Tophus

Human medicines European public assessment report (EPAR): Febuxostat Krka, febuxostat, Date of authorisation: 28/03/2019, Revision: 5, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Medical Subject Headings, Marketing, INN, Risk, ATC, Joint, Pain, International, Uric acid, Xanthine oxidase, Blood, Language, Inflammation, Cancer, History, European Medicines Agency, Tablet, Select, Patient, Anatomy, Bone marrow, IIA, Tumor lysis syndrome, Tophus, Medical device

Human medicines European public assessment report (EPAR): Febuxostat Krka, febuxostat, Date of authorisation: 28/03/2019, Revision: 5, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Febuxostat Krka, febuxostat, Date of authorisation: 28/03/2019, Revision: 5, Status: Authorised

Sustained Patient Response to KRYSTEXXA® (pegloticase) Injection with Methotrexate Compared to KRYSTEXXA Alone Shown through Month 12 in MIRROR Randomized Controlled Trial

Retrieved on: 
Tuesday, November 8, 2022
Science, Cardiology, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, SUA, MSU, Tophus, RCT, SUV, Mount Sinai Hospital, Uric acid, Inflammation, DECT, Gout, Methotrexate, American College, Azathioprine, Serial, Randomized controlled trial, MIRROR, Rheumatology, Randomness, Mount Sinai Hospital (Manhattan), Artery, Nasdaq, Week, User experience, Joint, Safety, Time, Pharmaceutical industry, Medical imaging, Patient

This analysis extends the MIRROR randomized controlled trial body of data through Month 12 (Weeks 48-52) of treatment, showing a sustained patient response rate and similar safety profile to the Month 6 findings.

Key Points: 
  • This analysis extends the MIRROR randomized controlled trial body of data through Month 12 (Weeks 48-52) of treatment, showing a sustained patient response rate and similar safety profile to the Month 6 findings.
  • Pharmacokinetic and immunogenicity findings through treatment Month 12 were consistent with Month 6 findings, indicating higher KRYSTEXXA exposure and lower KRYSTEXXA immunogenicity in those who received methotrexate co-therapy.
  • In total, eight participants were included in the imaging analysis (six received KRYSTEXXA with methotrexate and two received KRYSTEXXA with placebo).
  • A Randomized Placebo-Controlled Study of Methotrexate to Increase Response Rates in Patients with Uncontrolled GOut Receiving Pegloticase (MIRROR RCT): Primary Efficacy and Safety Findings.

Data Support the Use of KRYSTEXXA® (pegloticase) Injection with Methotrexate to Treat Uncontrolled Gout in People With Mild-to-Moderate Chronic Kidney Disease

Retrieved on: 
Saturday, November 5, 2022
Health, Other Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, Methotrexate, Randomized controlled trial, Kidney disease, Time, Week, Society, Gout, MIRROR, RCT, Glomerular filtration rate, Chronic kidney disease, EGFR, Tophus, Health, Nasdaq, ASN, American Society of Nephrology, Clinical trial, Disease, SUA, Pharmaceutical industry, Medical device, Patient

Horizon Therapeutics plc (Nasdaq: HZNP) today announced new data that indicate KRYSTEXXA (pegloticase) injection with weekly oral methotrexate effectively treats uncontrolled gout without compromising kidney function.

Key Points: 
  • Horizon Therapeutics plc (Nasdaq: HZNP) today announced new data that indicate KRYSTEXXA (pegloticase) injection with weekly oral methotrexate effectively treats uncontrolled gout without compromising kidney function.
  • These data are encouraging and suggest that KRYSTEXXA with methotrexate effectively addressed the systemic burden of uncontrolled gout and did not lead to renal function decline in patients with mild-to-moderate chronic kidney disease.
  • In both the KRYSTEXXA with methotrexate and KRYSTEXXA with placebo treatment groups, eGFR levels were stable during the four-week methotrexate/placebo run-in period.
  • The renal function improvements seen in both treatment arms were shared by these patients with baseline mild-to-moderate chronic kidney disease.

Alnylam Submits CTA Application for ALN-XDH, an Investigational RNAi Therapeutic for the Treatment of Gout

Retrieved on: 
Monday, December 20, 2021
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Intellectual property, Biology, Gout, Private Securities Litigation Reform Act, Risk, RNA interference, Medication, Disease, Therapy, Physiology, Instagram, LinkedIn, RNA, RNA transfection, FDA, Pain, CTA, Therapeutic index, Inflammatory arthritis, Uric acid, Growth, Nobel Prize in Physiology or Medicine, Medicines and Healthcare products Regulatory Agency, EMA, U.S. Securities and Exchange Commission, United Kingdom, Xanthine dehydrogenase, MHRA, Edema, Safety, Security (finance), Drug development, Flare, Chronic kidney disease, Sanofi, Alnylam Pharmaceuticals, Kidney, Tophus, Cell, Program, Nasdaq, Science, COVID-19, Enhance, SEC, Marketing, Twitter, Nobel Prize, Government, Company, XDH, CNS, Regeneron Pharmaceuticals, Gene silencing, Novartis, Patient, Sirna, Central nervous system, Joint, Pharmaceutical industry, Vaccine, Medicine, ALN-XDH, Gout, RNAi, Alnylam Pharmaceuticals, ALN-XDH, Gout, RNAI, ALNYLAM PHARMACEUTICALS

We believe the existing treatment landscape for gout has substantial limitations attributed to an incomplete response to standard of care, tolerability issues, and poor patient convenience.

