Nephrocalcinosis

American Regent Introduces Potassium Phosphates, USP; FDA-Approved and "AP" Rated¹

Retrieved on: 
Thursday, October 19, 2023

In addition, inappropriate intravenous administration of undiluted or insufficiently diluted potassium phosphates as a rapid "IV push" has resulted in cardiac arrest, cardiac arrhythmias, hypotension, and death.

Key Points: 
  • In addition, inappropriate intravenous administration of undiluted or insufficiently diluted potassium phosphates as a rapid "IV push" has resulted in cardiac arrest, cardiac arrhythmias, hypotension, and death.
  • Vein Damage and Thrombosis: Potassium Phosphates Injection must be diluted and administered in intravenous fluids or used as an admixture in parenteral nutrition.
  • You are encouraged to report adverse drug events to American Regent, Inc. at 1-800-734-9236 or to the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.
  • You are encouraged to report adverse drug events (ADEs) to American Regent:
    ADEs may also be reported to the FDA:

BridgeBio Pharma Announces Positive Phase 1 Data and Phase 2/3 Trial Design for BBP-711, a Potentially Best-In-Class GO Inhibitor for Primary Hyperoxaluria Type 1 (PH1) and Recurrent Kidney Stone Formers

Retrieved on: 
Monday, June 27, 2022

PALO ALTO, Calif., June 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive Phase 1 data for BBP-711 in healthy volunteers, supporting the development of the investigational therapy for patients with primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers. The data were shared in a feature oral presentation at European Society for Pediatric Nephrology (ESPN) 2022, taking place in Ljubljana, Slovenia.

Key Points: 
  • BBP-711 is an orally-administered small molecule inhibitor of glycolate oxidase (GO) that is being developed to treat conditions of excess oxalate.
  • Overproduction of oxalate in hyperoxaluria, including PH1 and recurrent kidney stone formers with elevated oxalate, can lead to kidney stone formation, nephrocalcinosis and renal impairment.
  • These data are encouraging for patients with PH1 as BBP-711 could become a novel oral therapy to prevent hyperoxaluria and its long-term consequences.
  • Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time.

Dicerna Announces Results for PHYOX™4, Single-Dose Study of Nedosiran in Primary Hyperoxaluria Type 3 (PH3)

Retrieved on: 
Tuesday, October 19, 2021

Nedosiran demonstrated safety and tolerability results in this trial consistent with previously reported studies in the PHYOX clinical development program.

Key Points: 
  • Nedosiran demonstrated safety and tolerability results in this trial consistent with previously reported studies in the PHYOX clinical development program.
  • Dicerna plans to submit an NDA to the FDA for nedosiran for the treatment of PH1 in the fourth quarter of 2021.
  • I would like to extend our sincere thanks to the patients, caregivers, investigators and their staff involved in the PHYOX4 study for their important contributions.
  • Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys.

Dicerna Announces Data to Be Presented at American Society of Nephrology (ASN) Kidney Week 2021

Retrieved on: 
Friday, October 15, 2021

Given the significant unmet medical need, we believe further assessment of nedosirans potential in treating PH2 is warranted.

Key Points: 
  • Given the significant unmet medical need, we believe further assessment of nedosirans potential in treating PH2 is warranted.
  • Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys.
  • There are three known subtypes of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes.
  • Excess production and accumulation of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease requiring intensive dialysis.

Synlogic Presents Data on Hyperoxaluria Program at American Society of Nephrology Kidney Week 2021

Retrieved on: 
Friday, October 15, 2021

CAMBRIDGE, Mass., Oct. 15, 2021 /PRNewswire/ -- Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced two poster presentations at the upcoming American Society for Nephrology (ASN) Kidney Week 2021, to be held virtually November 4 7th, 2021.

Key Points: 
  • CAMBRIDGE, Mass., Oct. 15, 2021 /PRNewswire/ -- Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced two poster presentations at the upcoming American Society for Nephrology (ASN) Kidney Week 2021, to be held virtually November 4 7th, 2021.
  • At this dose, the percent reduction from baseline UOx levels was -28.6% (90% CI: -42.4 to -11.6) compared to placebo in diet-induced hyperoxaluria.
  • Enteric Hyperoxaluria results in dangerously high levels of urinary oxalate, which causes progressive kidney damage, kidney stone formation, and nephrocalcinosis.
  • However, while Synlogic may elect to update these forward-looking statements in the future, Synlogic specifically disclaims any obligation to do so.

