PANTHERx is proud to join the Centene family of companies. Through this transaction, we are reaffirming our relentless commitment to transforming lives and removing barriers for individuals living with complex and rare diseases.
Read the official press release.
Today is a transformative day and the culmination of a great deal of effort and diligence to support the future of PANTHERx. We are very excited to announce that we have taken a significant step towards positioning ourselves for more sustainable growth by announcing a signed agreement under which PANTHERx will become a part of the Centene family of companies.
Read the official press release
Read the Official PANTHERx Press Release.
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.”
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.
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.
Read the Official PANTHERx Press Release.
Ernst & Young LLP (EY US) yesterday announced that Dr. Gordon J. Vanscoy, Chairman & CEO of both PANTHERx Rare and RareMed Solutions, was named an Entrepreneur Of The Year® 2020 National Award winner following selection as an East Central regional award winner in October. This is the first time since 2002 that Pittsburgh has been home to an EY Entrepreneur of the Year National Award winner.
The Entrepreneur Of The Year Awards program is one of the world’s most prestigious competitive forums for entrepreneurs and leaders of high-growth companies. Dr. Gordon Vanscoy was one of 10 national winners announced Thursday, November 19, during a virtual awards celebration. The awards were the culmination of the Strategic Growth Forum®, one of the nation’s most prestigious gatherings of high-growth, market-leading companies. Dr. Vanscoy was selected by an independent panel of judges from a pool of more than 200 regional award winners.
“I am humbled to have been honored as an EY Entrepreneur of the Year National Award Winner,” said Dr. Gordon J. Vanscoy, Chairman & CEO of PANTHERx Rare & RareMed Solutions. “I accept this award on the shoulders of two fantastic teams. I am so proud of the companies that we have created, but most of all, I am proud of the quality manner in which we serve the rare disease community. To be recognized so auspiciously on the national level in the midst of a pandemic is sobering, but I plan to represent my companies and my hometown of Pittsburgh in an honorable manner.”
Since 1986, EY US has honored entrepreneurs whose ingenuity, spirit of innovation and tenacity have driven their companies’ success, transformed their industries and made a positive impact on their communities. Dr. Gordon Vanscoy will go on to become a lifetime member of the esteemed multi-industry community of award winners, with exclusive, ongoing access to the experience, insight and wisdom of fellow alumni and other ecosystem members in over 60 countries — all supported by EY’s vast resources.
Sponsors
Founded and produced by Ernst & Young LLP, the Entrepreneur Of The Year Awards are nationally sponsored by SAP America and the Kauffman Foundation.
About Entrepreneur Of The Year®
Entrepreneur Of The Year® is the world’s most prestigious business awards program for unstoppable entrepreneurs. These visionary leaders deliver innovation, growth and prosperity that transform our world. The program engages entrepreneurs with insights and experiences that foster growth. It connects them with their peers to strengthen entrepreneurship around the world. Entrepreneur Of The Year is the first and only truly global awards program of its kind. It celebrates entrepreneurs through regional and national awards programs in more than 145 cities in over 60 countries. Winners go on to compete for the EY World Entrepreneur Of The Year™ title. ey.com/us/eoy
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Read the Official PANTHERx Press Release.
PANTHERx® Rare Pharmacy announces that it has been selected by Blueprint Medicines as a limited distribution partner for new drug GAVRETO™ (pralsetinib), which was granted -accelerated approval for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. Developed by Blueprint Medicines, GAVRETO is a once-daily oral precision therapy designed to selectively and potently target oncogenic RET alterations.
RET activating fusions and mutations are key disease drivers in many cancer types, including NSCLC. RET fusions are implicated in approximately 1 to 2 percent of patients with NSCLC.
“PANTHERx is honored to be chosen by Blueprint Medicines to deliver this breakthrough oncology therapy to people living with RET fusion-positive metastatic NSCLC,” said Dr. Gordon Vanscoy, Chairman and CEO of PANTHERx. “As a partner, we are proud to do our part in advancing rare oncology by providing award-winning white-glove services to this previously underserved patient population as part of our innovative RxARECARE™ model.”
For more information about the symptoms, diagnosis, and treatment of RET fusion-positive NSCLC, 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.
Trademarks
Blueprint Medicines, GAVRETO, and associated logos are trademarks of Blueprint Medicines Corporation.
Read the Official PANTHERx Press Release.
