Lumasiran

Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1

Retrieved on: 
Tuesday, March 1, 2022
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Risk, Therapy, Type, ESC, Therapeutic index, European Medicines Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pain, Kidney, European Commission, Injection site reaction, Medical Affairs Bureau, Medication, Royal commission, Safety, Itch, Child, Alnylam Pharmaceuticals, Review, Erythema, Swelling, New Drug Application, Food, Adult, PH1, PDUFA, Shanda, European Directive on Traditional Herbal Medicinal Products, FDA, RNA interference, Nasdaq, EMA, Committee for Medicinal Products for Human Use, EVP, Patient, Enhance, GO, Prescription Drug User Fee Act, Milk, Committee, CHMP, Plasma, EGFR, Pregnancy, Profile, Pharmaceutical industry, Medical device, Medicine, Birth control, HAO1, 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

This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.

Key Points: 
  • This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.
  • 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%).
  • 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 Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease

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Monday, December 20, 2021
Health, Genetics, Clinical Trials, General Health, Pharmaceutical, Biotechnology, the Phase 2 Study in Patients with Recurrent Kidney Stone Disease, Lumasiran, Recurrent Kidney Stone Disease, RNAi, Alnylam Pharmaceuticals, THE PHASE 2 STUDY IN PATIENTS WITH RECURRENT KIDNEY STONE DISEASE, LUMASIRAN, RECURRENT KIDNEY STONE DISEASE, RNAI, ALNYLAM PHARMACEUTICALS

Recurrent kidney stone disease is a prevalent disease, associated with significant clinical burden, including pain, infection, hospitalizations, and an increased risk of developing chronic kidney disease and kidney failure.

Key Points: 
  • Recurrent kidney stone disease is a prevalent disease, associated with significant clinical burden, including pain, infection, hospitalizations, and an increased risk of developing chronic kidney disease and kidney failure.
  • The Phase 2 trial is a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of lumasiran administered subcutaneously in patients with recurrent calcium oxalate kidney stone disease and elevated urinary oxalate levels.
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1) and recurrent kidney stone disease.
  • Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate the metabolite that directly contributes to the pathophysiology of PH1 and recurrent kidney stone disease.

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

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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).

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

Retrieved on: 
Thursday, July 29, 2021
Research, FDA, Clinical trials, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, Medicine, Clinical medicine, Medical specialties, Membrane technology, Renal dialysis, Kidney diseases, Primary hyperoxaluria, Hyperoxaluria, Alnylam Pharmaceuticals, Hemodialysis, Lumasiran, Oxalate, Lumasiran, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals, LUMASIRAN, PRIMARY HYPEROXALURIA TYPE 1 (PH1), RNAI, ALNYLAM PHARMACEUTICALS

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive topline results from the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of all ages with advanced primary hyperoxaluria type 1 (PH1) associated with progressive decline in renal function.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive topline results from the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of all ages with advanced primary hyperoxaluria type 1 (PH1) associated with progressive decline in renal function.
  • Results of the primary analysis at six months demonstrated substantial reduction in plasma oxalate from baseline in patients (N=21) with advanced disease, including those on hemodialysis.
  • Cohort A enrolled six patients with advanced PH1 who do not yet require dialysis, and Cohort B enrolled 15 patients who are hemodialysis-dependent.
  • Lumasiran also demonstrated positive results across key secondary endpoints, including measures of urinary oxalate (for patients in Cohort A) and additional measures of plasma oxalate.

Alnylam to Webcast Presentations at Upcoming May Investor Conferences

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Tuesday, May 4, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Life sciences, Biotechnology, Branches of biology, RNA interference, RNA, Alnylam Pharmaceuticals, Therapeutic gene modulation, Inclisiran, XC2, Lumasiran, Novartis

Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases.

