Read the Official PANTHERx Press Release.

For a fifth time, PANTHERx Rare has been recognized as the winner of the annual, Specialty Pharmacy Patient Choice Award™. Since 2016, the Managed Markets Insights & Technology Zitter Health Insight’s (MMIT ZHI) Patient Choice Award has honored the winning specialty pharmacy’s exemplary 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) – an index of -100 to 100 that measures patients’ willingness to recommend their pharmacy to others – from all four quarterly surveys conducted during the 2021 calendar year.

The MMIT ZHI quarterly surveys use a robust, statistically validated methodology and assess numerous aspects of patient satisfaction. Each year, responses are analyzed from more than 10,000 specialty pharmacy patients across all specialty pharmacy sectors. 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.

“Since our founding, PANTHERx has held close the belief that if we make the best decisions for our patients, we will have always made the best decisions for our company. For a fifth time, the MMIT ZHI Patient Choice Award has validated this philosophy that those of us at PANTHERx always knew to be true,” said PANTHERx President, Rob Snyder. “We staff, train, innovate, invest, and perform our daily functions with our patients front of mind. Our associates have not only embraced, but elevated our culture, adding their own expertise, empathy, tenacity, and care. I would like to thank our associates for their tremendous work on behalf of the people living with rare diseases with whom we serve and interact every day. I would also like to thank our patients for expressing their utmost gratification with our team. It is your trust and satisfaction for which we so greatly strive and are so proud to have achieved.”

“Words fall short in expressing my pride for the PANTHERx team. It is because of the daily commitment of every associate, whether directly or indirectly interacting with our patients, that we are being recognized on the national stage and receiving the Patient Choice Award for the fifth time,” said Richard Faris, Senior Vice President of Pharmacy at PANTHERx.

About PANTHERx Rare Pharmacy

PANTHERx Rare is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Although the overall incidence of rare diseases is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an FDA-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 now a five-time winner of the prestigious MMIT Patient Choice Award, including the 2022 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

Read the Official PANTHERx Press Release.

PANTHERx Rare announces that it has been selected by Aadi Bioscience as the exclusive U.S. pharmacy distribution partner for new drug FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound). FYARRO, an albumin-bound mTOR inhibitor administered as an injectable suspension, is the first FDA-approved therapy for the treatment of advanced malignant perivascular epithelioid cell neoplasm (PEComa), an ultra-rare sarcoma.

Advanced PEComa is a rare, aggressive subset of soft tissue sarcomas for which there are no other approved therapeutic options. PEComas are composed of histologically and immunohistochemically distinctive epithelioid cells and most commonly arise from visceral, retroperitoneal, and abdominopelvic sites.

“It is truly special when we are able to provide a new FDA-approved treatment option to a group of people who previously had none,” said Rob Snyder, President of PANTHERx. “PANTHERx is proud to partner with Aadi Bioscience to distribute FYARRO, provide tailored patient support services, and foster hope in the PEComa community.”

PANTHERx supports rare disease patients across the country, addressing the unique needs of every patient and assisting with their personal journeys. Communications and support services are tailored to the desires of each patient and the requirements of their life-changing medications. PANTHERx, with its culture of sincere care and dedication to innovation, continues to define rare pharmacy.

For more information about the symptoms, diagnosis, and treatment of advanced malignant PEComa please click here. For more information about FYARRO, including full prescribing information, please click here.

About PANTHERx Rare

PANTHERx Rare is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Although the overall incidence of rare diseases is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an FDA-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 four-time winner of the prestigious MMIT Patient Choice Award, including the 2021 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

Read the Official PANTHERx Press Release.

PANTHERx Rare announces that it has been selected by Xeris Pharmaceuticals, Inc., a wholly owned subsidiary of Xeris Biopharma Holdings, Inc., as the exclusive U.S. pharmacy distribution partner for a new FDA-approved medication, Recorlev^® (levoketoconazole). Recorlev is a cortisol synthesis inhibitor indicated for the treatment of endogenous hypercortisolemia in adult patients with Cushing’s syndrome for whom surgery is not an option or has not been curative. Cushing’s syndrome, a disease associated with increased production of cortisol, occurs most commonly in adults between ages 30-50 and affects women two times more often than men.

