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Full Name Abilify MyCite® (aripiprazole tablets with sensor)
Drug Abilify MyCite
Manufacturer Otsuka Pharmaceutical Co., Ltd.
Route of Administration Oral
Site of Care Home
Approved Indication Treatment of adults with schizophrenia, bipolar I disorder, and adjunctive treatment of adults with major depressive disorder (MDD)
Disease Schizophrenia; Bipolar Disorder; Major Depressive Disorder (MDD)
Therapeutic Area Psychiatry, Mental Health & Addiction
Enrollment Form Link Enrollment Form
Phone Number 844-692-4833
Fax Number 847-789-9274
Product Website abilifymycitehcp.com
Full Name Adzynma (ADAMTS13, recombinant-krhn)
Drug Adzynma
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP)
Disease Congenital thrombotic thrombocytopenic purpura (c-TTP)
Therapeutic Area Hematology
Enrollment Form Link Enrollment Form
Phone Number 800-720-0789
Fax Number 877-251-0709
Product Website adzynma.com
Full Name Aldurazyme® (laronidase)
Drug Aldurazyme
Manufacturer Genzyme Corporation
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms
Disease Mucopolysaccharidosis I
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website aldurazyme.com/healthcare
Full Name Amondys 45™ (casimersen)
Drug Amondys 45
Manufacturer Sarepta Therapeutics, Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping
Disease Duchenne Muscular Dystrophy (DMD)
Therapeutic Area Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-356-5034
Fax Number 877-339-4602
Product Website amondys45.com
Full Name Amvuttra® (vutrisiran)
Drug Amvuttra
Manufacturer Alnylam Pharmaceuticals, Inc.
Route of Administration Subcutaneous
Site of Care Home or Healthcare Facility
Approved Indication Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Disease Hereditary ATTR amyloidosis (hATTR) – Polyneuropathy (PN)
Therapeutic Area Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-372-9581
Fax Number 877-349-7938
Product Website amvuttrahcp.com
Full Name Arcalyst® (rilonacept)
Drug Arcalyst
Manufacturer Kiniksa Pharmaceuticals (UK), Ltd.
Route of Administration Subcutaneous
Site of Care Home
Approved Indication Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS), and Muckle-Wells Syndrome (MWS) in adults and children 12 years and older; maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and pediatric patients weighing 10 kg or more; and treatment of recurrent pericarditis (RP) and reduction in risk of recurrence in adults and children 12 years and older
Disease Cryopyrin-Associated Periodic Syndromes (CAPS); Deficiency of Interleukin-1 Receptor Antagonist (DIRA); Recurrent Pericarditis (RP)
Therapeutic Area Cardiology
Enrollment Form Link Enrollment Form
Phone Number 800-473-3261
Fax Number 877-576-6745
Product Website arcalyst.com/hcp
Full Name Austedo® (deutetrabenazine)
Drug Austedo
Manufacturer Teva Neuroscience, Inc.
Route of Administration Oral
Site of Care Home
Approved Indication Indicated in adults for the treatment of Chorea associated with Huntington’s disease and tardive dyskinesia
Disease Tardive Dyskinesia (TD); Huntington’s Disease Chorea
Therapeutic Area Psychiatry, Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-691-0718
Fax Number 877-819-2424
Product Website austedohcp.com
Full Name Austedo® XR (deutetrabenazine)
Drug Austedo XR Extended Release
Manufacturer Teva Neuroscience, Inc.
Route of Administration Oral
Site of Care Home
Approved Indication Indicated in adults for the treatment of Chorea associated with Huntington’s disease and tardive dyskinesia
Disease Tardive Dyskinesia (TD); Huntington’s Disease Chorea
Therapeutic Area Psychiatry, Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-691-0718
Fax Number 877-819-2424
Product Website www.austedohcp.com
Full Name Berinert® [C1 esterase inhibitor (human)]
Drug Berinert
Manufacturer CSL Behring
Route of Administration Intravenous
Site of Care Home
Approved Indication Treatment of acute abdominal, facial, or laryngeal hereditary angioedema (HAE) attacks in adult and pediatric patients
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website berinert.com/professional
Full Name Brineura® (cerliponase alfa)
Drug Brineura
Manufacturer BioMarin Pharmaceutical Inc.
