Orsini Specialty Pharmacy issued the following announcement on Dec. 3.
Orsini Specialty Pharmacy, a leader in rare diseases and gene therapies, announced that it has been selected by Alnylam® Pharmaceuticals as a limited distribution partner for OXLUMO™ (lumasiran). OXLUMO is the first and only treatment approved for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
PH1 is an ultra-rare genetic disease characterized by the overproduction of oxalate, a waste product that usually is eliminated by the kidneys and excreted in the urine. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and systemic organ dysfunction. 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. "Our dedicated OXLUMO Care Team is committed to providing compassionate support and individualized services to assist people with PH1 and their families throughout their journey and help them navigate through the complexities of our healthcare system."
About Orsini Specialty Pharmacy
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. Orsini's high touch care model is centered around experienced, therapy-specific care teams who provide personalized care to patients based on their specific condition and treatment. The company's comprehensive solutions include medication adherence programs, data analytics, customized manufacturer programs and nationwide nursing coverage for convenient in-home infusion services. Headquartered in Elk Grove Village, IL, Orsini Specialty Pharmacy holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC, NABP, and most recently was awarded ACHC's Distinction in Rare Diseases and Orphan Drugs.
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.
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.
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.
For more information about Orsini's specialty pharmacy services, contact us at 847-734-7373 ext. 545, e-mail us at orsini@orsinihc.com, or visit https://www.orsinispecialtypharmacy.com/.
Original source can be found here.
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