Endogenous Cushing’s syndrome is a rare and serious endocrine disease, often the result of a benign pituitary tumor stimulating the body to overproduce cortisol for long periods of time, leading to unwanted physical and emotional symptoms. Additionally, the internal manifestations of the disease are potentially life-threatening and include metabolic changes such as high blood sugar, or diabetes, high blood pressure, high cholesterol, fragility of various tissues including blood vessels, skin, muscle and bone, and psychological disturbances such as depression, anxiety and insomnia. Recorlev will be commercially available in the first quarter.

“PANTHERx is proud to partner with Xeris to address the needs of adults with endogenous Cushing’s syndrome,” said Rob Snyder, President of PANTHERx. “As the sole pharmacy for Recorlev, PANTHERx recognizes the importance of outstanding execution in order to deliver innovative medication, customized support services, and hope to this vulnerable patient population.”

PANTHERx is driven by our mission to transform the lives of people across the country who are living with rare diseases. Our communications and support services are tailored to the desires of each patient and the requirements of their life-changing medications. The culture of sincere care fostered at PANTHERx propels the company forward, encouraging the development of new systems, services, and programs for the benefit of each and every PANTHERx patient.

For more information about the symptoms, diagnosis, and treatment of endogenous Cushing’s syndrome, please click here.

Indication & Important Safety Information for Recorlev^® (levoketoconazole)

BOXED WARNING: HEPATOTOXICITY AND QT PROLONGATION

HEPATOTOXICITY

Cases of hepatotoxicity with fatal outcome or requiring liver transplantation have been reported with oral ketoconazole. Some patients had no obvious risk factors for liver disease. Recorlev is associated with serious hepatotoxicity. Evaluate liver enzymes prior to and during treatment

QT PROLONGATION

Recorlev is associated with dose-related QT interval prolongation. QT interval prolongation may result in life-threatening ventricular dysrhythmias such as torsades de pointes.  Perform ECG and correct hypokalemia and hypomagnesemia prior to and during treatment

INDICATION

Recorlev is a cortisol synthesis inhibitor indicated for the treatment of endogenous hypercortisolemia in adult patients with Cushing’s syndrome for whom surgery is not an option or has not been curative.

Limitations of Use

Recorlev is not approved for the treatment of fungal infections.

CONTRAINDICATIONS

• Cirrhosis, acute liver disease or poorly controlled chronic liver disease, baseline AST or ALT > 3 times the upper limit of normal, recurrent symptomatic cholelithiasis, a prior history of drug induced liver injury due to ketoconazole or any azole antifungal therapy that required discontinuation of treatment, or extensive metastatic liver disease.
• Taking drugs that cause QT prolongation associated with ventricular arrhythmias, including torsades de pointes.
• Prolonged QTcF interval > 470 msec at baseline, history of torsades de pointes, ventricular tachycardia, ventricular fibrillation, or prolonged QT syndrome.
• Known hypersensitivity to levoketoconazole, ketoconazole or any excipient in Recorlev.
• Taking certain drugs that are sensitive substrates of CYP3A4 or CYP3A4 and P-gp.

WARNINGS AND PRECAUTIONS

Hepatotoxicity

Serious hepatotoxicity has been reported in patients receiving Recorlev, irrespective of the dosages used or the treatment duration. Drug-induced liver injury (peak ALT or AST greater than 3 times upper limit of normal) occurred in patients using Recorlev.  Avoid concomitant use of Recorlev with hepatotoxic drugs. Advise patient to avoid excessive alcohol consumption while on treatment with Recorlev. Routinely monitor liver enzymes and bilirubin during treatment.

QT Prolongation

Use Recorlev with caution in patients with other risk factors for QT prolongation, such as congestive heart failure, bradyarrhythmias, and uncorrected electrolyte abnormalities, with more frequent ECG monitoring considered. Routinely monitor ECG and blood potassium and magnesium levels during treatment.

