Focal segmental glomerulosclerosis

Orphan designation: Repagermanium Treatment of focal segmental glomerulosclerosis, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, EMA, IRIS, Focal segmental glomerulosclerosis, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of focal segmental glomerulosclerosis on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Travere Therapeutics to Present Abstracts on FILSPARI® (sparsentan) in IgA Nephropathy at World Congress of Nephrology and the American Nephrology Nurses Association

Retrieved on: 
Wednesday, April 3, 2024
ART, IgA nephropathy, Congress, Quality of life (healthcare), Patient, SAN, Kidney failure, Immunoglobulin A, RAAS, FSGS, PROTECT, Focal segmental glomerulosclerosis, Abstract, Kidney disease, Therapy, WCN, Proteinuria, ANNA, Communications satellite

SAN DIEGO, April 03, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present nine abstracts in rare kidney disease at the World Congress of Nephrology (WCN) in Buenos Aires, Argentina, on April 13-16, 2024, and the American Nephrology Nurses Association (ANNA) National Symposium in Orlando, Florida, on April 14-17, 2024.

Key Points: 
  • SAN DIEGO, April 03, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present nine abstracts in rare kidney disease at the World Congress of Nephrology (WCN) in Buenos Aires, Argentina, on April 13-16, 2024, and the American Nephrology Nurses Association (ANNA) National Symposium in Orlando, Florida, on April 14-17, 2024.
  • At WCN, the Company will present subgroup analyses of the Phase 3 PROTECT Study of FILSPARI® (sparsentan) in IgA nephropathy (IgAN) showing the treatment effect across participants with different levels of baseline proteinuria.
  • At ANNA, the Company will present additional insights from the HONUS trial, including health-related quality of life (HRQoL) data and the humanistic burden experienced by patients with IgAN and focal segmental glomerulosclerosis (FSGS).
  • “The data we are presenting at WCN and ANNA reinforce the wealth of evidence supporting FILSPARI’s profile to become an effective foundational treatment replacing RAAS inhibition, with the potential to reduce the lifetime risk of kidney failure for patients with IgAN,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics.

ZyVersa Therapeutics Announces IRB Approval of Phase 2a Clinical Trial Protocol to Evaluate Cholesterol Efflux Mediator™ VAR 200 in Patients with Diabetic Kidney Disease

Retrieved on: 
Monday, March 18, 2024
Alport syndrome, Diabetic nephropathy, VAR, Institutional review board, Lipid, Patient, Focal segmental glomerulosclerosis, Kidney disease, Fibrosis, Clinical trial, Kidney, Therapy, Chronic kidney disease, Safety, Dialysis, Cholesterol, IRB, Pharmaceutical industry

Cholesterol Efflux MediatorTM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression.

Key Points: 
  • Cholesterol Efflux MediatorTM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression.
  • WESTON, Fla., March 18, 2024 (GLOBE NEWSWIRE) -- ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for the treatment of renal and inflammatory diseases with high unmet medical needs, announces Institutional Review Board (IRB) approval of the Phase 2a clinical trial protocol to evaluate the efficacy and safety of Cholesterol Efflux Mediator VAR 200 in patients with diabetic kidney disease.
  • It is the first in human trial for VAR 200 intended to substantiate that the promising preclinical results demonstrated in three different animal models of kidney disease (diabetic kidney disease, focal segmental glomerulosclerosis, and Alport syndrome) translate to patients with kidney disease.
  • We are excited about the potential of Cholesterol Efflux MediatorTM VAR 200 to protect against kidney injury and reduce kidney disease progression.”

Ligand Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 27, 2024
Oncology, Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, MRK, Research, CRL, New Drug Application, Investment, Eisai, Growth, Sale, Parent, Novin, Focal segmental glomerulosclerosis, Streptococcus, LGND, FSGS, Breakthrough therapy, BLA, AED, GAAP, Therapy, NASH, Liver, NDA, Depreciation, Breast cancer, Total, Forecasting, Perampanel, Shanda, Telecanthus, ID, Retirement, PDUFA, Vaccine, Symantec Endpoint Protection, Type 2 diabetes, Safety, VRNA, Molluscum contagiosum, F2, Acquisition, Caregiver, Merck, AASLD, FDA, Telephone, Food, Pneumonia, Income tax, Hepatology, COVID-19, Element, Patient, SPA, Pharmaceutical industry

Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022.