Route of Administration Intraventricular
Site of Care Healthcare Facility
Approved Indication Indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency
Disease Ceroid Lipofuscinosis (C2LN)
Therapeutic Area Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website brineura.com/hcp
Full Name Cerdelga® (eliglustat)
Drug Cerdelga
Manufacturer Sanofi Genzyme
Route of Administration Oral
Site of Care Home
Approved Indication Long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test
Disease Gaucher Type I Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website hcp.cerdelga.com
Full Name Cerezyme® (imiglucerase)
Drug Cerezyme
Manufacturer Sanofi Genzyme
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly or splenomegaly
Disease Gaucher Type I Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website cerezyme.com/hcp
Full Name Cinryze® [C1 esterase inhibitor (human)]
Drug Cinryze
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Routine prophylaxis against angioedema attacks in adults, adolescents, and pediatric patients (6 years of age and older) with Hereditary Angioedema (HAE)
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website cinryze.com/hcp
Full Name Crysvita® (burosumab-twza)
Drug Crysvita
Manufacturer Kyowa Kirin North America
Route of Administration Subcutaneous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older
Disease Tumor-induced Osteomalacia (TIO); X-linked Hypophosphatemia (XLH)
Therapeutic Area Endocrinology & Bone Specialty
Enrollment Form Link Enrollment Form
Phone Number 800-240-7614
Fax Number 877-793-4897
Product Website crysvitahcp.com
Full Name deflazacort
Drug deflazacort
Manufacturer Generic
Route of Administration Oral
Site of Care Home
Approved Indication Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older
Disease Duchenne Muscular Dystrophy (DMD)
Therapeutic Area Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-605-1524
Fax Number 877-765-6254
Full Name Dojolvi® (triheptanoin)
Drug Dojolvi
Manufacturer Ultragenyx
Route of Administration Oral or Gastrointestinal Tube
Site of Care Home
Approved Indication The treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD)
Disease Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-546-7102
Fax Number 877-389-5186
Product Website dojolvihcp.com
Full Name Elaprase® (idursulfase)
Drug Elaprase
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II)
Disease Mucopolysaccharidosis II
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website elaprase.com/hcp
Full Name Elelyso® (taliglucerase alfa)
Drug Elelyso
Manufacturer Pfizer
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease
Disease Gaucher Type I Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website elelyso.pfizerpro.com
Full Name Elevidys™ (delandistrogene moxeparvovec-rokl)
Drug Elevidys
Manufacturer Sarepta Therapeutics, Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Treatment of adult patients with symptomatic gene mutation confirmed limb-girdle muscular dystrophy type R5 (LGMDR5)
Disease Limb-Girdle Muscular Dystrophy Type R5 (LGMDR5)
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website elevidyshcp.com
Full Name Elfabrio® (pegunigalsidase alfa-iwxj)
Drug Elfabrio
Manufacturer Chiesi
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of adults with confirmed Fabry disease
Disease Fabry disease
Therapeutic Area Neurology, Nephrology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 833-656-1056
Fax Number 636-355-3610
Product Website hcp.elfabrio.com
Full Name Emflaza® (deflazacort)
Drug Emflaza
Manufacturer PTC Therapeutics, Inc.
