Specialty Pipeline

Last updated: 05.10.22

DRUG NAME/ MANUFACTURER	PROPOSED USE	COST ESTIMATE PER PATIENT	HOW IT WORKS	WHAT’S IMPORTANT
adagrasib (Mirati Therapeutics)	to treat KRAS G12C mutated locally advanced or metastatic non-small cell lung cancer (NSCLC)	$215,000/yr	KRAS is an oncogene. When mutated, oncogenes can lead to normal cells becoming cancerous. A mutation at position 12 leads to improved tumor cell survival. Adagrasib is a KRAS G12C inhibitor which is meant to inhibit growth and lead to destruction of tumor cells.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 4Q2022 Impact: Around 14% of NSCLC patients carry KRAS G12C mutations, and the cancer-linked gene is implicated in up to a third of all human cancers. KRAS G12C mutations are frequently linked to negative outcomes. Each year in the US, approximately 25,000 patients with NSCLC with G12C mutations may be candidates for treatment with a KRAS inhibitor.
arimoclomol (Miplyffa - Orphazyme)	Niemann- Pick Disease Type C (NPC)	$500,000/yr	Arimoclomol is a molecular chaperone activator that stimulates the normal cellular protein repair pathway for the treatment of Niemann- Pick Disease Type C (NPC), a very rare conditions wherein patients cannot break down lipids, including cholesterol, which collects in the liver, lungs, spleen, and brain leading to loss of nerve function.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 2023 Impact: At an estimated one case of NPC in 120,000 live births, fewer than 50 new cases are discovered in the U.S each year. The total U.S. patient population is believed to be about 200 individuals.
bardoxolone methyl (Imbarkyd - Reata)	chronic kidney disease caused by Alport Syndrome	$55,000/yr	Bardoxolone activates Nrf2, a mediator of cellular pathways that decrease inflammation. It is being developed for the treatment of chronic kidney disease (CKD) due to Alport syndrome, a genetic condition that causes faulty type IV collagen production in the small blood vessels of the kidney leading to irreversible weakening, scarring, and loss of function that eventually requires transplant or dialysis.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 2023 Impact: There are approximately 30K-60K Americans with Alport syndrome. No drugs are currently approved for Alport syndrome. On Dec. 8, 2021, an FDA advisory panel voted unanimously against approval of bardoxolone indicating the drug has not demonstrated that it is effective in slowing the progression of CKD and potential benefits don't outweigh risks. Additional efficacy and safety data are required for approval.
betibeglogene autotemcel (Zynteglo - Bluebird Bio)	β thalassemia	$2 million+ for one-time infusion	Zynteglo is a gene therapy for the treatment of transfusion-dependent β thalassemia, an inherited blood disorder caused by a mutation in the β-globin gene which leads to ineffective red blood cell production and severe anemia requiring regular transfusions. Upon extraction of the patients own stem cells, the cells are genetically modified using a lentiviral gene vector to deliver working copies of the gene to these cells. Once modified, they are returned to the patient and begin to create healthy red blood cells reducing or eliminating the need for transfusions.	Route of administration: Intraveneous Benefit coverage: Medical Anticipated FDA decision: 3Q2022 Impact: β-thalassemia is estimated to affect approximately 1 in 100,000 individuals in the general population. In the United States, thalassemia’s prevalence is approximately 1 in 272,000 or about 1,000 people.
bimekizumab (Bimzelx - UCB)	moderate-to-severe plaque psoriasis	$65,000/yr	Bimekizumab is a monoclonal antibody that blocks the effects of inflammatory mediators IL-17A and IL-17F for the treatment of moderate-to-severe plaque psoriasis. Psoriasis is an autoimmune disease caused by overactivity of the immune system resulting in overproduction of skin cells. The chronic inflammation can also contribute to cardiovascular disease and depression.	Route of administration: Subcutaneous Benefit coverage: Pharmacy Anticipated FDA decision: 2Q22 Impact: Plaque psoriasis affects approximately eight million patients in the US. Moderate-to-severe plaque psoriasis accounts for nearly 35% of psoriasis cases.
cipaglucosidase alfa (Amicus Therapeutics)	Pompe disease	$630,000/yr	Cipaglucosidase alfa is a recombinant human GAA enzyme replacement therapy for late-onset Pompe disease, a disorder caused by the deficiency of an enzyme known as acid alpha-glucosidase (GAA). Pompe disease leads to weakened muscles, and symptoms can range from moderately restricted mobility when skeletal muscles are weakened to life threatening cardiac and respiratory failure when the muscle of the heart and those that assist in breathing are impacted.	Route of administration: Intravenous Benefit coverage: Medical Anticipated FDA decision: 3Q2022 Impact: Pompe disease affects about 3,500 patients in the U.S.
