Specialty Drug Approvals 2017 HIGHLIGHTS & 2018 PROJECTIONS
This document contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements give current expectations or forecasts of future events or our future financial or operating performance. The forward-looking statements contained in this document are based on management’s good-faith belief and reasonable judgment based on current information, and these statements are qualified by important risks and uncertainties, many of which are beyond our control, that could cause our actual results to differ materially from those forecasted or indicated by such forward-looking statements. These risks include the number of patients prescribed such drug(s) currently and in the future, patient’s adherence to such drug(s), the number of distributors on panel and our relative distribution share, the timing of drug sales, the cost of such drug(s) and reimbursement rates by payors, drug competition, and the factors set forth in “Risk Factors” in Diplomat’s Annual Report on Form 10-K for the year ended Dec. 31, 2016, and in subsequent reports filed with or furnished to the Securities and Exchange Commission. Except as may be required by any applicable law, Diplomat assumes no obligation to publicly update such forward-looking statements, which are made as of the date hereof or the earlier date specified herein, whether as a result of new information, future developments, or otherwise. Certain information in this presentation concerning our industry and the markets in which we operate is derived from publicly available information released by third-party sources, including independent industry and research organizations, and management estimates. Management estimates are derived from publicly available information released by independent industry and research analysts and other third-party sources, as well as data from our internal research, and are based on assumptions made by us upon reviewing such data and our knowledge of such industry and markets, which we believe to be reasonable. We believe the data from these third-party sources is reliable. In addition, projections, assumptions, and estimates of the future performance of the industry in which we operate and our future performance are necessarily subject to uncertainty and risk due to a variety of factors, as discussed in Diplomat’s reports filed with the Securities and Exchange Commission. These and other factors could cause results to differ materially from those expressed in the estimates made by these third-party sources.
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Diplomat Pharmacy Services | Specialty Drug Approvals
AT D I P L O M AT,
Diplomat Pharmacy Services | Specialty Drug Approvals
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DRUG APPROVAL TRENDS1,2 The FDA's Center for Drug Evaluation and Research (CDER) approved 46 novel new drugs in 2017 (as of Dec. 28). That's the most novel new drug approvals in a year since 1996. It's more than double the total from 2016. Year
Novel New Drug Approvals*
2017 (as of Dec. 22)
46
2016
22
2015
45
2014
41
2005–2013
25 (average per year)
Of the 46 novel new drug approvals in 2017, about half are considered specialty drugs (depending on the definition). This is a similar proportion to recent years. The FDA's Center for Biologics Evaluation and Research (CBER) approved approximately 20 biologic applications in 2017. Excluding the approvals used for laboratory tests, the CBER approved nine new branded biologics to treat or prevent diseases.** Combining the CDER approvals with the biologics that meet the above criteria, there were 55 total drug and biologic approvals in 2017 (as of Dec. 28).
*Values reflect the FDA CDER website for novel drug approval totals. Includes novel new drug approvals only. Expanded indications, non-novel drug approvals, and new formulations or combinations are not included. **Values reflect the FDA CBER website.
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After a substantial decrease in 2016, the number of novel new drug approvals once again reached an impressive total for 2017. Beyond the numbers, it was an eventful year in terms of the types specialty products approved. Highlights include: • The first chimeric antigen receptor T cell (CAR-T) therapies reached the market, with approvals for specific types of leukemia and lymphoma. This innovative mechanism of action involves modifying a patient's own T cells to fight cancer. • The first drug to earn FDA approval for primary progressive multiple sclerosis (PPMS) provided an on-label treatment option for patients with this subtype of multiple sclerosis.
• A new treatment for amyotrophic lateral sclerosis (ALS) reached the market for the first time in more than 20 years. • The first directly administered gene therapy was approved a few days before the end of the year. The initial indication for this product is for a rare form of vision loss. However, gene therapy overall has generated a tremendous interest as a potentially game-changing form of treatment for a broad scope of diseases rooted in genetic abnormalities.
Diplomat Pharmacy Services | Specialty Drug Approvals
Introduction1,2,3,4,5,6
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RARE DISEASES6,7,8 Rare diseases—conditions affecting fewer than 200,000 patients at a given time—have been in the spotlight for drug development this year. Conditions that had novel drug approvals in 2017 include hemophilia, mucopolysaccharidosis, ALS, hereditary angioedema (HAE), and Duchenne muscular dystrophy. Many rare diseases have limited or no available treatments, so focus on drug development in this area is expected to continue. Radicava® (edaravone) became the second FDA-approved treatment for ALS and the first since 1995. Approximately 12,000–15,000 patients in the U.S. have ALS. The disease has a poor life expectancy, with most patients not living more than five years after symptoms appear. Edaravone is administered via IV infusion in cycles of 10–14 days on with 14 days off. Another interesting product is Hemlibra® (emicizumab-kxwh), a subcutaneously administered treatment approved for hemophilia A in patients with factor VIII
inhibitors. About one in every 5,000 males born in the U.S. has hemophilia, with approximately 80 percent of those having hemophilia A. Emicizumab-kxwh allows for a more convenient route of administration for patients compared to traditional IV factor products. Trials are also ongoing for emicizumab-kxwh for patients without inhibitors. Luxturna™ (voretigene neparvovec-rzyl) earned FDA approval to treat biallelic RPE mutation– associated retinal dystrophy. This disease leads to vision loss and is caused by genetic mutation. This product is the first directly administered gene therapy approved in the U.S., affecting about 1,000–2,000 patients. Voretigene is administered as a one-time dose and works by placing a normal copy of the RPE65 gene into the retinal cells. This corrects the retinal cells to create a normal version of a protein needed to convert light into an electric signal in the eye, leading to improved vision. The industry is watching closely, as gene therapy could later be an option for larger patient populations.