Route of Administration Oral
Site of Care Home or Healthcare Facility
Approved Indication Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older
Disease Duchenne Muscular Dystrophy (DMD)
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website hcp.emflaza.com
Full Name Esbriet® (pirfenidone)
Drug Esbriet
Manufacturer Genentech
Route of Administration Oral
Site of Care Home
Approved Indication The treatment of idiopathic pulmonary fibrosis (IPF)
Disease Idiopathic Pulmonary Fibrosis (IPF)
Therapeutic Area Pulmonology
Enrollment Form Link Enrollment Form
Phone Number 800-355-9366
Fax Number 877-358-9246
Product Website esbriethcp.com
Full Name Evkeeza® (evinacumab-dgnb)
Drug Evkeeza
Manufacturer Regeneron Pharmaceuticals
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older, with homozygous familial hypercholesterolemia (HoFH)
Disease Homozygous Familial Hypercholesterolemia (HoFH)
Therapeutic Area Cardiology
Enrollment Form Link Enrollment Form
Phone Number 800-645-4142
Fax Number 877-473-0199
Product Website evkeezahcp.com
Full Name Exondys 51® (eteplirsen)
Drug Exondys 51
Manufacturer Sarepta Therapeutics, Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping
Disease Duchenne Muscular Dystrophy (DMD)
Therapeutic Area Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-356-5034
Fax Number 877-339-4602
Product Website exondys51.com
Full Name Fabrazyme® (agalsidase beta)
Drug Fabrazyme
Manufacturer Sanofi Genzyme
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease
Disease Fabry disease
Therapeutic Area Neurology, Nephrology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website hcp.fabrazyme.com
Full Name Firazyr® (icatibant)
Drug Firazyr
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Subcutaneous
Site of Care Home or Healthcare Facility
Approved Indication Treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website firazyr.com/hcp
Full Name Haegarda® [C1 esterase inhibitor subcutaneous (human)]
Drug Haegarda
Manufacturer CSL Behring
Route of Administration Subcutaneous
Site of Care Home
Approved Indication Routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in patients 6 years of age and older
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website haegarda.com/hcp
Full Name Hemgenix® (etranacogene dezaparvovec-drlb)
Drug Hemgenix
Manufacturer CSL Behring
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes
Disease Hemophilia B
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Form Link Enrollment Form
Phone Number 800-975-8693
Fax Number 877-740-7535
Product Website hemgenix.com/hcp
Full Name Icatibant Acetate
Drug icatibant acetate
Manufacturer Generic
Route of Administration Subcutaneous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Full Name Ingrezza® (valbenazine)
Drug Ingrezza
Manufacturer Neurocrine Biosciences
Route of Administration Oral
Site of Care Home
Approved Indication The treatment of adults with tardive dyskinesia or chorea associated with Huntington’s disease
Disease Tardive Dyskinesia (TD); Huntington’s Disease Chorea
Therapeutic Area Psychiatry, Neurology
Enrollment Form Link Enrollment Form
Phone Number 800-279-1676
Fax Number 877-868-1681
Product Website ingrezzahcp.com
Full Name intravenous immunoglobulin (IVIG)
Drug IVIG
Manufacturer Generic
Route of Administration Subcutaneous or Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Treatment of primary humoral immunodeficiency, chronic immune thrombocytopenic purpura (ITP), or chronic inflammatory demyelinating polyneuropathy (CIDP) in adults
Disease Primary Immune Deficiency
Therapeutic Area Allergy & Immunology
Enrollment Form Link N/A
Phone Number 847-725-8100
Full Name Kalbitor® (ecallantide)
Drug Kalbitor
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Subcutaneous
Site of Care Home
Approved Indication Treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of age and older
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website kalbitor.com/hcp
Full Name Kanuma® (sebelipase alfa)
Drug Kanuma
Manufacturer Alexion Pharmaceuticals, a subsidiary of AstraZeneca
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency
Disease Lysosomal Acid Lipase Deficiency (LAL-D)
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website kanuma.com/hcp
Drug Name Leqvio® (inclisiran)
Manufacturer Novartis Pharmaceuticals