Key Points: 
  • Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022.
  • Costs of Captisol sales were $1.6 million for the fourth quarter of 2023, compared with $21.6 million for the same period in 2022, with the decrease due to lower total Captisol sales during the fourth quarter of 2023 and $9.8 million in accelerated depreciation on Captisol manufacturing equipment during the fourth quarter of 2022.
  • Amortization of intangibles was $8.3 million for the fourth quarter of 2023, compared with $8.5 million for the same period in 2022.
  • Travere also announced fourth quarter 2023 results reporting that it received 459 new patient start forms for FILSPARI in the fourth quarter of 2023 and net product sales of $14.9 million for the fourth quarter.

American Kidney Fund Announces April 30 as the First Annual APOL1-Mediated Kidney Disease (AMKD) Awareness Day

Retrieved on: 
Thursday, January 25, 2024
Vertex Pharmaceuticals, Apolipoprotein L1, Dialysis, Central African, Focal segmental glomerulosclerosis, Kidney failure, Health, Foreign policy, Kidney, FSGS, APOL1, Gene, AKF, Risk, Kidney disease, Genetic testing, Mutation, Disease, Congress, L1, Education, American Kidney Fund, Pharmaceutical industry

ROCKVILLE, Md., Jan. 25, 2024 (GLOBE NEWSWIRE) -- The American Kidney Fund (AKF) is proud to announce that April 30, 2024 will mark the nation’s first ever AMKD Awareness Day .

Key Points: 
  • ROCKVILLE, Md., Jan. 25, 2024 (GLOBE NEWSWIRE) -- The American Kidney Fund (AKF) is proud to announce that April 30, 2024 will mark the nation’s first ever AMKD Awareness Day .
  • Building on AKF’s educational campaign for the condition, AMKD Awareness Day aims to spread awareness of APOL1-mediated kidney disease (AMKD) , a spectrum of kidney diseases associated with variants (mutations) in the apolipoprotein L1 (APOL1) gene and linked to an increased risk for rapidly progressing kidney disease in people of Western and Central African descent.
  • People with two mutations are at higher risk of developing genetic forms of kidney disease such as focal segmental glomerulosclerosis (FSGS).
  • Additionally, AKF will provide resources and ways to engage and raise awareness of the important connection between APOL1 gene variants and kidney disease in the months ahead.

ZyVersa Therapeutics CEO, Stephen C. Glover, Issues Letter to Shareholders Providing Outlook for 2024

Retrieved on: 
Wednesday, January 3, 2024
Alport syndrome, Quality of life, Entrepreneurship, Inflammasome, NLRP1, Survival, IND, VAR, Letter, Kidney disease, Inflammation, FSGS, AIM2, Health, Human, Therapy, Clinical trial, Diabetic nephropathy, Focal segmental glomerulosclerosis, Kidney, Proteinuria, NLRP3, DKD, ACE, Optimism, SGLT2, Patient, Capital, Fibrosis, Dialysis, Environment, Pharmaceutical industry

Combined, our two proprietary product platforms target a global total addressable market of around $75 billion.

Key Points: 
  • Combined, our two proprietary product platforms target a global total addressable market of around $75 billion.
  • On the financial front, various market watchers are seeing signals that the biotech and pharma investment environment is set to trend positively in 2024.
  • Capital is again starting to flow to companies that are seeking to innovate through differentiated technologies or novel mechanisms of action.
  • We look forward to embarking on what we believe will be a productive 2024.

XORTX Submits a New Patent for the Treatment of Chronic Kidney Disease

Retrieved on: 
Wednesday, January 3, 2024
DN, Immunoglobulin A, IgA nephropathy, Kidney disease, Diabetic nephropathy, Uric acid, Trial of the century, Oxipurinol, Therapy, Autosome, Pharmacokinetics, Focal segmental glomerulosclerosis, Patent, Chronic kidney disease, ADPKD, Treating, Study, Polycystic kidney disease, XRX, Safety, Lupus nephritis, XOI, CKD, Pharmaceutical industry

CALGARY, Alberta, Jan. 03, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces submission of a new patent for the treatment of chronic kidney disease (“CKD”).

Key Points: 
  • CALGARY, Alberta, Jan. 03, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces submission of a new patent for the treatment of chronic kidney disease (“CKD”).
  • This patent is designed to protect new discoveries and strategies for the treatment of individuals with varied degrees of kidney function in the setting of CKD.
  • This patent application is intended to claim new opportunities to enhance how the xanthine oxidase inhibitor class of drugs may be dosed in the future.
  • Importantly, how to further improve the safe and effective administration of this class of drugs, including oxypurinol.”