deucravacitinib (Bristol Myers Squibb)	plaque psoriasis	$45,000/yr	Deucravacitinib is a tyrosine kinase 2 (TYK2) inhibitor for use in patients with moderate-to-severe plaque psoriasis. It inhibits the interleukin (IL)-12, IL-23, and Type 1 interferon pathways, which are implicated in the pathogenesis of psoriasis and other immune-mediated diseases.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 3Q2022 Impact: Plaque psoriasis affects approximately eight million patients in the US. Moderate-to-severe plaque psoriasis accounts for nearly 35% of psoriasis cases.
eladocagene exuparvovec (PTC Therapeutics)	aromatic-L-amino-acid decarboxylase (AADC) deficiency	$4M for one-time infusion	Eladocagene is a recombinant virus that delivers the gene to code for an enzyme, AADC, the deficiency of which is a rare neurologic disease which may be fatal. AACD deficiency results in decreased levels of neurotransmitters that pass signals between nerve cells leading to loss of function, seizures, problems with movement, and death.	Route of administration: Intracerebral infusion Benefit coverage: Medical Anticipated FDA decision: 4Q2022 Impact: AACD deficiency is extremely rare; about 100 patients with this disease have been identified in the U.S.
etranacogene dezaparvovec (EtranaDez - iuniQure)	hemophilia B	$1.5 million	Hemophilia B is a rare genetic bleeding disorder, characterized by a lack of blood-clotting factor IX, a blood protein that is required for the clotting cascade. Bleeding, which may be fatal, can occur externally and internally. The disease is hereditary and mainly affects males. EtranaDez uses a virus to deliver a functional copy of the faulty gene to the liver, where clotting factors are formed, allowing for the production of the missing clotting factor.	Route of administration: Intravenous infusion Benefit coverage: Medical Anticipated FDA decision: Second Half of 2022 Impact: Hemophilia B has an incidence of 1 in 500 male births with a total prevalence of around 4,000 people living with the disease in the U.S.
futibatinib (Taiho Oncology)	Locally advanced or metastatic cholangiocarcinoma	$250,000/yr	Futibatinib is a fibroblast growth inhibitor for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma, a rare cancer that forms in the bile duct and is associated with specific gene mutations. Futibatinib is a potent inhibitor of the FGFR cellular pathway, that when dysregulated, leads to tumor cell growth.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 3Q2022 Impact: Approximately 8,000 patients are diagnosed with cholangiocarcinoma each year in the United States. FGFR2 genetic aberrations are present in approximately 15% to 20% of these patients.
lecanemab (Eisai)	Alzheimer's disease (AD)	$28,000/yr	Although the cause of AD is not completely understood, certain brain changes are associated with the condition. These are beta-amyloid plaques, which are associated with atrophy of the brain and surrounding blood vessels, and tau proteins, which cause tangles within brain cells. Lecanemab is an antibody that targets the beta amyloid for the for the treatment of early Alzheimer's disease (AD).	Route of administration: Intravenous Benefit coverage: Medical Anticipated FDA decision: 2022 Impact: According to the Alzheimer’s Association, over 6.2 million Americans age 65 years and older were living with AD in 2021. Its prevalence is expected to double over the next three decades.
mirikizumab (Lilly)	moderate-to-severe ulcerative colitis (UC)	$70,000/yr	Mirikizumab is an monoclonal antibody that inhibits the action of IL-23, an inflammatory mediator associated with UC. The immune system in patients with UC attacks the large intestine causing inflammation and ulcerations that can be debilitating, life threatening, and are associated with an increased risk of colon cancer.	Route of administration: Intravenous and subcutaneous Benefit coverage: Medical/Pharmacy Anticipated FDA decision: 2023 Impact: There are approximately 1 million Americans with UC. It is the most common type of inflammatory bowel disease.
obeticholic acid (Intercept Pharmaceuticals)	nonalcoholic steatohepatitis (NASH)	$15K-$20K/yr	Obeticholic acid is a farnesoid X receptor (FXR) agonist for the treatment of NASH or nonalcoholic fatty liver disease, a common form of chronic liver disease characterized by fat buildup in the liver. It damages liver cells and causes inflammation, potentially resulting in fibrosis, cirrhosis, end-stage liver disease, liver transplant, liver cancer, and liver-related death. FXR regulates bile production, as well as fat and glucose metabolism. Obeticholic acid increases FXR activity to decrease fatty liver deposits.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 2023 Impact: It’s estimated that there are around 3M NASH patients with advanced fibrosis in the US, of which around 500,000 patients are thought to be under the care of a hepatologist or gastroenterologist. These 500,000 patients are Intercept’s target patient population for obeticholic acid.
olipudase alfa (Sanofi)	the long-term treatment of non–central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD)	$500,000+/yr	ASMD is a rare progressive genetic disorder that results from a deficiency of the enzyme acid sphingomyelinase, which is required to break down a fatty substance called sphingomyelin. In patients with ASMD, sphingomyelin and other substances accumulate in various tissues of the body. ASMD causes a spectrum of symptoms and disease severity depending on areas affected. It may cause symptoms in the nervous system, as well as gastrointestinal, bleeding, bone, cardiovascular, and lung complications. Olipudase alfa replaces the enzyme allowing for the breakdown of sphingomyelin.	Route of administration: Intravenous infusion Benefit coverage: Medical Anticipated FDA decision: 3Q2022 Impact: An estimated 1,300 people have ASMD in the U.S.