PANTHERx® Rare Pharmacy announces that it has been selected by Ultragenyx Pharmaceutical as a limited distribution partner for new drug DOJOLVITM (triheptanoin). DOJOLVI™ (a highly purified, pharmaceutical-grade, synthetic seven carbon fatty acid triglyceride) is a specially designed synthetic triglyceride compound, which was developed to treat patients with long-chain fatty acid oxidation disorders (LC-FAOD). DOJOLVI™ provides these patients with a medium-length, odd-chain fatty acid that LC-FAOD patients can metabolize and serve as an alternative source of energy (by increasing intermediate substrates in the Krebs cycle, a key energy-generating process).
“PANTHERx Rare is proud to be chosen by Ultragenyx Pharmaceutical to deliver hope, white glove services, and DOJOLVI™ to people living with LC-FAOD,” said Dr. Gordon Vanscoy, Chairman & CEO of PANTHERx. “Our unique RxARECARE™ Team structure ensures that new patients, and all of our patients, receive the level of superior quality, award-wining care that has become synonymous with PANTHERx. We look forward to making a difference in the lives of people living with LC-FAOD.”
LC-FAODs are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body, and serious liver, muscle, and heart disease, which can lead to hospitalizations or early death. Currently, LC-FAOD patients are treated with the avoidance of fasting, low-fat/high carbohydrate diets, carnitine, and medium-chain triglyceride (MCT) oil, a medical food product. Despite current therapy, many patients have significant metabolic events including hospitalizations and mortality due to LC-FAOD.
Learn more about the symptoms, diagnosis, and treatment of Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD).
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, ACHC, and CPPA.
Read the Official PANTHERx Press Release.
PANTHERx® Rare Pharmacy announces that it has been selected by Viela Bio, Inc. as their sole rare pharmacy partner for new drug Uplizna™ (inebilizumab-cdon). The drug has been approved for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
NMOSD is a rare neuroinflammatory autoimmune disease characterized by unpredictable attacks that often lead to severe, irreparable disability including loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure. Each NMOSD attack can lead to further damage and disability.
Uplizna is a humanized monoclonal antibody targeting CD19+ B cells. Inebilizumab-cdon specifically binds to CD19, a cell surface antigen present on pre-B and mature B cell lymphocytes. B cells are believed to play a central role in the pathogenesis of NMOSD.
“PANTHERx is proud to be an exclusive partner with Viela Bio to bring Uplizna to patients suffering from neuromyelitis optica spectrum disorder,” said Dr. Gordon Vanscoy, Chairman and CEO of PANTHERx. “As the sole pharmacy for Uplizna, PANTHERx recognizes the importance of flawless execution as we deliver this breakthrough medication, customized support services, and hope to this vulnerable patient population. We are honored to make a difference in the lives of people living with NMOSD.”
For more information about the symptoms, diagnosis, and treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD), 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, ACHC, and CPPA.
Read the Official PANTHERx Press Release.
PANTHERx Rare Pharmacy (PANTHERx), was officially recognized as a prestigious US Best Managed Company for overall business performance and sustained growth. The US Best Managed Company designation, sponsored by Deloitte Private and The Wall Street Journal, is the preeminent distinction for privately owned and managed businesses in the United States, based on criteria such as management abilities in the areas of strategy, execution, culture, financials, and overall business practices.
As one of only 27 US Best Managed Company honorees, PANTHERx has proven its dedication to not only fostering a culture of growth and sustainability for its associates, but also to providing the best rare pharmacy care and service to its patients afflicted by rare diseases.
“We are incredibly proud of our team’s recognition and what it means for PANTHERx. It’s a sign of our relentless commitment to doing better and being better. As 1PANTHER, we’ll continue to look for novel ways to improve the employee, patient, and partner experience,” says PANTHERx Chairman & CEO, Dr. Gordon Vanscoy. “Our growth has been fueled by innovation, technology, and exceeding expectations for all our patients and partners. PANTHERx is truly unique in the healthcare marketplace. Becoming one of the elite US Best Managed Companies was fueled by incredibly dedicated associates led by an executive team second to none.”
About the Best Managed Companies Program
The Best Managed Companies program is a mark of excellence for private companies. U.S. designees have annual revenues of at least $250 million. Hundreds of private companies around the world have competed for this designation in a rigorous and independent process that evaluates four key criteria in their management skills and practices—strategy, execution, culture and financials. U.S. program sponsors are Deloitte Private and The Wall Street Journal.
Read the Official PANTHERx Press Release.