Route of Administration Subcutaneous
Approved Indication The treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of low-density lipoprotein cholesterol (LDL-C)
Disease Primary Hyperlipidemia; Heterozygous Familial Hypercholesterolemia (HeFH)
Therapeutic Area Cardiology
Enrollment Form Link Enrollment Form
Phone Numbers 800-372-6153 (Enrollment), 877-515-9670 (Support)
Product Website leqvio.com
Full Name Lumizyme® (alglucosidase alfa)
Drug Lumizyme
Manufacturer Sanofi Genzyme
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Patients with Pompe disease (GAA deficiency)
Disease Pompe Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Enrollment Form Link
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website lumizyme.com
Full Name MACI® (autologous cultured chondrocytes on a porcine collagen membrane)
Drug MACI
Manufacturer Vericel Corporation
Route of Administration Surgery
Site of Care Healthcare Facility
Approved Indication The repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults
Disease Cartilage Defect Repair
Therapeutic Area Orthopedics; Cell & Gene Therapy
Enrollment Form Link MACI Enrollment Form
Phone Number 800-388-1903
Fax Number N/A
Product Website maci.com

 

Full Name Mifepristone
Drug Mifepristone
Manufacturer Generic
Route of Administration Oral
Site of Care Home
Approved Indication Control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery
Disease Cushing’s Syndrome
Therapeutic Area Endocrinology
Enrollment Form Link Mifepristone Enrollment Form
Phone Number (888) 204-7730
Fax Number (877) 218-6288
Product Website https://www.tevausa.com/products/mifepristonetablets
Full Name miglustat
Drug Miglustat
Manufacturer Generic
Route of Administration Oral
Site of Care Home or Healthcare Facility
Approved Indication Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
Disease Gaucher Type I Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Miglustat Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Full Name Naglazyme® (galsulfase)
Drug Naglazyme
Manufacturer BioMarin Pharmaceutical Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome)
Disease Mucopolysaccharidosis VI
Therapeutic Area Neurology, Genetics
Enrollment Form Link Naglazyme Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website naglazyme.com
Full Name Nexviazyme® (avalglucosidase alfa-ngpt)
Drug Nexviazyme
Manufacturer Sanofi Genzyme
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency)
Disease Pompe Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Nexviazyme Enrollment Form Link
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website nexviazyme.com
Full Name Ofev® (nintedanib)
Drug Ofev
Manufacturer Boehringer Ingelheim
Route of Administration Oral
Site of Care Home
Approved Indication Treatment of idiopathic pulmonary fibrosis (IPF) in adults, treatment of chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype in adults, or slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD)
Disease Idiopathic Pulmonary Fibrosis (IPF); Systemic Sclerosis Interstitial Lung Disease (SSc-ILD)
Therapeutic Area Pulmonology
Enrollment Form Link Ofev Enrollment Form
Phone Number 800-373-1452
Fax Number 888-975-1456
Product Website ofev.com
Full Name Onpattro® (patisiran)
Drug Onpattro
Manufacturer Alnylam Pharmaceuticals
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Disease Hereditary ATTR amyloidosis (hATTR) – Polyneuropathy (PN)
Therapeutic Area Neurology
Enrollment Form Link Onpattro Start Form
Phone Number 800-690-8236
Fax Number 877-445-8481
Product Website onpattro.com
Full Name Oxlumo® (lumasiran)
Drug Oxlumo
Manufacturer Alnylam Pharmaceuticals
Route of Administration Subcutaneous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients
Disease Primary Hyperoxaluria Type 1 (PH1)
Therapeutic Area Nephrology
Enrollment Form Link Oxlumo Enrollment Form
Phone Number 800-460-5217
Fax Number 877-276-8563
Product Website oxlumo.com
Full Name PEDMARK® (sodium thiosulfate injection)
Drug Pedmark
Manufacturer Fennec Pharmaceuticals
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors
Disease Ototoxicity associated with cisplatin
Therapeutic Area Otolaryngology
Enrollment Form Link Pedmark Enrollment Form
Phone Number 800-385-8596
Fax Number 877-694-2545
Product Website pedmark.com
Full Name Photrexa® (riboflavin 5’-phosphate ophthalmic solution)
Drug Photrexa