Hypocortisolism

Recorlev lowers cortisol levels and may lead to hypocortisolism with a potential for life-threatening adrenal insufficiency.  Lowering of cortisol levels can cause nausea, vomiting, fatigue, abdominal pain, loss of appetite, and dizziness. Significant lowering of serum cortisol levels may result in adrenal insufficiency that can be manifested by hypotension, abnormal electrolyte levels, and hypoglycemia. Routinely monitor 24-hour urine free cortisol, morning serum or plasma cortisol, and patient’s signs and symptoms for hypocortisolism during treatment.

Hypersensitivity Reactions

Hypersensitivity to Recorlev has been reported. Anaphylaxis and other hypersensitivity reactions including urticaria have been reported with oral ketoconazole.

Risks Related to Decreased Testosterone

Recorlev may lower serum testosterone in men and women. Potential clinical manifestations of decreased testosterone concentrations in men may include gynecomastia, impotence and oligospermia. Potential clinical manifestations of decreased testosterone concentrations in women include decreased libido and mood changes.

ADVERSE REACTIONS

Most common adverse reactions (incidence > 20%) are nausea/vomiting, hypokalemia, hemorrhage/contusion, systemic hypertension, headache, hepatic injury, abnormal uterine bleeding, erythema, fatigue, abdominal pain/dyspepsia, arthritis, upper respiratory infection, myalgia, arrhythmia, back pain, insomnia/sleep disturbances, and peripheral edema.

DRUG INTERACTIONS

• Consult approved product labeling for drugs that are substrates of CYP3A4, P-gp, OCT2, and MATE prior to initiating Recorlev.
• Sensitive CYP3A4 or CYP3A4 and P-gp Substrates: Concomitant use of Recorlev with these substrates is contraindicated or not recommended.
• Atorvastatin: Use lowest atorvastatin dose possible and monitor for adverse reactions for dosages exceeding 20 mg daily.
• Metformin: Monitor glycemia, kidney function, and vitamin B12 and adjust metformin dosage as needed.
• Strong CYP3A4 Inhibitors or Inducers: Avoid use of these drugs 2 weeks before and during Recorlev treatment.
• Gastric Acid Modulators: See Full Prescribing Information for recommendations regarding concomitant use with Recorlev.

USE IN SPECIFIC POPULATIONS

Lactation: Advise not to breastfeed during treatment and for one day after final dose

To report SUSPECTED ADVERSE REACTIONS, contact Xeris Pharmaceuticals, Inc. at 1-877-937-4737 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information including Boxed Warning.

About PANTHERx Rare

PANTHERx Rare, part of Envolve Pharmacy Solutions, is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients living with rare and devastating conditions. Although the overall incidence of rare diseases 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 four-time winner of the prestigious MMIT Patient Choice Award, including the 2021 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

About Xeris Biopharma

Xeris (Nasdaq: XERS) is a biopharmaceutical company developing and commercializing unique therapies for patient populations in endocrinology, neurology, and gastroenterology. Xeris has two commercially available products; Gvoke^®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia, and Keveyis^®, the first and only FDA-approved therapy for primary periodic paralysis. In addition to Recorlev^® for the treatment of endogenous Cushing’s syndrome, Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol^™ and XeriJect^™, supporting long-term product development and commercial success.

Xeris Biopharma Holdings is headquartered in Chicago, IL. For more information, visit www.xerispharma.com or follow us on Twitter, LinkedIn, or Instagram.

Read the Official PANTHERx Press Release.

PANTHERx Rare has again been recognized as the winner of the annual, national Specialty Pharmacy Patient Choice Award™.

“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,” said PANTHERx President, Rob Snyder. “PANTHERx has achieved an enormous feat, earning the award for a fourth time. We are proud of the level of excellence that our associates have come to expect of themselves and the superior level of service that we have become known for in the industry. To have our patients express their utmost satisfaction with our team is the best affirmation that we can receive.”

PANTHERx focuses on listening to the needs and desires of every patient, to provide the right care at the right time. This philosophy, coupled with the spirit of innovation that exists at PANTHERx, has allowed the organization to provide award-winning service year after year.

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 2020 calendar year.