PANTHERx® Rare Pharmacy announces that it has been selected by Deciphera Pharmaceuticals as a limited distribution partner for new drug QINLOCKTM (ripretinib). QINLOCK™ is a kinase inhibitor indicated for the treatment of adult patients with advanced gastrointestinal stromal tumors (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. Deciphera is focused on developing and commercializing innovative drugs that address the unmet medical needs of cancer patients by addressing key mechanisms of drug resistance.
“PANTHERx is thrilled to be chosen by Deciphera Pharmaceuticals to deliver hope, this new oncology therapy with a novel mechanism of action, and our award winning white-glove services to patients living with advanced gastrointestinal stromal tumors,” said Dr. Gordon Vanscoy, Chairman & CEO of PANTHERx. “As a partner, PANTHERx is proud to do our part in advancing oncology care and we are excited to offer more treatment options for people living with rare cancers.”
QINLOCKTM (ripretinib) targets the broad spectrum of KIT and PDGFRα mutations known to drive GIST, including KIT mutations known to cause resistance to currently approved agents. Metastatic GIST is a disease characterized by many mutations, with over 90% of GIST patients harboring multiple mutations that drive progression of their disease. The complex heterogeneity of mutations within individual tumors and individual patients is a major cause of resistance to existing therapies.
While approved kinase inhibitors control certain initiating and drug resistance-causing mutations in KIT and PDGFRα, the kinases that drive disease progression in most GIST patients, these approved drugs fail to inhibit all known mutations. QINLOCKTM (ripretinib) was specifically designed to improve the treatment of GIST patients by inhibiting the full spectrum of known mutations in KIT and PDGFRα.
Learn more about the symptoms, diagnosis, and treatment of Gastrointestinal Stromal Tumors.
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, ACHC, and CPPA.
Read the Official PANTHERx Press Release.
PANTHERx Rare Pharmacy (PANTHERx) has again been recognized as the winner of the annual, national Specialty Pharmacy Patient Choice Award™. “We are honored and humbled to be recognized as the top independent specialty pharmacy in overall patient satisfaction for a third time,” says PANTHERx Chairman & CEO, Dr. Gordon Vanscoy. “No other measure and recognition could be more meaningful to us. The results of the Managed Markets Insights & Technology Zitter Health Insights (MMIT ZHI) Patient Satisfaction Surveys validate the success of our mission to redefine the
level of care provided in specialty and rare pharmacy. We are honored by this award because it is derived from the patients we serve, patients that are expressing their utmost satisfaction with our team. Valid, independent patient satisfaction survey benchmarking raises the quality bar for the entire industry and patients win as a result.”
PANTHERx’s win follows on the heels of two recent peer recognitions: PANTHERx was awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). “PANTHERx’s uniqueness stems from our individualized rare disease patient approach guided by a proprietary technology platform. Every PANTHERx patient is met on their journey, at whatever stage they are in, and navigated along a path of better health with a dedicated team of PANTHERx associates by their side,” says Dr. Richard Faris, SVP of Pharmacy at PANTHERx. “We focus on listening to the needs and desires of every patient so that we can provide them with the right care at the right time. This award, where patients are given the platform to openly assess the service they receive, is the best indicator that our RxARECARE teams seamlessly execute the 1PANTHERxTM way of providing care.”
About the Award
The MMIT ZHI Patient Choice Award honors a specialty pharmacy’s commitment to patients. The criteria for the award were formulated by an external awards committee made up of prominent individuals representing pharmaceutical manufacturers, payors, trade organizations, consultants, and equity research firms. Pharmacies were selected as finalists for the Patient Choice Award based on their average Net Promoter Score (NPS) from all four quarterly surveys conducted during the 2019 calendar year. PANTHERx Rare Pharmacy’s average NPS for 2019 topped the charts.
About the Survey
The MMIT ZHI survey uses a robust, statistically validated methodology and assesses numerous aspects of patient satisfaction and analyzes responses from more than 10,000 specialty pharmacy patients across all specialty pharmacies every year. By leveraging an independent entity to aggregate unbiased patient feedback, specialty pharmacies can objectively assess how they are perceived by their patients, as well as benchmark how they compare to competitor performance in the areas of patient perception, access, and adherence. The NPS–an index of -100 to 100 that measures patients’ willingness to recommend their pharmacy to others–is the primary metric of the survey.
About PANTHERx® Rare Pharmacy
The mission of PANTHERx Rare Pharmacy, the largest independent specialty pharmacy in the U.S., 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. As a quad-accredited pharmacy, PANTHERx holds accreditations from URAC, NABP, ACHC, and CPPA. PANTHERx is the first and only pharmacy with an Accredited Distinction in Rare Diseases and Orphan Drugs from ACHC.