Trending...
- New research identifies The Discovery Gap: Seven in 10 Americans say travel is no longer just about getting away
- Studica Robotics Receives 2026 Partner Excellence Award from FIRST® Robotics Canada
- Creator Space LA brings together industry leaders for an immersive AI showcase, demonstrations, and film hackathon
PITTSBURGH, Dec. 3, 2020 /PRNewswire/ -- PANTHERx® Rare Pharmacy announces that it has been selected by Alnylam Pharmaceuticals, the leading RNAi therapeutics company, as a very limited distribution pharmacy partner for new drug OXLUMO™ (lumasiran). 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.
Primary hyperoxaluria (PH) is a group of autosomal-recessive diseases resulting from defects in different enzymes involved in glyoxylate metabolism leading to overproduction of oxalate in the liver. There are 3 types of PH: PH1, PH2, and PH3. PH1 is the most common and most severe type of PH caused by a mutation in the AGXT gene that renders the liver enzyme alanine glyoxylate aminotransferase (AGT) dysfunctional.
OXLUMO is a subcutaneously administered RNAi therapeutic that targets the messenger RNA of the hydroxyacid oxidase 1 (HAO1) gene, which encodes glycolate oxidase (GO) – the liver enzyme responsible for oxalate production and upstream of the AGT enzyme deficient in patients with PH1. By reducing the synthesis of GO, OXLUMO decreases the production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1.
In the pivotal trial, ILLUMINATE-A, OXLUMO was compared against placebo in patients 6 years of age and older with PH1 and an eGFR ≥30 mL/min/1.73 m2. 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. Specifically, OXLUMO treatment (N=26) resulted in a mean reduction of 53.5 percent relative to placebo (N=13) (p=1.7 x 10-14), and showed a 65.4 percent mean reduction in urinary oxalate relative to baseline. The most common (≥20%) adverse reaction reported was injection site reaction. Injection site reactions occurred throughout the study period and included erythema, pain, pruritus, and swelling.
"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. "This rare disease medication provides a new option for patients and their families who previously had no approved pharmacologic treatment. PANTHERx is proud to serve as the link between biopharma and patients, delivering medications to the people who need them most."
More on The PennZone
For more information about the symptoms, diagnosis, and treatment of primary hyperoxaluria type 1 (PH1), please click here.
About PANTHERx® Rare Pharmacy
PANTHERx Rare Pharmacy, the largest independent and fastest growing specialty pharmacy in the United States, transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Although the incidence is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a three-time winner of the prestigious MMIT Patient Choice Award, including the 2020 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.
For more information, visit www.pantherxrare.com
About OXLUMOTM (lumasiran)
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. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase potency and durability. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, visit OXLUMO.com.
More on The PennZone
IMPORTANT SAFETY INFORMATION
Adverse Reactions
The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.
Pregnancy and Lactation
No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother's clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.
For additional information about OXLUMO, please see the full Prescribing Information.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. 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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (lumasiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
Contact: Allyson Ayoob, 855-726-8479 x 1142, [email protected]
SOURCE PANTHERx Rare Pharmacy
Related Links
http://www.pantherxrare.com
Primary hyperoxaluria (PH) is a group of autosomal-recessive diseases resulting from defects in different enzymes involved in glyoxylate metabolism leading to overproduction of oxalate in the liver. There are 3 types of PH: PH1, PH2, and PH3. PH1 is the most common and most severe type of PH caused by a mutation in the AGXT gene that renders the liver enzyme alanine glyoxylate aminotransferase (AGT) dysfunctional.
OXLUMO is a subcutaneously administered RNAi therapeutic that targets the messenger RNA of the hydroxyacid oxidase 1 (HAO1) gene, which encodes glycolate oxidase (GO) – the liver enzyme responsible for oxalate production and upstream of the AGT enzyme deficient in patients with PH1. By reducing the synthesis of GO, OXLUMO decreases the production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1.
In the pivotal trial, ILLUMINATE-A, OXLUMO was compared against placebo in patients 6 years of age and older with PH1 and an eGFR ≥30 mL/min/1.73 m2. 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. Specifically, OXLUMO treatment (N=26) resulted in a mean reduction of 53.5 percent relative to placebo (N=13) (p=1.7 x 10-14), and showed a 65.4 percent mean reduction in urinary oxalate relative to baseline. The most common (≥20%) adverse reaction reported was injection site reaction. Injection site reactions occurred throughout the study period and included erythema, pain, pruritus, and swelling.
