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Read the Official PANTHERx Press Release.

PANTHERx® Rare Pharmacy announces that it has been selected by Alnylam Pharmaceuticals as a very limited distribution partner for the novel drug GIVLAARI™ (givosiran). GIVLAARI is the first and only FDA-approved medicine indicated for the treatment of adults with Acute Hepatic Porphyria (AHP).

GIVLAARI becomes the second RNAi therapeutic from Alnylam approved by the FDA in the last 16 months and the first-ever approved GalNAc-Conjugate RNA therapeutic, a landmark in the advancement of precision genetic medicines. The FDA approval of GIVLAARI was received less than four months after the New Drug Application (NDA) filing acceptance and was based on positive results from the ENVISION Phase 3 study, a randomized, double-blind, placebo-controlled, multinational study of 94 patients with AHP, at 36 study sites in 18 countries – the largest ever interventional study conducted in AHP. In the study, the most common adverse reactions (reported in ≥20% of patients) with GIVLAARI were nausea (27%) and injection site reactions (25%). GIVLAARI is contraindicated in patients with known severe hypersensitivity to givosiran. GIVLAARI has Warnings & Precautions for Anaphylactic Reaction, Hepatic Toxicity, Renal Toxicity, and Injection Site Reactions.

AHP is a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. Long-term complications of AHP can include chronic neuropathic pain, hypertension, chronic kidney disease, and liver disease.

“PANTHERx is proud to deliver innovative medicine to adults with acute hepatic porphyria,” said Dr. Gordon Vanscoy, Chairman and CEO of PANTHERx®. “As a proud partner with Alnylam, we are appreciative of the opportunity to bring new hope to a population of patients who previously had limited therapeutic options. We are thrilled to have a partner whose mission so closely aligns with our own as we collaborate to deliver a new treatment to this patient community.”

About PANTHERx Rare Pharmacy
PANTHERx Rare Pharmacy, the nation’s only health entity with an accredited distinction (ACHC) in rare pharmacy, is headquartered in Pittsburgh, Pennsylvania. PANTHERx Rare’s mission is to transform lives by delivering medical breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Every day we Define RxARE®, by developing focused solutions, bold innovations, and novel clinical services associated with the life-transforming therapies that we deliver.

PANTHERx was awarded the 2018 Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy. Zitter Health Insights awarded PANTHERx the Specialty Pharmacy Patient Choice Award winner for two consecutive years. As a quad-accredited pharmacy, PANTHERx holds accreditations from URAC, VIPPS, ACHC, and CPPA.
For more information, visit rrtest.info.