Key Points: 
  • Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.
  • Alnylam\xe2\x80\x99s commercial RNAi therapeutic products are ONPATTRO\xc2\xae (patisiran), GIVLAARI\xc2\xae (givosiran), OXLUMO\xc2\xae (lumasiran), and Leqvio\xc2\xae (inclisiran) being developed and commercialized by Alnylam\xe2\x80\x99s partner Novartis.
  • Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development.

Alnylam Announces Publication of ILLUMINATE-A Phase 3 Study Results for Lumasiran in The New England Journal of Medicine

Retrieved on: 
Wednesday, March 31, 2021
Health, Genetics, Other Health, Clinical trials, Research, Science, Pharmaceutical, Health sciences, Health, Breakthrough therapy, Lumasiran, Health care quality, Clinical research, Pharmacovigilance, OXLUMO™ (lumasiran), ILLUMINATE-A Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals

Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events (AEs).

Key Points: 
  • Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events (AEs).
  • The most common AEs that occurred more frequently with lumasiran than placebo were injection site reactions (38 versus 0 percent).
  • We believe the publication of the ILLUMINATE-A Phase 3 study results in the NEJM is a testament to lumasiran as an oxalate-lowering therapy which is expected to confer significant clinical benefit to children and adults living with this disease.
  • In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A.

Aerie Pharmaceuticals Receives European Commission Approval for Roclanda® (netarsudil and latanoprost ophthalmic solution) 0.02%/0.005%

Retrieved on: 
Monday, January 11, 2021
Biotechnology, FDA, Health, Pharmaceutical, Optical, Health sciences, Health care, Drugs, Drug Safety, European Medicines Agency, European Union, Pharmaceuticals policy, Marketing Authorisation Application, Marketing authorisation, Committee for Medicinal Products for Human Use, Lumasiran, Potassium citrate/potassium hydrogen carbonate

The marketing authorisation application (MAA) for Roclanda was accepted for review by the European Medicines Agency (EMA) in January 2020.

Key Points: 
  • The marketing authorisation application (MAA) for Roclanda was accepted for review by the European Medicines Agency (EMA) in January 2020.
  • Aerie received a positive scientific opinion recommending approval of the Roclanda MAA from the EMAs Committee for Medicinal Products for Human Use (CHMP) in November 2020.
  • The marketing authorisation is valid in all 27 countries of the European Union, plus Iceland, Norway and Liechtenstein.
  • Any forward-looking statements that we make in this press release speak only as of the date of this press release.

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.

Orsini Specialty Pharmacy Expands Partnership With Alnylam® Pharmaceuticals As A Limited Distribution Partner For OXLUMO™ (lumasiran)

Retrieved on: 
Thursday, December 3, 2020
Life sciences, Biotechnology, Biology, Alnylam Pharmaceuticals, Therapeutic gene modulation, Specialty pharmacy, Lumasiran, Rare disease, Pharmacy

ELK GROVE VILLAGE, Ill., Dec. 3, 2020 /PRNewswire/ --Orsini Specialty Pharmacy, a leader in rare diseases and gene therapies, announced today that it has been selected by Alnylam Pharmaceuticals as a limited distribution partner for OXLUMO (lumasiran).

Key Points: 
  • ELK GROVE VILLAGE, Ill., Dec. 3, 2020 /PRNewswire/ --Orsini Specialty Pharmacy, a leader in rare diseases and gene therapies, announced today that it has been selected by Alnylam Pharmaceuticals as a limited distribution partner for OXLUMO (lumasiran).
  • It is estimated that PH1 affects one to three individuals per million in the United States and Europe.
  • "We are pleased to expand our partnership with Alnylam Pharmaceuticals and add OXLUMO to the therapies we provide for the treatment of ultra-rare genetic conditions," said Mike Fieri, Orsini Chief Executive Officer.
  • Providing patients with comprehensive and compassionate care since 1987, Orsini is a leading, independent specialty pharmacy focused on rare and ultra-rare diseases and gene therapies.

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).