Manufacturer Glaukos
Route of Administration Intraocular
Site of Care Healthcare Facility
Approved Indication The treatment of progressive keratoconus (1.1) and corneal ectasia following refractive surgery
Disease Progressive Keratoconus; Corneal Ectasia
Therapeutic Area Ophthalmology
Enrollment Form Link Photrexa Enrollment Form
Phone Number 800-550-7207
Fax Number 877-277-3139
Product Website
Full Name pirfenidone
Drug Pirfenidone
Manufacturer Generic
Route of Administration Oral
Site of Care Home
Approved Indication The treatment of idiopathic pulmonary fibrosis (IPF)
Disease Idiopathic Pulmonary Fibrosis (IPF)
Therapeutic Area Pulmonology
Enrollment Form Link Pirfenidone Enrollment Form
Phone Number 800-355-9366
Fax Number 877-358-9246
Product Website
Full Name Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat)
Drug Pombiliti & Opfolda
Manufacturer Amicus Therapeutics
Route of Administration Intravenous and oral
Site of Care Home or Healthcare Facility
Approved Indication The treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT)
Disease Pompe Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Pombiliti & Opfolda Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website pombilitiopfolda.com
Full Name Pradaxa® (dabigatran etexilate) oral pellets – pediatric
Drug Pradaxa Pediatric
Manufacturer Boehringer Ingelheim
Route of Administration Oral
Site of Care Home
Approved Indication To reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation, for the treatment of deep venous thrombosis (DVT) and pulmonary embolism (PE) in adult patients who have been treated with a parenteral anticoagulant for 5-10 days, to reduce the risk of recurrence of DVT and PE in adult patients who have been previously treated, for the prophylaxis of DVT and PE in adult patients who have undergone hip replacement surgery, for the treatment of venous thromboembolic events (VTE) in pediatric patients 8 to less than 18 years of age who have been treated with a parenteral anticoagulant for at least 5 days, or to reduce the risk of recurrence of VTE in pediatric patients 8 to less than 18 years of age who have been previously treated
Disease Venous Thromboembolism (VTE)
Therapeutic Area Hematology
Enrollment Form Link Pradaxa Enrollment Form
Phone Number 800-593-0310
Fax Number 877-709-9184
Product Website https://patientpatient.boehringer-ingelheim.com/us/pradaxa/
Full Name Roctavian™ (valoctocogene roxaparvovec-rvox)
Drug Roctavian
Manufacturer BioMarin Pharmaceutical Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test
Disease Hemophilia A
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Form Link Roctavian Enrollment Form
Phone Number 800-358-0013
Fax Number 877-631-9228
Product Website roctavian.com
Full Name Ruconest® [C1 esterase inhibitor (recombinant)]
Drug Ruconest
Manufacturer Pharming Group
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE)
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Ruconest Patient Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website ruconest.com
Full Name Sinuva® (mometasone furoate)
Drug Sinuva
Manufacturer Intersect ENT
Route of Administration Sinus Implant
Site of Care Healthcare Facility
Approved Indication The treatment of nasal polyps in patients ≥ 18 years of age who have had ethmoid sinus surgery
Disease Nasal Polyps
Therapeutic Area Allergy & Immunology
Enrollment Form Link Sinuva Enrollment Form
Phone Number 800-356-4354
Fax Number 877-574-0550
Product Website sinuva.com
Full Name Soliris® (eculizumab)
Drug Soliris
Manufacturer Alexion Pharmaceuticals, a subsidiary of AstraZeneca
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis
Disease Paroxysmal Nocturnal Hemoglobinuria (PNH); Atypical Hemolytic Uremic Syndrome (aHUS)
Therapeutic Area Hematology
Enrollment Form Link Soliris Enrollment Form
Phone Number N/A
Fax Number N/A
Product Website solirisnmosd.com
Full Name Sublocade® (buprenorphine extended-release)
Drug Sublocade
Manufacturer Indivior
Route of Administration Subcutaneous
Site of Care Healthcare Facility
Approved Indication The treatment of moderate to severe opioid use disorder in patients who have initiated treatment with a transmucosal buprenorphine-containing product, followed by dose adjustment for a minimum of 7 days
Disease Opioid Dependence
Therapeutic Area Mental Health & Addiction
