, 2023
Other Manufacturing, Medical Supplies, Hospitals, Transport, Manufacturing, Practice Management, General Health, Pharmaceutical, Health, Logistics, Supply Chain Management, Oklahoma Hospital Association, Risk, Dimercaprol, Hyperparathyroidism, Supraventricular tachycardia, ASHP, Patient, EDSA, Calcitriol, Organic acid, Knowledge, Akorn, Physostigmine, East End Drugs, Bankruptcy, Adenosine, CHA, Anticholinergic, Mayo Clinic, Kidney, Pharmaceutical industry, Risk management, Medical device, Nursing, Vizient, Inc.

The End Drug Shortages Alliance (EDSA) today released a report assessing the impact to the U.S. market caused by the recent closure of a drug manufacturer.

Key Points: 
  • The End Drug Shortages Alliance (EDSA) today released a report assessing the impact to the U.S. market caused by the recent closure of a drug manufacturer.
  • “Transparency regarding drug shortages is important to ensure patient care remains uninterrupted,” said Mittal Sutaria, senior vice president, Vizient pharmacy contract and program services and EDSA board member.
  • Clinicians and providers, to exercise a stewardship mindset and review purchasing and formulary status to determine conservation and mitigation strategies.
  • “Advance knowledge of supply disruptions is part of the transparency that the End Drug Shortages Alliance has advocated for since forming.

Ascendis Pharma Submits Marketing Authorisation Application to the European Medicines Agency for TransCon™ PTH in Adult Patients with Hypoparathyroidism

Retrieved on: 
Monday, November 14, 2022
Amnesia, Priority review, Brain, Senior, Headache, Degenerative disease, European Medicines Agency, Safety, Disease, Eye, Judgement, Blood, Risk, Investment, Medical Affairs Bureau, MAA, PTH, Calcitriol, Kidney, Paresthesia, Science, Marketing, Research, PDUFA, Depression, Regulation of tobacco by the U.S. Food and Drug Administration, COVID-19, Health, EMA, Patient, Weakness, Quality of life, Hypoparathyroidism, Annual report, Hypoesthesia, Bipolar disorder, Food, Exocrine gland, Parathyroid hormone, Southern Transcon, Marketing Authorisation Application, Calcium, Pharmaceutical industry, Fine chemical, Princeton University

COPENHAGEN, Denmark, Nov. 14, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon PTH (palopegteriparatide) in adult patients with hypoparathyroidism.

Key Points: 
  • COPENHAGEN, Denmark, Nov. 14, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon PTH (palopegteriparatide) in adult patients with hypoparathyroidism.
  • TransCon PTH is an investigational prodrug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism.
  • The MAA is based on data from the global Phase 3 PaTHway Trial and the Phase 2 PaTH Forward Trial of TransCon PTH in adult patients with hypoparathyroidism.
  • Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma Group.

FDA Accepts for Priority Review Ascendis Pharma’s NDA for TransCon™ PTH in Adult Patients with Hypoparathyroidism

Retrieved on: 
Monday, October 31, 2022
Safety, Hypoparathyroidism, Eye, Forward-looking statement, U.S. Securities and Exchange Commission, Headache, Risk, Investment, Judgement, Weakness, Hormone replacement therapy, Science, Degenerative disease, Calcitriol, Paresthesia, Amnesia, Patient, EMA, Disease, GLOBE, Bipolar disorder, Technology, Exocrine gland, Quality of life, Review, Parathyroid hormone, European Medicines Agency, Annual report, Brain, Compliance, Research, Kidney, PDUFA, SEC, Hypoesthesia, Depression, FDA, Senior, PTH, Prescription Drug User Fee Act, Calcium, COVID-19, Nasdaq, Medical Affairs Bureau, Southern Transcon, Priority review, Blood, NDA, Adult, Priority, Company, MAA, Advisory Committee, Pharmaceutical industry, Fine chemical, Princeton University

COPENHAGEN, Denmark, Oct. 31, 2022 (GLOBE NEWSWIRE) --  Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has accepted for Priority Review its New Drug Application (NDA) for TransCon PTH (palopegteriparatide) in adult patients with hypoparathyroidism and has set a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2023. TransCon PTH is an investigational prodrug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism. The FDA said that it is not currently planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • We understand the urgency and need these patients face and will continue our work to make TransCon PTH available as quickly as possible.
  • Ascendis plans to submit a Marketing Authorisation Application (MAA) for TransCon PTH in adult patients with hypoparathyroidism to the European Medicines Agency (EMA) during the fourth quarter of this year.
  • During the fourth quarter, the Company also expects to announce topline results for PaTHway Japan, the Phase 3 trial of TransCon PTH in adult Japanese patients with hypoparathyroidism.
  • Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma Group.

