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 DOJOLVI^TM (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 QINLOCK^TM (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.”

QINLOCK^TM (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. QINLOCK^TM (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.

In a landmark, non-commercial publication available for download, PANTHERx Rare Pharmacy explores the details of how changes in U.S. Federal policy and advances in scientific research have led to the dawn of medical breakthroughs for rare disorders, and how rare pharmacy has emerged distinctly from specialty pharmacy to address the unique needs of patients, biopharma, providers, and payors. The document highlights the fact that patients with rare and devastating disorders require and deserve a higher acuity of care and service that only an accredited rare pharmacy can deliver.

RATIONALE:
Over 93% of the 7,000 known rare and devastating disorders have no approved therapy, but changes in policy and advances in science have provided tremendous hope. Once explored, this noticeable gap in medicine spurred changes to legislation, encouraging manufacturers to pursue orphan drug development. This shift of focus eventually brought us to the present day, where the phrase “rare pharmacy” is garnering more attention, and the experience of some pharmacies, coupled with new accreditation standards, is creating a distinction between a specialty pharmacy and a rare pharmacy.

SYNOPSIS:
This white paper guides readers through the evolution of the rare pharmacy industry, with an in-depth analysis of the following:

1. Background on Rare Disease Medication Development – To better understand the emergence of the orphan drug market, it is necessary to review legislation and grant programs that catalyzed the development of orphan drugs tangential to traditional specialty drugs. The genesis of, and relationships between, programs such as the Orphan Drug Act, the 2012 FDA Safety and Innovation Act, The Orphan Products Clinical Trials Grants Program, The Natural History Grants Program, and The Pediatric Device Consortia (PDC) Grants Program are explored.

2. Challenges and Impact of Rare Disease – The number and diversity of industry stakeholders with unique perspectives and objectives necessitates examination of challenges faced by drug manufacturers, payors, rare disease patients, providers, and pharmacies. Each of these groups must implement resources, structures, technologies, and unique programs more encompassing than ever created before to assist patients and their families through the complex health journeys surrounding rare disease.

3. Rare Pharmacy Solutions – What makes a rare pharmacy different than a specialty pharmacy? Among a long list of tangible differentiating factors, rare pharmacies invest in the unique patient journey in more deliberate and personalized ways, provide more extensive prior authorization support and payment assistance, and utilize systematic and customizable data reporting capabilities. As the list of necessary criteria has grown, accrediting bodies have developed high standards and strict requirements for specialty pharmacies to distinguish themselves as rare pharmacies.

Learn more by downloading the entire white paper: Rare Pharmacy Emerges from Specialty (Pharmacy).

ABOUT: PANTHERx® Rare Pharmacy

PANTHERx Rare Pharmacy’s mission is to transform lives by delivering medical breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. PANTHERx was awarded 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. PANTHERx is a finalist for the 2019 MMIT Patient Choice Award to be held on May 7, 2020. 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.

Read the Official PANTHERx Press Release.

PANTHERx® Rare Pharmacy announces that it has been selected by Blueprint Medicines Corporation as a very limited distribution partner for breakthrough drug AYVAKIT™ (avapritinib). It has been approved for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. With nationwide distribution of AYVAKIT, PANTHERx establishes a formidable presence in the precision oncology space.

“PANTHERx is thrilled to be chosen by Blueprint Medicines to collectively make historic strides in precision oncology,” said Dr. Gordon Vanscoy, Chairman and CEO of PANTHERx. “As a partner, we are grateful to offer hope to a genomically defined population of patients with rare gastrointestinal stromal tumors. PANTHERx is proud to be part of an elite network advancing precision oncology and bringing new hope to rare cancer patients.”

AYVAKIT is a potent and selective KIT and PDGFRA inhibitor. It is the only FDA-approved type 1 inhibitor for GIST that works by directly binding to the active kinase conformation from which mutant KIT and PDGFRA signal. AYVAKIT has demonstrated inhibition of a broad range of KIT and PDGFRA mutations associated with GIST, including potent clinical activity against activation loop mutations that are associated with resistance to currently approved therapies.