Synlogic Presents Data on SYNB8802 for Enteric Hyperoxaluria at American Urological Association (AUA) Annual Meeting

Retrieved on: 
Friday, September 10, 2021

Synlogic will share previously communicated results of the SYNB8802 Phase 1A study including:

Key Points: 
  • Synlogic will share previously communicated results of the SYNB8802 Phase 1A study including:
    SYNB8802 was generally well tolerated in healthy volunteers.
  • Enteric hyperoxaluria results in dangerously high levels of urinary oxalate, which causes progressive kidney damage, kidney stone formation, and nephrocalcinosis.
  • SYNB8802 is currently undergoing Phase 1 studies in healthy volunteers and patients with enteric hyperoxaluria.
  • Synlogic's proprietary pipeline includes Synthetic Biotics for the treatment of metabolic disorders including Phenylketonuria (PKU) and Enteric Hyperoxaluria (HOX).

Synlogic Presents Additional Preclinical Data on Therapeutic Candidates SYNB1934 for Phenylketonuria (PKU) and SYNB8802 for Enteric Hyperoxaluria at Synthetic Biology: Engineering, Evolution & Design (SEED) Conference

Retrieved on: 
Tuesday, June 15, 2021

Data from preclinical in vivoand in vitrostudies demonstrated a greater than 2-fold improvement in the ability of SYNB1934 to metabolize Phe compared to SYNB1618.

Key Points: 
  • Data from preclinical in vivoand in vitrostudies demonstrated a greater than 2-fold improvement in the ability of SYNB1934 to metabolize Phe compared to SYNB1618.
  • "The progression of SYNB1934 highlights Synlogic's ability to use synthetic biology to rapidly design and optimize Synthetic Biotic medicines with the potential for greater patient benefit.
  • Enteric Hyperoxaluria results in dangerously high levels of urinary oxalate, which causes progressive kidney damage, kidney stone formation, and nephrocalcinosis.
  • Synlogic's proprietary pipeline includes Synthetic Biotics for the treatment of metabolic disorders including Phenylketonuria (PKU) and Enteric Hyperoxaluria.

Chinook Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration for CHK-336 for Treatment of Primary Hyperoxaluria

Retrieved on: 
Tuesday, February 2, 2021

Through the FDAs Rare Pediatric Disease Designation and Voucher Programs, the FDA may grant a priority review voucher at the time of product approval for a rare pediatric disease.

Key Points: 
  • Through the FDAs Rare Pediatric Disease Designation and Voucher Programs, the FDA may grant a priority review voucher at the time of product approval for a rare pediatric disease.
  • We are pleased the FDA has granted Chinook rare pediatric disease designation for CHK-336 for the treatment of primary hyperoxaluria, a devastating disease that usually presents in childhood, with life-threatening complications into adulthood, said Alan Glicklich, M.D., chief medical officer at Chinook.
  • Serious manifestations of PH include kidney stones, nephrocalcinosis, growth failure including failure to thrive and reduced linear growth, systemic oxalosis and end-stage kidney disease.
  • In addition, Chinook is advancing CHK-336, an investigational oral small molecule LDHA inhibitor for the treatment of primary hyperoxaluria, as well as research programs for other rare, severe chronic kidney diseases, including polycystic kidney disease.

Synlogic Presents SYNB8802 for Enteric Hyperoxaluria at the American Institute of Chemical Engineers Conference on Microbiome Engineering

Retrieved on: 
Monday, December 7, 2020

SYNB8802 will be assessed for safety and tolerability, and the potential to reduce urinary oxalate.

Key Points: 
  • SYNB8802 will be assessed for safety and tolerability, and the potential to reduce urinary oxalate.
  • Enteric Hyperoxaluria results in dangerously high levels of urinary oxalate, which causes progressive kidney damage, kidney stone formation, and nephrocalcinosis.
  • Synlogic's proprietary pipeline includes Synthetic Biotics for the treatment of metabolic disorders including Phenylketonuria (PKU) and Enteric Hyperoxaluria (HOX).
  • These forward-looking statements should not be relied upon as representing Synlogic's view as of any date subsequent to the date hereof.

Alnylam Presents Positive Results from ILLUMINATE-B Phase 3 Study in Pediatric Patients with Primary Hyperoxaluria Type 1 at the American Society of Nephrology Kidney Week

Retrieved on: 
Thursday, October 22, 2020

Results were presented from ILLUMINATE-B, as well as new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study, at the American Society of Nephrology (ASN) Kidney Week 2020 held as a virtual event on October 22-25.

Key Points: 
  • Results were presented from ILLUMINATE-B, as well as new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study, at the American Society of Nephrology (ASN) Kidney Week 2020 held as a virtual event on October 22-25.
  • Lumasiran also demonstrated positive results across secondary endpoints, including proportion of patients (9/18 or 50 percent) achieving urinary oxalate levels at or below 1.5 times ULNa.
  • Preliminary analysis of exploratory endpoints indicated improvements in nephrocalcinosis in 8 out of 18 patients (44 percent), while estimated glomerular filtration rates (eGFR) remained stable.
  • As expected, given the 6-month duration of the study, there was no change in the rate of renal stone events (RSEs)b .