Hydroxyacid oxidase (glycolate oxidase) 1

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

Sunday, June 7, 2020 - 12:15pm

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

Tuesday, May 26, 2020 - 12:00pm

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)

Friday, January 10, 2020 - 9:00pm

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).

Alnylam Reports Positive Topline Results from ILLUMINATE-A Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1

Tuesday, December 17, 2019 - 12:00pm

The study also achieved statistically significant results for all six tested secondary endpoints (p less than or equal to 0.001).

Key Points: 
  • The study also achieved statistically significant results for all six tested secondary endpoints (p less than or equal to 0.001).
  • We are very pleased to report positive topline Phase 3 results for lumasiran, our third wholly owned investigational RNAi therapeutic.
  • Lumasiran results in ILLUMINATE-A mark our third positive Phase 3 study readout in 2019, positioning Alnylam with the potential for four marketed products by the end of 2020, assuming positive regulatory reviews.
  • 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 ILLUMINATE-C Phase 3 Study of Lumasiran for the Treatment of Advanced Primary Hyperoxaluria Type 1 and Presents New Positive Results from Phase 2 Open-Label Extension Study

Saturday, November 9, 2019 - 5:00pm

This study complements our comprehensive clinical development plan for lumasiran, led by our ILLUMINATE-A pivotal study with results expected later this year and our ILLUMINATE-B study in young pediatric patients.

Key Points: 
  • This study complements our comprehensive clinical development plan for lumasiran, led by our ILLUMINATE-A pivotal study with results expected later this year and our ILLUMINATE-B study in young pediatric patients.
  • Patients also experienced an 82 percent mean maximal reduction in urinary oxalate:creatinine ratio (range: 62-94 percent) after lumasiran dosing across all cohorts (N=20).
  • The Phase 2 OLE safety results were based on a median study duration of 10.4 months (range: 7-17 months) since the first dose administered in the OLE study.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).