Enrollment Form Link Sublocade Enrollment Form
Phone Number 800-241-1534
Fax Number 877-785-7737
Product Website sublocade.com
Full Name Takhzyro® (lanadelumab-flyo)
Drug Takhzyro
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Subcutaneous
Site of Care Home
Approved Indication Prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 2 years and older
Disease Hereditary Angioedema (HAE)
Therapeutic Area Allergy & Immunology
Enrollment Form Link Takhzyro Enrollment Form
Phone Number 800-356-4252
Fax Number 847-631-6918
Product Website takhzyro.com
Full Name tiopronin
Drug Tiopronin
Manufacturer Generic
Route of Administration Oral
Site of Care Home
Approved Indication In combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 9 years of age and older with severe homozygous cystinuria, who are not responsive to these measures alone
Disease Homozygous Cystinuria
Therapeutic Area Nephrology
Enrollment Form Link Tiopronin Enrollment Form
Phone Number 800-764-0147
Fax Number 877-848-6579
Product Website
Full Name Tzield® (teplizumab-mzwv)
Drug Tzield
Manufacturer Sanofi
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Delay the onset of Stage 3 type 1 diabetes (T1D) in adults and pediatric patients aged 8 years and older with Stage 2 T1D
Disease Type 1 Diabetes (T1D)
Therapeutic Area Endocrinology & Bone Specialty
Enrollment Form Link Tzield Enrollment Form
Phone Number 800-670-5321
Fax Number 877-655-4364
Product Website tzield.com
Full Name Ultomiris® (ravulizumab-cwvz)
Drug Ultomiris
Manufacturer Alexion (purchased by AZ)
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH)
Disease Paroxysmal Nocturnal Hemoglobinuria (PNH); Atypical Hemolytic Uremic Syndrome (aHUS)
Therapeutic Area Hematology
Enrollment Form Link Enrollment Form
Phone Number N/A
Fax Number N/A
Product Website ultomirishcp.com/nmosd
Full Name Veopoz™ (pozelimab-bbfg)
Drug Veopoz
Manufacturer Regeneron Pharmaceuticals
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease
Disease CD55-deficient protein-losing enteropathy (CHAPLE) Disease
Therapeutic Area Allergy & Immunology, Genetics
Enrollment Form Link Veopoz Enrollment Form
Phone Number 800-438-2375
Fax Number 877-440-0891
Product Website veopoz.com
Full Name Vigabatrin
Drug Generic
Manufacturer N/A
Route of Administration Oral
Site of Care Home
Approved Indication Adjunctive therapy for adults and pediatric patients 2 years of age and older with refractory complex partial seizures who have inadequately responded to several alternative treatments and for whom the potential benefits outweigh the risk of vision loss
Disease Refractory Complex Partial Seizures; Infantile Spasms
Therapeutic Area Neurology
Enrollment Form Link Vigabatrin Enrollment Form
Phone Number 800-976-9809
Fax Number 877-569-6004
Product Website N/A
Full Name Viltepso® (viltolarsen)
Drug Viltepso
Manufacturer NS Pharma
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping
Disease Duchenne Muscular Dystrophy (DMD)
Therapeutic Area Neurology
Enrollment Form Link Viltepso Enrollment Form
Phone Number 800-759-0445
Fax Number 877-286-3620
Product Website viltepso.com
Full Name Vimizim® (elosulfase alfa)
Drug Vimizim
Manufacturer BioMarin Pharmaceutical Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome)
Disease Mucopolysaccharidosis IVA
Therapeutic Area Neurology, Genetics
Enrollment Form Link Vimizim Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website vimizim.com
Full Name Vivitrol® (naltrexone)
Drug Vivitrol
Manufacturer Alkermes
Route of Administration Intramuscular
Site of Care Healthcare Facility
Approved Indication The treatment of alcohol dependence in patients who can abstain from alcohol in an outpatient setting prior to initiation of treatment with VIVITROL; The prevention of relapse to opioid dependence following opioid detoxification
Disease Alcohol Dependence; Opioid Dependence
Therapeutic Area Mental Health & Addiction
Enrollment Form Link Vivitrol Enrollment Form
Phone Number 800-373-1406
Fax Number 847-427-7975, 855-677-4641, 877-496-3716
Product Website vivitrolhcp.com
Full Name Vowst™ (fecal microbiota spores live-brpk)
Drug Vowst
Manufacturer Aimmune Therapeutics
Route of Administration Oral
Site of Care Home
Approved Indication Prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI)
Disease Recurrent C. difficile Infection (rCDI)
