Trending...
- Star-powered Kappa Takeover Weekend Returns to the DMV June 18- 21, 2026, Hosted By Comedian Joe Clair W/ Dj Quick Silva (the Party Kingpin)
- Strategic Expansion with 3 New Alliances — Jefferson Beach Yacht Sales, CFR YS & flyExclusive Incentive Partnership: Off The Hook YS: (N Y S E: OTH)
- InspireTech Global and SKADI Cyber Defense Announce Strategic Partnership to Deliver Autonomous Cybersecurity to Canadian Education and Public Sector
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
- Norisia Launches AI Formulated Luxury Multivitamin to Transform Daily Wellness in the UK
- Jacob Emrani's Annual "Supper Bowl" Expected To Donate Thousands Of Meals
- NASA / Glenn Research Center Collaboration to Help Meet Rising Demand for Space Energy Beaming Tech / CIGS PV Modules from Ascent Solar: NAS DAQ: ASTI
- When Interpretation Becomes Conversation: Rethinking Engagement in the Museum Age
- Half of Finnish Online Gambling Expenditure Now Flows to Offshore Instant Casinos as License Applications Open March 1, 2026
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
- RTC Communications Completes Next Level Connect Fiber Expansion Bringing Multi-Gig Broadband to West Boggs Community
- EPP Pricing Platform announces leadership transition to support long-term growth and continuity
- Stolen Hearts: Reclaiming Your Child From Parental Alienation (narcissistic abuse)
- Roshni Online Services Unveils Plans for Innovative Digital Consultation Platform
- VIP Vacations Named Winner Of The Knot Best Of Weddings 2026
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
- Nevada Man Launches Nationwide Animal Abuse Registry
- Star-powered Kappa Takeover Weekend Returns to the DMV June 18- 21, 2026, Hosted By Comedian Joe Clair W/ Dj Quick Silva (the Party Kingpin)
- Scoop Social Co. Wins The Knot and WeddingWire Awards as Brand Expands Nationwide
- Denise Murphy Lenci Promoted to Century Fasteners Corp. – General Manager, Northeast Sales
- TiNY Puts Real Pets on the Payroll for New PetArmor Campaign
- P‑Wave Classics Launches Definitive New Edition of Hannah Webster Foster's The Coquette
- Strategic Expansion with 3 New Alliances — Jefferson Beach Yacht Sales, CFR YS & flyExclusive Incentive Partnership: Off The Hook YS: (N Y S E: OTH)
- Super League (N A S D A Q: SLE) Advances AI-Driven Playable Media with AdArcade, Solsten, and Meta-Stadiums Partnerships, Plus Roblox Theatre Launch
- purelyIV Expands Concierge Wellness Platform with New IV Therapies, Memberships, and Digital Experience
- CCHR: Europe Rejects Forced Psychiatry—Landmark Vote Declares Coercive Practices Incompatible with Human Rights
- Crossroads4Hope Kicks Off Its 25th Year of Caring with the Launch of Free Breast and Colorectal Cancer Resources for Patients and Families Nationwide
- OpenSSL Corporation Advisory Committees' Elections 2026: Voting Now Open
- Good Vibes Club and Instant IP Forge Strategic Partnership to Secure IP Brand Value in a Booming Digital Economy
- Inkdnylon Simplifies Digitizing and Vector Art Nationwide With Clear Pricing and Guided File Support
- goldsilbermarkt.de Awarded "Business Champion" in Online Retail by DISQ
- Daniel Guest of Imagen Golf Named to GRAA Top 100 Emerging Golf Professionals
- InspireTech Global and SKADI Cyber Defense Announce Strategic Partnership to Deliver Autonomous Cybersecurity to Canadian Education and Public Sector
- Kaltra Expands Microchannel Innovation to Deliver Lower Refrigerant Charge
- Georgia's Lanier Islands Resort Tees Up for a New Era of Golf in Spring 2026
- We Have Lots Of Loads LLC Expands Nationwide