The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation and one for the treatment of advanced systemic mastocytosis (SM), including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

For more information about PANTHERx RxARECARE® services for patients with GIST, contact us at 833-918-2015.

Gastrointestinal Stromal Tumors
Gastrointestinal Stromal Tumors (GISTs) are a rare form of cancer located in the gastrointestinal (GI) tract. More than half of GISTs start in the stomach although many start in the small intestine and other parts of the GI tract. Doctors will look at specific features of a GIST to help predict whether it is likely to grow and spread to other parts of the body. GISTs are evaluated based on the size of the tumor, where it is located in the GI tract, and how fast the tumor cells are dividing (mitotic rate).

The GIST tumors often grow into empty spaces inside the GI tract so they may not cause symptoms until they reach a certain size or are in a certain location. As a result, a small tumor may not cause any symptoms. Common symptoms of GISTs include: include throwing up bright red or dark blood, bleeding into the stool, abdominal pain, a noticeable mass or swelling in the abdomen, nausea or vomiting, loss of appetite and weight loss, and feeling full after eating small amounts of food.

Treatment options depend on a several factors, including the tumor size, the mitotic rate, where the tumor is located in the GI tract, the genetic makeup of the tumor, and if the tumor has spread to other parts of the body. Treatment options include surgery to remove the tumor and targeted therapies with tyrosine kinase inhibitors (TKIs).

For more information about the symptoms, diagnosis, and treatment of Gastrointestinal Stromal Tumors please click here (https://www.pantherxrare.com/rare-disorders/gastrointestinal-stromal-tumors/).

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 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.

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 www.pantherxrare.com.

Read the Official PANTHERx Press Release.

PANTHERx Rare Pharmacy is proud to have again earned the #1 ranking in the quarterly patient satisfaction survey conducted by Zitter Health Insights, an independent national survey entity. For the third quarter of 2019, PANTHERx obtained the highest Net Promoter Score (NPS) among all specialty pharmacies surveyed including retail, hospital/health system, PBM/payer, and independent specialty pharmacies. Net Promoter Score (NPS) is compiled by asking patients, on a scale of 0 to 10, how likely they would be to recommend their specialty pharmacy to a friend or family member. With an NPS of 92 (out of 100) PANTHERx scored 41 points higher than the third-quarter 2019 industry average of 51 points, and 12 points above the next highest-scoring independent specialty pharmacy. PANTHERx is also a two-time winner of the annual Zitter Specialty Pharmacy Patient Choice Award.

“PANTHERx’s achievements on the Zitter Health Insights surveys are indicative of our relentless commitment to patient care. Despite the rapid growth of the organization, every member of our team is completely engaged and focused on the care, health, happiness, and satisfaction of our patients. PANTHERx is proud of every award we receive, but the awards from our patients, whom we serve with a passion, are the most humbling,” said Dr. Gordon J. Vanscoy, Chairman & CEO of PANTHERx.

PANTHERx also received perfect NPS scores from patients afflicted with Autosomal Dominant Polycystic Kidney Disease (ADPKD) and Hypophosphatasia (HPP), amongst others.

The PANTHERx team is proud to have earned such high marks from its patients, validating the team’s authentic dedication to individualized rare patient care:
• PANTHERx ranked best when patients were asked if they would be unhappy if they were forced to switch to a different specialty pharmacy.
• PANTHERx ranked best when patients were asked how satisfied they were with the time to speak with a live person.
• All (100%) patients surveyed indicated that they are satisfied with the existing telephone hold times at PANTHERx.

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 for two consecutive years. As a quadruple-accredited pharmacy, PANTHERx holds accreditations from URAC, VIPPS, ACHC, and CPPA.