Ascendis Pharma Submits TransCon™ PTH New Drug Application to the U.S. FDA for Adult Patients with Hypoparathyroidism

Retrieved on: 
Wednesday, August 31, 2022
COVID-19, Technology, Safety, Research, Brain, Conditional sentence, Temperature, Science, U.S. Securities and Exchange Commission, Depression, Oklahoma Office of the Chief Medical Examiner, GLOBE, Adult, Risk, PTH, Patient, Exocrine gland, Blood, Degenerative disease, Southern Transcon, SEC, Headache, Kidney, Nasdaq, FDA, NDA, Disease, Judgement, MAA, PH, Hypoparathyroidism, Quality of life, Investment, Parathyroid hormone, European Medicines Agency, Calcitriol, Hypoesthesia, Weakness, Bipolar disorder, Hormone replacement therapy, Amnesia, Calcium, Annual report, Eye, Forward-looking statement, Paresthesia, Pharmaceutical industry, Fine chemical, Princeton University

COPENHAGEN, Denmark, Aug. 31, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon PTH, an investigational prodrug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism. TransCon PTH has been granted Orphan designation in the United States and European Union.

Key Points: 
  • COPENHAGEN, Denmark, Aug. 31, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon PTH, an investigational prodrug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism.
  • In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug.
  • A phase 3 trial of TransCon PTH in hypoparathyroidism is ongoing in Greater China through VISEN Pharmaceuticals.
  • Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma Group.

Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist Drug Pipeline Insight Market Report 2022 Featuring Hybrigenics, MC2 Therapeutics, Lipidor/Cerbios-Pharma, OPKO Renal, & Chugai Pharmaceutical - ResearchAndMarkets.com

Retrieved on: 
Wednesday, February 9, 2022
Biotechnology, Pharmaceutical, Health, Calcitriol, Vitamin D receptor, Agonist, VDR, Therapy, Fine chemical, Pharmaceutical industry

The "Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist - Pipeline Insight, 2022" clinical trials has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist - Pipeline Insight, 2022" clinical trials has been added to ResearchAndMarkets.com's offering.
  • "Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist - Pipeline Insight, 2022" report offers comprehensive insights of the pipeline (under development) therapeutics scenario and growth prospects across Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist development.
  • The report assesses the active Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist pipeline products by developmental stage, product type, molecule type, and administration route.
  • Offers detailed therapeutic product profiles of Vitamin D Receptor (VDR or Calcitriol Receptor) Agonist with key coverage of developmental activities including licensing & collaboration deals, patent details, designations, technologies, indications and chemical information
    Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration

RAYALDEE® Launched in Germany by OPKO Health’s Licensee, VFMCRP

Retrieved on: 
Thursday, February 3, 2022
Private Securities Litigation Reform Act, CKD, Fresenius Medical Care, Patient, Safety, Legislation, Cooperation between China and Central and Eastern European Countries, Food, NASDAQ, Health, Conditional sentence, PSLRA, Dialysis, ISIN, OPKO Health, Nephrology, Patent, Adoption, Chronic kidney disease, OPK, Parathyroid hormone, Serum, Adult, Medicine, U.S. Securities and Exchange Commission, Calcitriol, SIX Swiss Exchange, Vifor Pharma, GLOBE, Regulation of tobacco by the U.S. Food and Drug Administration, Security (finance), D3, Calcifediol, Pharmaceutical industry, Dietary supplement, Fine chemical, SHPT, European

VFMCRP has received marketing authorizations for RAYALDEE in 11 European countries and expects to launch in additional markets later this year.

Key Points: 
  • VFMCRP has received marketing authorizations for RAYALDEE in 11 European countries and expects to launch in additional markets later this year.
  • The launch of RAYALDEE in Germany is a significant commercial milestone with more European countries expected to come on board, beginning with Switzerland in early March.
  • Europe represents a large market opportunity for RAYALDEE with an estimated 26 million people suffering from stage 3 or 4 CKD, said Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO.
  • VFMCRP has significant expertise in nephrology and we are confident in their ability to market RAYALDEE successfully in Germany and other licensed territories.

Inozyme Pharma Announces Presentation of Data from ENPP1 Deficiency Development Program at the ASBMR 2021 Annual Meeting

Retrieved on: 
Monday, October 4, 2021
Private Securities Litigation Reform Act, Heart, Cardiovascular disease, Phenotype, ABCC6, AAV, Enzyme replacement therapy, Investment, Pacific Plate, Annual general meeting, Gene, American Society for Bone and Mineral Research, Disease, ENPP1, Bone, Technology, Plasma, Clinical trial, Rickets, Myocardial infarction, Hearing loss, Annual report, Therapy, Hypophosphatemia, U.S. Securities and Exchange Commission, FDA, Company, Lists of diseases, Risk, Artery, Calcification, Society, GLOBE, Pyrophosphate, Diagnosis, Security (finance), Generalized arterial calcification of infancy, Patient, Master of Science, Skeleton, Osteomalacia, Aorta, MBA, Research, Blood, Calcitriol, Yale University, Nasdaq, ERT, Pharmaceutical industry, Medical imaging

BOSTON, Oct. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the presentation of data from the Company’s ENPP1 Deficiency Natural History Study and its gene therapy program for the treatment of ENPP1 Deficiency. The data were presented at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting held October 1-4.

Key Points: 
  • The data were presented at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting held October 1-4.
  • ENPP1 Deficiency is a progressive condition that manifests as a spectrum of disease.
  • These data highlight implications for the appropriate diagnosis and treatment of patients with ENPP1 Deficiency.
  • Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.