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 pharmacy sectors each 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

PANTHERx Rare Pharmacy, part of Envolve Pharmacy Solutions, is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients living with rare and devastating conditions. Although the overall incidence of rare diseases 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 four-time winner of the prestigious MMIT Patient Choice Award, including the 2021 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

Read the Official PANTHERx Press Release.

PANTHERx Rare Pharmacy announces that it has been selected by ChemoCentryx as a limited distribution partner for new drug TAVNEOS (avacopan). TAVNEOS, an orally administered selective complement 5a receptor inhibitor, received FDA approval as an adjunctive treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis (also known as ANCA-associated vasculitis) in combination with standard therapy.

ANCA-associated vasculitis is a rare, severe, and often life-threatening autoimmune disease caused by over activation of neutrophils, causing inflammation and destruction of small blood vessels. Initial symptoms may be vague, including prodromal flu-like symptoms, weight loss, lung and/or joint symptoms. ANCA-associated vasculitis affects many organ systems, most commonly the kidneys. People living with this chronic disease often experience cycles of treatment-induced remission followed by relapse, resulting in organ damage and failure, and in severe cases, death.

“PANTHERx is proud to announce our partnership with ChemoCentryx for the distribution of TAVNEOS, a first-in-class orally administered novel medication,” said Rob Snyder, President of PANTHERx Rare. “Together we can provide life changing medication and white glove patient support services to people living with ANCA-associated vasculitis.”

As the nation’s leading rare pharmacy, PANTHERx supports tens of thousands of patients across the country with 25+ uniquely customized RxARECARE^® programs. These highly specialized teams utilize their clinical expertise with our proprietary SWFT^® technology system to address the specific needs of every patient we serve. The culture of sincere care fostered at PANTHERx propels the company’s mission forward, encouraging the development of new systems, services, and programs for the benefit of each and every PANTHERx patient.

For more information about the symptoms, diagnosis, and treatment of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis please click here.

About PANTHERx^® Rare Pharmacy

PANTHERx Rare Pharmacy, part of Envolve Pharmacy Solutions, is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients living with rare and devastating conditions. Although the overall incidence of rare diseases 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.

Read the Official PANTHERx Press Release.

PANTHERx Rare announced today that it was selected by Mitsubishi Tanabe Pharma America, Inc. (MTPA) as the exclusive U.S. pharmacy distribution partner for EXSERVAN™ (riluzole). The drug, an oral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS).

“PANTHERx is excited to be supporting ALS patients who have been prescribed EXSERVAN through medication dispensing and patient education as part of our proprietary RxARECARE^® model,” said Rob Snyder, President of PANTHERx.

PANTHERx utilizes technology and human capital to create RxARECARE programs tailored to the unique needs of the medications we dispense and the preferences of each patient we support.

About PANTHERx Rare

PANTHERx, part of Envolve Pharmacy Solutions, is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx 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.

About EXSERVAN (riluzole) oral film

EXSERVAN, an oral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) in November 2019. EXSERVAN was developed by Aquestive Therapeutics using its PharmFilm^® innovative drug delivery technology. The oral film is placed on top of the patient’s tongue and dissolves. Under the terms of a licensing and supply deal agreement, MTPA is commercializing EXSERVAN in the U.S. and Aquestive is serving as the exclusive sole manufacturer and supplier for the product. For more information, visit www.EXSERVAN.com.

INDICATION

EXSERVAN™ (riluzole) is indicated for the treatment of amyotrophic lateral sclerosis (ALS).

IMPORTANT SAFETY INFORMATION

Do not use if you are allergic to riluzole or to any of its ingredients.

Before using EXSERVAN, tell your doctor about all the medicines you take and all your health conditions, including if you:

• Have liver problems.
• Are taking certain drugs known as strong or moderate CYP1A2 inhibitors such as ciprofloxacin, enoxacin, fluvoxamine, methoxsalen, mexiletine, oral contraceptives, thiabendazole, vemurafenib, or zileuton.
• Are pregnant or intend to become pregnant during EXSERVAN therapy, or if you are breastfeeding or intend to breastfeed during EXSERVAN therapy.

