Hydroxyacid oxidase (glycolate oxidase) 1

Alnylam Submits Regulatory Applications to the U.S. Food and Drug Administration and European Medicines Agency to Support Label Expansion for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1

Retrieved on: 
Tuesday, December 14, 2021
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, PH1, Dialysis, Itch, Food, Shanda, FDA, Injection site reaction, EGFR, CKD, European Medicines Agency, Pain, Child, Swelling, Alnylam Pharmaceuticals, Pregnancy, New Drug Application, Therapeutic index, ASN, Adult, Milk, Nasdaq, GO, EMA, Enhance, Hydroxyacid oxidase (glycolate oxidase) 1, Plasma, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Medication, General manager, Safety, Patient, Therapy, Profile, Society, Kidney, Chronic kidney disease, Erythema, HAO1, ESC, RNA interference, American Society of Nephrology, Skin, Heart, Pharmaceutical industry, Birth control

OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Key Points: 
  • OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
  • The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%).
  • No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production.
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1).

Alnylam Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

Retrieved on: 
Friday, November 5, 2021
Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, GO, Society, Milk, Safety, Alnylam Pharmaceuticals, AES, American Society of Nephrology, Pregnancy, CKD, Cohort study, Plasma, Kidney, Therapeutic index, Baylor College of Medicine, AUC, General manager, Disease, RNA interference, Swelling, Department, ESC, EGFR, EMA, Food, Profile, European Medicines Agency, Associate professor, Chronic kidney disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Medication, Dialysis, 1991 World Sportscar Championship, Pain, Itch, Hyperoxaluria, Therapy, Diagnosis, Severe cognitive impairment, Enhance, ASN, Hydroxyacid oxidase (glycolate oxidase) 1, PH1, Cardiomyopathy, Nasdaq, Patient, HAO1, Risk, AE, Bone, Child, Injection site reaction, Erythema, Adult, Pharmaceutical industry, Birth control, Lumasiran, ILLUMINATE-C Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals, LUMASIRAN, ILLUMINATE-C PHASE 3 STUDY, PRIMARY HYPEROXALURIA TYPE 1 (PH1), RNAI, ALNYLAM PHARMACEUTICALS

PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis.

Key Points: 
  • PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis.
  • ILLUMINATE-C enrolled 21 patients: six patients in Cohort A who did not require dialysis and 15 patients on hemodialysis in Cohort B.
  • OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).

PANTHERx Rare® Pharmacy Chosen as Partner by Alnylam Pharmaceuticals for OXLUMO™ (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1 (PH1) to Lower Urinary Oxalate Levels

Retrieved on: 
Thursday, December 3, 2020
Kidney diseases, Hyperoxaluria, Primary hyperoxaluria, Medical specialties, Clinical medicine, Lumasiran, Medicine, Hydroxyacid oxidase (glycolate oxidase) 1, Oxalate, Urinary system

The drug has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Key Points: 
  • The drug has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
  • OXLUMO met the primary efficacy endpoint of percent reduction from baseline in 24-hour urinary oxalate excretion corrected for BSA, averaged over Months 3 through 6.
  • "We are honored to partner with Alnylam and provide PANTHERx's award-winning RxARECARE services to people living with primary hyperoxaluria type 1," said Dr. Gordon Vanscoy, Chairman & CEO of PANTHERx Rare.
  • OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Alnylam Completes Enrollment in ILLUMINATE-C Phase 3 Study of Lumasiran, an RNAi Therapeutic, for the Treatment of Advanced Primary Hyperoxaluria Type 1

Retrieved on: 
Thursday, December 3, 2020
Research, Genetics, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Branches of biology, Lumasiran, Biotechnology, Biology, Hyperoxaluria, Primary hyperoxaluria, RNA, Hydroxyacid oxidase (glycolate oxidase) 1, RNA interference, Alnylam Pharmaceuticals, Oxalate, ILLUMINATE-C Phase 3 Study, Lumasiran, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals

We are pleased to have completed enrollment in the ILLUMINATE-C Phase 3 study, said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam.

Key Points: 
  • We are pleased to have completed enrollment in the ILLUMINATE-C Phase 3 study, said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam.
  • It was also recently approved by the EMA for the treatment of PH1 in all age groups.
  • The primary endpoint of the study is the percent change in plasma oxalate from baseline to month 6.
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).

Alnylam Announces U.S. Food and Drug Administration (FDA) Approval of OXLUMO™ (lumasiran), the First and Only Treatment Approved for Primary Hyperoxaluria Type 1 to Lower Urinary Oxalate Levels in Pediatric and Adult Patients

Retrieved on: 
Tuesday, November 24, 2020
Health, FDA, Clinical trials, Research, Science, Biotechnology, Branches of biology, Kidney diseases, Hyperoxaluria, Medical specialties, Primary hyperoxaluria, Clinical medicine, Hydroxyacid oxidase (glycolate oxidase) 1, Lumasiran, Oxalate, RNA interference

Primary analysis results from the ILLUMINATE-B study were presented in October 2020 at the virtual American Society of Nephrology Annual Congress.