Key Points: 
  • We believe the existing treatment landscape for gout has substantial limitations attributed to an incomplete response to standard of care, tolerability issues, and poor patient convenience.
  • ALN-XDH is an investigational, subcutaneously administered RNAi therapeutic targeting xanthine dehydrogenase (XDH) in development for the treatment of gout.
  • Reducing XDH with an RNAi therapeutic is expected to result in potent urate lowering, essential in preventing gout-associated flares and managing the disease.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.

First Patient Enrolls in FORWARD Trial to Evaluate Monthly Dosing of KRYSTEXXA® (pegloticase injection) and Methotrexate to Treat Uncontrolled Gout

Retrieved on: 
Thursday, March 25, 2021
Health, Clinical trials, Research, Pharmaceutical, Science, Biotechnology, Medical specialties, Health, Medicine, Rheumatology, Uric acid, Inborn errors of purine-pyrimidine metabolism, Gross pathology, Tophus, Gout, Clinical trial, Pegloticase, ULT, KRYSTEXXA, Horizon

"When we successfully treat patients with KRYSTEXXA we can reduce the uric acid levels and resolve tophi.

Key Points: 
  • "When we successfully treat patients with KRYSTEXXA we can reduce the uric acid levels and resolve tophi.
  • The Four-Weekly Administration for Urate Reduction (FORWARD) is an adaptive, staggered-start trial designed to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of KRYSTEXXA administered monthly (Q4W).
  • In total, the trial aims to enroll up to 30 adults with uncontrolled gout.
  • The FORWARD trial complements our clinical trial assessing shorter-infusion duration options, which enrolled its first patient last fall and continues our efforts to evolve the patient experience with KRYSTEXXA.

Sobi and Selecta Announce Strategic Licensing Agreement for SEL-212, a Phase 3-ready Novel Treatment for Chronic Refractory Gout

Retrieved on: 
Thursday, June 11, 2020
Medical specialties, Medicine, Rheumatology, Health, Inborn errors of purine-pyrimidine metabolism, Gout, Uric acid, Arthritis, Tophus, Rasburicase

Chronic refractory gout occurs in patients who do not respond to conventional therapies and therefore suffer from high tissue MSU-burden that can cause frequent gout flares and chronic arthritis.

Key Points: 
  • Chronic refractory gout occurs in patients who do not respond to conventional therapies and therefore suffer from high tissue MSU-burden that can cause frequent gout flares and chronic arthritis.
  • The chronic refractory gout market is estimated to be worth at least USD 1 billion in sales in the US alone.
  • SEL-212 has the potential to reduce serum uric acid and MSU deposits in patients with chronic refractory gout.
  • SEL-212 is a combination therapy comprised of pegadricase, a recombinant enzyme that metabolizes uric acid, and ImmTOR, a platform technology.

Sobi and Selecta announce strategic licensing agreement for SEL-212, a phase 3-ready novel treatment for Chronic Refractory Gout

Retrieved on: 
Thursday, June 11, 2020
Medical specialties, Medicine, Rheumatology, Health, Inborn errors of purine-pyrimidine metabolism, Gout, Uric acid, Arthritis, Tophus, Rasburicase

Chronic refractory gout occurs in patients who do not respond to conventional therapies and therefore suffer from high tissue MSU-burden that can cause frequent gout flares and chronic arthritis.

Key Points: 
  • Chronic refractory gout occurs in patients who do not respond to conventional therapies and therefore suffer from high tissue MSU-burden that can cause frequent gout flares and chronic arthritis.
  • The chronic refractory gout market is estimated to be worth at least USD 1 billion in sales in the US alone.
  • SEL-212 has the potential to reduce serum uric acid and MSU deposits in patients with chronic refractory gout.
  • SEL-212 is a combination therapy comprised of pegadricase, a recombinant enzyme that metabolizes uric acid, and ImmTOR, a platform technology.

New Insights on an Ancient Disease and Evidence of Effective Therapy

Retrieved on: 
Thursday, March 21, 2019
Medical specialties, Medicine, Rheumatology, Health, Gout, Tophus, Pegloticase, Arthritis, Joint effusion

The new findings show that the presence of tophi is a surrogate for more severe disease with more tender and swollen joints and a worse health related quality of life.

Key Points: 
  • The new findings show that the presence of tophi is a surrogate for more severe disease with more tender and swollen joints and a worse health related quality of life.
  • Importantly for patients with gout, the new findings also indicated that Pegloticase treatment resulted in significant clinical improvement regardless of the presence of tophi.
  • Gout, an ancient disease and the most common form of inflammatory arthritis in the US, affects more than 8 million people.
  • "The good news for patients with gout is that pegloticase offers substantial clinical benefit in people with chronic or advanced gout regardless of the presence of tophi."