pegunigalsidase alfa (Protalix BioTherapeutics)	Fabry disease	NA	Pegunigalsidase alfa is a plant cell-expressed, recombinant enzyme for the treatment of Fabry disease (FD), a rare genetic disease caused by genetic mutations that result in galactosidase-A deficiency. Deficiency of this enzyme leads to the inability to break down and repurpose old cell materials which build-up within blood vessels and organs.	Route of administration: Intraveneous Benefit coverage: Medical Anticipated FDA decision: 2023 Impact: FD affects an estimated 1 in 40,000 to 60,000 males, while female prevalence remains unknown. There are about 7,000 patients in the US with FD.
spesolimab (Boehringer Ingelheim)	generalized pustular psoriasis (GPP) flares	NA	GPP is a rare, chronic, systemic, potentially life-threatening skin disease, which is clinically distinct from plaque psoriasis. GPP is caused by white blood cells accumulating in the skin, resulting in painful pustules all over the body. Spesolimab is a monocolonal antibody that inhibits the action of IL-36, an inflammatory mediator called a cytokine, by blocking its receptor and interfering with the inflammatory pathway in GPP.	Route of administration: Intraveneous infusion Benefit coverage: Medical Anticipated FDA decision: 2Q2022 Impact: GPP has a varied prevalence across different geographical regions and more women are affected than men. It is estimated to affect approximately 2 per million people; therefore, the U.S. prevalence may be around 600 patients.
teclistamab (Janssen)	elapsed or refractory multiple myeloma	$420,000/yr	Teclistamab is an antibody for the treatment of relapsed or refractory multiple myeloma, an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow. Teclistamab is an antibody targeting both BCMA and CD3 receptors. Multiple myeloma cells have a high amount of BCMA on their surfaces. Teclistamab directs T-cells, or "killer" cells, to destroy BCMA-expressing tumor cells.	Route of administration: Subcutaneous Benefit coverage: Medical Anticipated FDA decision: 3Q2022 Impact: Approximately 32,000 new cases of multiple myeloma are diagnosed each year in the U.S. Most patients eventually become refractory to available treatment options.
teplizumab (Provention Bio)	to delay or prevent the onset of type 1 diabetes in at-risk individuals	$500,000 per patient over two cycles	Teplizumab is a humanized monoclonal antibody to prevent or delay clinical type one diabetes (T1D) in at-risk individuals. There is a genetic predisposition to inheriting T1D which, coupled with environmental triggers that are not fully known, leads to destruction of the insulin producing cells of the pancreas by the body's T-cells. Teplizumab is engineered to alter the function of the T-cells that mediate this destruction and prevent or delay T1D onset.	Route of administration: Intravenous Benefit coverage: Medical Anticipated FDA decision: 3Q2022 Impact: Newly diagnosed T1D, also known as juvenile diabetes, is considered an orphan indication due to its high disease burden and unmet need in the U.S. Provention Bio believes teplizumab could have a target population of 30,000 annually, representing about 15% of the 200,000 who have two or more autoantibodies, dysglycemia and would be genetic relatives at risk for TD1. The company also believes that awareness will lead to further screening of T1D relatives and could encourage broader testing globally with a total addressable patient population of 2.3 million people.
valoctocogene roxaparvovec (Roctavian – BioMarin Pharmaceuticals)	hemophilia A	$2-3 million as one-time infusion	Valoctocogene is a viral vector gene therapy that enters cells to replace the mutated gene needed to produce factor VIII, a blood clotting factor. Factor VIII deficiency results in hemophilia A, a condition that causes spontaneous and uncontrolled bleeding.	Route of administration: Intraveneous Benefit coverage: Medical Anticipated FDA decision: Second Half of 2022 Impact: Hemophilia is a mainly inherited condition that affects about 16,000 Americans – predominantly boys and men. Approximately 50-60% of patients have severe hemophilia A (factor VIII < 2% of normal). According to BioMarin, about 2,400 patients in the US will be candidates for treatment with valoctocogene.
vutrisiran (Alnylam)	polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis	$500,000/yr	Vutrisiran targets a specific protein, transthyretin (TTR), for the treatment of the polyneuropathy (nerve damage) of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR is a rare, debilitating disease that is caused by misfolded TTR protein that causes amyloid to deposit in areas of the cardiovascular system, central nervous system (CNS), and gastrointestinal tract. This leads to dysfuntion in multiple bodily systems and is fatal.	Route of administration: Subcutaneous Benefit coverage: Medical Anticipated FDA decision: 3Q2022 Impact: hATTR affects about 7,000 patients in the US but is thought to be underdiagnosed.