Key Points: 
  • Primary analysis results from the ILLUMINATE-B study were presented in October 2020 at the virtual American Society of Nephrology Annual Congress.
  • Patients were randomized 2:1 to receive three monthly doses of OXLUMO or placebo at 3 mg/kg followed by a quarterly dosing regimen.
  • The study showed that OXLUMO met its primary endpoint, percent change in 24-hour urinary oxalate (corrected for body surface area and averaged from months three to six).
  • OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Alnylam Receives Positive CHMP Opinion for OXLUMO™ (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1 in All Age Groups

Retrieved on: 
Friday, October 16, 2020
Health, Genetics, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Branches of biology, Kidney diseases, Hyperoxaluria, Primary hyperoxaluria, Biotechnology, Medical specialties, Hydroxyacid oxidase (glycolate oxidase) 1, Alnylam Pharmaceuticals, RNA interference, Oxalate, OXLUMO™ (lumasiran), ILLUMINATE-A Phase 3 Study, ILLUMINATE-B Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam

This positive CHMP opinion recognizes the potential of OXLUMO to address the urgent unmet need that exists for patients of all ages impacted by primary hyperoxaluria type 1.

Key Points: 
  • This positive CHMP opinion recognizes the potential of OXLUMO to address the urgent unmet need that exists for patients of all ages impacted by primary hyperoxaluria type 1.
  • Current treatment approaches aim to delay progression to renal failure but do not prevent oxalate overproduction, said Pushkal Garg, M.D., Chief Medical Officer, Alnylam Pharmaceuticals.
  • The positive opinion is based on efficacy and safety findings of OXLUMO in PH1 patients, including data from both the ILLUMINATE-A and ILLUMINATE-B Phase 3 studies.
  • OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.

Alnylam Reports Positive Topline Results from ILLUMINATE-B Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 in Children Under the Age of Six

Retrieved on: 
Wednesday, September 30, 2020
Research, Children, Genetics, Clinical trials, Other Health, Biotechnology, Pharmaceutical, Consumer, Health, Science, Branches of biology, Hyperoxaluria, RNA, Primary hyperoxaluria, Hydroxyacid oxidase (glycolate oxidase) 1, RNA interference, Acid Pro, PH1b, RNAI, Biology, Molecular biology, ILLUMINATE-A Phase 3 Study, Lumasiran, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals

We are pleased to report these positive topline results that we believe hold promise for many families impacted by PH1.

Key Points: 
  • We are pleased to report these positive topline results that we believe hold promise for many families impacted by PH1.
  • We look forward to reporting complete data from the ILLUMINATE-B study at the ASN virtual congress later this fall.
  • The ILLUMINATE-B results signal hope for the many families with children whose lives are deeply impacted by PH1.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).

Alnylam Presents Positive Phase 3 Results from ILLUMINATE-A Study of Lumasiran, an Investigational RNAi Therapeutic for Treatment of Primary Hyperoxaluria Type 1

Retrieved on: 
Sunday, June 7, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Branches of biology, RNA, Gene expression, Hyperoxaluria, Biology, Primary hyperoxaluria, Molecular biology, RNA interference, Hydroxyacid oxidase (glycolate oxidase) 1, Oxalate, ILLUMINATE-A Phase 3 Study, Lumasiran, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals

We are very pleased to report positive Phase 3 results from the ILLUMINATE-A study of lumasiran.

Key Points: 
  • We are very pleased to report positive Phase 3 results from the ILLUMINATE-A study of lumasiran.
  • The ILLUMINATE-A study represents the sixth positive Phase 3 study for an investigational RNAi therapeutic, and we believe it further highlights the transformational potential of this modality as a whole new class of medicines.
  • Based on results from the ILLUMINATE-A study, lumasiran shows potential to substantially curb oxalate overproduction the cause of progressive kidney failure in PH1.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).

Alnylam Announces U.S. Food and Drug Administration Has Granted Priority Review of the Lumasiran New Drug Application for the Treatment of Primary Hyperoxaluria Type 1

Retrieved on: 
Tuesday, May 26, 2020
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Health, Articles, Pharmaceuticals policy, Life sciences, Hyperoxaluria, European Medicines Agency, Hydroxyacid oxidase (glycolate oxidase) 1, Primary hyperoxaluria, Orphan drug, Breakthrough therapy, Marketing authorization, RNA interference, Lumasiran, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals

Award of Priority Review status adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations.

Key Points: 
  • Award of Priority Review status adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations.
  • In addition, the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency (EMA).
  • Lumasiran has also previously received Orphan Drug Designations for the treatment of PH1 in the U.S. and Europe and has received a Priority Medicines (PRIME) designation from the EMA.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).

Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 (PH1)

Retrieved on: 
Friday, January 10, 2020
Biotechnology, FDA, Other Health, Health, Pharmaceutical, Branches of biology, Biotechnology, Biology, Hyperoxaluria, Alnylam Pharmaceuticals, Food and Drug Administration, Hydroxyacid oxidase (glycolate oxidase) 1, Primary hyperoxaluria, RNA interference

The rolling submission allows completed sections of an NDA to be reviewed by the FDA on an ongoing basis.

Key Points: 
  • The rolling submission allows completed sections of an NDA to be reviewed by the FDA on an ongoing basis.
  • Specifically, Alnylam has submitted the non-clinical components to the FDA and expects to submit the remaining components in early 2020.
  • Alnylam also announced that it has been granted a pediatric rare disease designation from the FDA for lumasiran for the treatment of PH1.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).