ZyVersa Therapeutics Engages CRO, George Clinical, for Phase 2a Clinical Trial for Cholesterol Efflux Mediator VAR 200

Retrieved on: 
Thursday, December 14, 2023
Cholesterol, VAR, Therapy, Clinical trial, Focal segmental glomerulosclerosis, Chronic kidney disease, DKD, Patient, Safety, Diabetic nephropathy, Pharmaceutical industry

WESTON, Fla., Dec. 14, 2023 (GLOBE NEWSWIRE) -- ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA; “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of patients with renal and inflammatory diseases who have unmet medical needs, announces selection of contract research organization (“CRO”) George Clinical to manage its Phase 2a clinical trial with Cholesterol Efflux MediatorTM VAR 200 in patients with diabetic kidney disease (DKD).

Key Points: 
  • WESTON, Fla., Dec. 14, 2023 (GLOBE NEWSWIRE) -- ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA; “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of patients with renal and inflammatory diseases who have unmet medical needs, announces selection of contract research organization (“CRO”) George Clinical to manage its Phase 2a clinical trial with Cholesterol Efflux MediatorTM VAR 200 in patients with diabetic kidney disease (DKD).
  • The clinical trial is expected to begin in the first quarter of 2024.
  • George Clinical, who combines scientific and clinical leadership with expert trial delivery to create distinctive world-class solutions, was the recipient of Citeline’s 2023 Clinical Research Team of the Year.
  • “Initiation of the Phase 2a trial in patients with DKD marks a major milestone in the development of Cholesterol Efflux MediatorTM VAR 200 and for ZyVersa.

American Kidney Fund Hosts Fourth National Summit on Unknown Causes of Kidney Disease

Retrieved on: 
Tuesday, December 5, 2023
Nephrology, Cardiology, Public policy, Genetic counseling, Food, Risk, Division, Research, Kidney disease, Vertex Pharmaceuticals, Health, Education, Legislation, Therapy, Columbia University, Focal segmental glomerulosclerosis, Kidney, COVID-19, Patient, Diagnosis, AKF, American Kidney Fund, Pharmaceutical industry

ROCKVILLE, Md., Dec. 05, 2023 (GLOBE NEWSWIRE) -- Today, the American Kidney Fund (AKF) will convene more than 100 medical leaders, patient advocates and academic and industry leaders for the fourth national summit of its Unknown Causes of Kidney Disease (UCKD) Project, which seeks to improve understanding of how undiagnosed or misdiagnosed causes of kidney disease directly impact patient care and outcomes.

Key Points: 
  • ROCKVILLE, Md., Dec. 05, 2023 (GLOBE NEWSWIRE) -- Today, the American Kidney Fund (AKF) will convene more than 100 medical leaders, patient advocates and academic and industry leaders for the fourth national summit of its Unknown Causes of Kidney Disease (UCKD) Project, which seeks to improve understanding of how undiagnosed or misdiagnosed causes of kidney disease directly impact patient care and outcomes.
  • This will be the first in-person summit after three years of virtual summits due to the pandemic.
  • This week’s summit, which will be held in Arlington, Virginia, will further explore how the kidney community can drive innovation in research, diagnosis and treatment of the underlying causes of kidney disease.
  • “Finding out the root cause of one’s kidney disease can bring crucial clinical insights to providers and have life-saving implications for individuals.

Travere Completes Successful pre-NDA Meeting for FILSPARI in IgAN; Provides Regulatory Updates for both IgAN and FSGS

Retrieved on: 
Monday, December 4, 2023
Workforce, Depreciation, Food, Nephrology, Immunoglobulin A, Shanda, FDA, SAN, Therapy, Focal segmental glomerulosclerosis, New Drug Application, Proteinuria, HCU, EGFR, Pharmaceutical industry

SAN DIEGO, Dec. 04, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the completion of a successful pre-NDA meeting with the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) in IgA nephropathy (IgAN). The Company will submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval. The Company also completed regulatory engagement on focal segmental glomerulosclerosis (FSGS) in which the FDA communicated that the Phase 3 DUPLEX Study results alone are not sufficient to support an sNDA submission for an FSGS indication for sparsentan. As a result, the Company will be conducting additional analyses of FSGS data with plans to re-engage FDA in 2024, and is implementing a strategic reorganization in Q4 2023 to focus near-term resources on the ongoing FILSPARI launch in IgAN and the advancement of pegtibatinase in classical homocystinuria (HCU).

Key Points: 
  • The Company will submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval.
  • “Following a successful pre-NDA meeting, we are pleased to be moving forward with our planned sNDA submission for full approval of FILSPARI in IgA nephropathy.
  • “Unfortunately, there is uncertainty around a regulatory path forward for FSGS.
  • Together with $634.6 million in cash and cash equivalents as of September 30, 2023, the Company expects to have a cash runway into 2028.