Therapeutic Area Infectious Disease; Gastroenterology
Enrollment Form Link Vowst Enrollment Form
Phone Number 800-485-4885
Fax Number 877-640-5179
Product Website vowsthcp.com
Full Name Vpriv® (velaglucerase alfa)
Drug Vpriv
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease
Disease Gaucher Type I Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Vpriv Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website hcp.vpriv.com
Full Name Vyepti® (eptinezumab-jjmr)
Drug Vyepti
Manufacturer Lundbeck
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The preventive treatment of migraine in adults
Disease Migraine
Therapeutic Area Neurology
Enrollment Form Link Vyepti Enrollment Form
Phone Number 800-259-7145
Fax Number 877-892-3019
Product Website vyeptihcp.com
Full Name Vyndamax® (tafamidis)
Drug Vyndamax
Manufacturer Pfizer
Route of Administration Oral
Site of Care Home
Approved Indication The treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization
Disease Hereditary ATTR amyloidosis (hATTR) – Cardiomyopathy (CM); Wild type transthyretin amyloid cardiomyopathy (wtATTR-CM)
Therapeutic Area Cardiology
Enrollment Form Link Vyndamax Enrollment Form
Phone Number 800-930-2043
Fax Number 877-684-3116
Product Website vyndamax.pfizerpro.com
Full Name Vyndaqel® (tafamidis meglumine)
Drug Vyndaqel
Manufacturer Pfizer
Route of Administration Oral
Site of Care Home
Approved Indication The treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization
Disease Hereditary ATTR amyloidosis (hATTR) – Cardiomyopathy (CM); Wild type transthyretin amyloid cardiomyopathy (wtATTR-CM)
Therapeutic Area Cardiology
Enrollment Form Link Vyndaqel Enrollment Form
Phone Number 800-930-2043
Fax Number 877-684-3116
Product Website vyndamax.pfizerpro.com
Full Name Vyondys 53® (golodirsen)
Drug Vyondys 53
Manufacturer Sarepta Therapeutics, Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping
Disease Duchenne Muscular Dystrophy (DMD)
Therapeutic Area Neurology
Enrollment Form Link Vyondys 53 Enrollment Form
Phone Number 800-356-5034
Fax Number 877-339-4602
Product Website vyondys53.com
Full Name Wainua
Drug Wainua
Manufacturer AstraZeneca
Route of Administration Subcutaneous
Site of Care Home
Approved Indication The treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Disease Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
Therapeutic Area Neurology
Enrollment Form Link Wainua Enrollment Form
Phone Number 800-986-4975
Fax Number 877-369-5207
Product Website wainuahcp.com
Full Name Xenpozyme® (olipudase alfa-rpcp)
Drug Xenpozyme
Manufacturer Sanofi Genzyme
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients
Disease Acid Sphingomyelinase Deficiency (ASMD)
Therapeutic Area Neurology, Genetics
Enrollment Form Link Xenpozyme Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Product Website xenpozyme.com
Full Name Yargesa™ (miglustat)
Drug Yargesa
Manufacturer Edenbridge Pharmaceuticals
Route of Administration Oral
Site of Care Home or Healthcare Facility
Approved Indication Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
Disease Gaucher Type I Disease
Therapeutic Area Neurology, Genetics
Enrollment Form Link Yargesa Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581
Full Name Zolgensma® (onasemnogene abeparvovec-xioi)
Drug Zolgensma
Manufacturer Novartis Gene Therapy
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene
Disease Spinal Muscular Atrophy (SMA)
Therapeutic Area Neurology; Cell & Gene Therapy
Referral Link Zolgensma Referral Form
Phone Number 800-697-5048
Fax Number 877-471-5704
Website zolgensma.com
Full Name Ztalmy® (ganaxolone)
Drug Ztalmy
Manufacturer Marinus Pharmaceuticals
Route of Administration Oral
Site of Care Home
Approved Indication The treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older
Disease Cyclin-dependent kinase-like 5 (CDKL5) Deficiency Disorder
Therapeutic Area Neurology
Referral Link Ztalmy Enrollment Form
Phone Number 844-ZTALMY1
Fax Number 844-ZTALMYF
Website ztalmy.com
Full Name Zulresso® (brexanolone)
Drug Zulresso
Manufacturer Sage Therapeutics
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of postpartum depression (PPD) in adults
Disease Postpartum Depression
Therapeutic Area Psychiatry, Mental Health & Addiction
Referral Link Zulresso Enrollment Form
Phone Number 800-811-6109
Fax Number 877-868-9682
Website zulresso.com