"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. "This rare disease medication provides a new option for patients and their families who previously had no approved pharmacologic treatment. PANTHERx is proud to serve as the link between biopharma and patients, delivering medications to the people who need them most."
More on The PennZone
- Unlocking Multi-Sector Growth; Graphite Acquisition Powers EV Entry While Streamlined Consumer Snack Business Fuels Growth: (N A S D A Q: SOWG)
- Permian Museum Adds Carbonaceous Chondrite Reference Photos
- L2 Aviation Receives FAA STC and PMA for Klatt Works SAVED™ Smoke Vision System
- Axencis Launches Performance Partnership for Brand Protection
- CCHR Says New OIG Report Raises Concerns about Drugging Elderly with Antipsychotics
For more information about the symptoms, diagnosis, and treatment of primary hyperoxaluria type 1 (PH1), please click here.
About PANTHERx® Rare Pharmacy
PANTHERx Rare Pharmacy, the largest independent and fastest growing specialty pharmacy in the United States, transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Although the incidence is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a three-time winner of the prestigious MMIT Patient Choice Award, including the 2020 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.
For more information, visit www.pantherxrare.com
About OXLUMOTM (lumasiran)
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. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase potency and durability. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, visit OXLUMO.com.
More on The PennZone
- Philadelphia Community Unites for Awareness 2 Wellness Annual Wellness Fair Focused on Healing, Hope
- West Park Arts Fest 2026 Returns to Philadelphia for a Day of Music, Art, and Cultural Celebration
- Project Pretzel Introduces a New System for Running Renovation Projects with Built In Contracts and Real Time Execution
- Freedomtech Solutions creates 'Global Data Centre Network (IDCN)'
- D&D Journey–Children with Disabilities Matter Names JBlair Brown as New PR & Marketing Director
IMPORTANT SAFETY INFORMATION
Adverse Reactions
The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.
Pregnancy and Lactation
No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother's clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.
For additional information about OXLUMO, please see the full Prescribing Information.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. 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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (lumasiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
Contact: Allyson Ayoob, 855-726-8479 x 1142, [email protected]
SOURCE PANTHERx Rare Pharmacy
Related Links
http://www.pantherxrare.com
Filed Under: Business
0 Comments
Latest on The PennZone
- 50 Years of Small Business Wisdom, Supercharged by AI: Shelly Berman Launches The Business Health Check
- Deborah E. Jones Releases Emotional Sovereignty, a Book on Emotional Awareness and Self-Regulation
- Finding the Best Lawyer: What Really Matters When Your Case Is on the Line
- The New World of Freight Brokering with AI
- Captain Notepad Launches Full-Service Direct Mail Platform for Small Businesses Nationwide
- World Cup Buzz Fuels Nayarit's Rise as Mexico's Pacific Standout
- Nayarit strengthens its position in Mexico's Culinary Elite with 8 restaurants in the 2026 Mexico Gastronomic Guide
- Winter Garden Ski Lake Home Sells for $2.05M in Cash Transaction, Highlighting Demand for Strategically Positioned Luxury Properties
- Strobert Tree Services' "50 Shades of Green" Campaign Encourages Arbor Day Action Across Delaware and Pennsylvania
- As Fluoride Debate Grips the Nation, Americans Turn to Whole-House Fluoride Filters for Answers
- FOCUS Hires Carrier-Side Operations Leader to Build the Next Generation of Insurance Service Delivery
- Adams Security Group LLC Launches New Website to Expand Professional Security Services Across Florida
- Presidential Acceleration of Psychedelic Therapies Enters a Defining Moment as Federal Policy, FDA Alignment & Breakthrough Neurotechnology Converge
- Peernovation And Inception Stratos Launch Joint Venture To Build A Global Peer-powered Performance Platform
- GDE Tree Services Expands Operations Across Logan, Ipswich and the Gold Coast
- UK AltNet FullFibre Solves IPv4 Shortage With netElastic BNG And CGNAT Networking Software
- Best Companies Group Opens Registration for Best Places to Work in Manufacturing Program
- Studica Robotics Receives 2026 Partner Excellence Award from FIRST® Robotics Canada
- Seven Games That Make You Think (and Smile) Earn 2026 Mensa Select® Honors
- Mark Schork Honored by Legal Intelligencer as Lawyer on the Fast Track