EXSERVAN can cause serious side effects, including:

Liver Problems: Cases of liver injury, some fatal, have occurred in patients taking riluzole. Consult your doctor promptly if you experience unexplained nausea, vomiting, stomach (abdominal) pain, fatigue, weight loss (anorexia), jaundice (yellowing of the skin or whites of the eyes), and/or dark urine.

Low White Blood Cell Counts (Neutropenia): Tell your doctor if you develop a fever while taking EXSERVAN.

Serious Lung Problems (Interstitial Lung Disease): Tell your doctor if you have problems with your lungs or breathing such as dry cough and difficult or labored breathing. Discontinue EXSERVAN immediately if you develop interstitial lung disease.

The most common side effects include numbness in the mouth or tongue, muscle weakness, nausea, lung problems, high blood pressure, and stomach (abdominal) pain.

These are not all the possible side effects of EXSERVAN. Consult your doctor for medical advice about side effects.

To report side effects, contact Mitsubishi Tanabe Pharma America, Inc. at 1-888-292-0058 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Use EXSERVAN as prescribed. The recommended dosage for EXSERVAN is 50 mg taken orally twice daily at least 1 hour before or 2 hours after a meal. Place EXSERVAN oral film strip on the top of the tongue where it will adhere and dissolve. Swallow in a normal manner. Do not cut or split the film or take liquids with EXSERVAN. Do not chew, spit, or talk while EXSERVAN is dissolving. Read the Instructions for Use for EXSERVAN.

Please click here to see the full Prescribing Information for EXSERVAN.

Read the Official PANTHERx Press Release.

PANTHERx^® Rare Pharmacy announced today that it was selected by Rhythm Pharmaceuticals as the exclusive U.S. pharmacy distribution partner for IMCIVREE^™ (setmelanotide). The drug was approved by the U.S. Food and Drug Administration (FDA) in November 2020 for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing.

Obesity due to POMC, PCSK1 or LEPR deficiency are ultra-rare diseases caused by variants in POMC, PCSK1 or LEPR genes that impair the melanocortin-4 receptor (MC4R) pathway, which is responsible for regulating hunger, energy expenditure and consequently body weight. People living with obesity due to POMC, PCSK1 or LEPR deficiency struggle with extreme, insatiable hunger beginning at a young age, resulting in early-onset, severe obesity. As an MC4R agonist, IMCIVREE is designed to restore impaired MC4R pathway activity arising due to genetic deficits.

“As the exclusive U.S. distribution partner for IMCIVREE, PANTHERx is pleased to provide innovative, life-changing medicines and award-winning white-glove services to people living with obesity due to POMC, PCSK1 or LEPR deficiency,” said Rob Snyder, President of PANTHERx. “PANTHERx’s partnership with Rhythm advances our mutual dedication to improving the lives of members.”

PANTHERx supports tens of thousands of patients across the country, addressing the unique needs of every patient and assisting with their personal journey. Our proprietary RxARECARE^® programs and SWFT^® technology systems have allowed PANTHERx to create fully customizable programs for the 25+ rare and orphan medications dispensed. Communications and support services are tailored to the desires of each patient and the requirements of their life-changing medications. PANTHERx, with its evolving suit of personalized services and culture of sincere care, continues to define rare pharmacy.

For more information about the symptoms, diagnosis, and treatment of obesity due to POMC, PCSK1, or LEPR deficiency please click here.

About PANTHERx^® Rare Pharmacy

PANTHERx Rare Pharmacy, part of Envolve Pharmacy Solutions, is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx Rare 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.

IMCIVREE Indication and Usage

IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1, or LEPR deficiency confirmed by genetic testing.

IMCIVREE is not indicated for the treatment of patients with obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign, or for other types of obesity associated with other genetic syndromes and general (polygenic) obesity.

Important Safety Information

WARNINGS AND PRECAUTIONS

• Spontaneous penile erections in males, sexual adverse reactions in females, and depression and suicidal ideation have occurred with IMCIVREE.
• IMCIVREE may cause generalized increased skin pigmentation and darkening of

pre-existing nevi.

Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in neonates and low birth weight infants: IMCIVREE is not approved for use in neonates or infants.

Most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

Please see full Prescribing Information available at www.IMCIVREE.com/hcp for additional Important Safety Information

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.