ONCOLOGY9,10,11,12 It was an impressive year for oncology drug approvals, with 11 new products approved in 2017 (as of Dec. 28). Kymriah™ (tisagenlecleucel) and Yescarta™ (axicabtagene ciloleucel) became the first two CAR-T therapies approved, earning indications for B-cell acute lymphoblastic leukemia and B-cell lymphoma, respectively. CAR-T is an innovative treatment that has created buzz in the oncology world. Although the initial FDA approvals for these products provided a rather narrow label, CAR-T as a mechanism of action is thought to have wide-ranging potential across multiple cancer types. Expect to see CAR-T continue to be a hot topic in oncology, with more new products and expanded indications reaching the market. CAR-T treatments deservingly earned a lot of attention in oncology. However, they can be accessed and dispensed at certified treatment centers only. This is due to their unique manufacturing process: using a patient’s own T cells, modifying them in a lab, and sending them back for in-clinic
administration. In the specialty pharmacy world, notable new oncolytics to reach the market in 2017 include Rydapt® (midostaurin) and Idhifa® (enasidenib). These were each approved as oral treatments for acute myeloid leukemia (AML). Before these approvals, IV oncolytics were the only available treatments for AML. Approximately 21,380 diagnoses of AML were expected in 2017, but these drugs are approved only for patients with specific genetic mutations. Midostaurin is approved for patients with the FLT3 genetic mutation, while enasidenib is for patients with IDH2 genetic mutation. Recently, oncology drug development has focused on immunotherapy options and targeted agents. These targeted options have led to the use of basket clinical trials, where drugs are studied for multiple cancer types at once. Manufacturers also continue to pursue expanded indications for drugs to be used earlier in treatment or for more cancer types. This has made the oncology drug development landscape intriguing.
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IMMUNOLOGY & MULTIPLE SCLEROSIS4,13,14 The treatment landscape for immunology and multiple sclerosis (MS) has become extremely competitive. For many disease states (such as rheumatoid arthritis, psoriasis, and relapsing forms of multiple sclerosis), several effective treatment options are already available. One exception is primary progressive multiple sclerosis (PPMS), where Ocrevus™ (ocrelizumab) became the first approved product for its treatment. It also acquired approval for relapsing forms of MS. About 15 percent of patients with MS have the primary progressive form. The total population of MS patients in the U.S. is approximately 400,000, so about 60,000 PPMS patients now have an FDA-approved treatment option. Before the launch of ocrelizumab, most patients with PPMS were likely approved for other types of MS. Dupixent® (dupilumab) was approved with a novel mechanism of action to treat eczema (atopic dermatitis) in patients not adequately controlled with current treatment options. Given the large population affected by eczema—about 17.8 million people in the
U.S.—and lack of targeted therapies providing market competition, dupilumab could be one of the most-used drugs approved in 2017. Dupilumab is administered subcutaneously. Previously available eczema treatments were primarily topical options, phototherapy, or common immunomodulatory treatments— all having limited scope in specialty pharmacy. Dupilumab's subcutaneous route of administration and high cost give it some qualities often associated with drugs dispensed by a specialty pharmacy. Since it is an open-distribution product, dispensing of Dupixent will not be limited to specialty pharmacies unless directed by payers and/or industry. Focusing on psoriasis, Tremfya™ (guselkumab) became the first selective IL-23 blocker approved in the U.S. It demonstrated impressive results in clinical trials, showing better skin clearance than Humira® (adalimumab). Guselkumab is most similar to Stelara® (ustekinumab), an IL-12 and -23 antagonist, in terms of mechanism of action.
New Approvals & Expanded Indications (Does not include IV oncology products)
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Diplomat Pharmacy Services | Specialty Drug Approvals
2017 SPECIALTY DRUGS
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Approval Date
Drug
Manufacturer(s)
Indication(s)
Route
Diplomat Pharmacy Services | Specialty Drug Approvals
Q1 2017 Jan. 18
Imbruvica® (ibrutinib)*
Pharmacyclics
Marginal zone lymphoma
Oral
Feb. 9
Emflaza™ (deflazacort)
Marathon
Duchenne muscular dystrophy
Oral
Feb. 15
Siliq™ (brodalumab)
Valeant
Psoriasis
Sub-Q
Feb. 22
Revlimid® (lenalidomide)*
Celgene
Multiple myeloma as maintenance therapy after stem-cell transplant
Oral
Feb. 28
Xermelo™ (telotristat ethyl)
Lexicon
Carcinoid syndrome
Oral
March 13
Kisqali® (ribociclib)
Astex, Novartis
Breast cancer, HR+, HER2-
Oral
March 27
Zejula™ (niraparib)
Tesaro
Ovarian cancer
Oral
March 28
Dupixent® (dupilumab)
Sanofi, Regeneron
Atopic dermatitis
Sub-Q
March 28
Ocrevus™ (ocrelizumab)
Genentech, Roche
Multiple sclerosis, primary progressive and relapsing forms
IV
March 31
Ibrance® (palbociclib)*
Pfizer
First-line HR+, HER2- breast cancer
Oral
Q2 2017 April 3
Austedo™ (deutetrabenazine)
Teva
Huntington's disease
Oral
April 7
Harvoni® (ledipasvir + sofosbuvir)*
Gilead
Hepatitis C in pediatric patients
Oral
April 7
Sovaldi® (sofosbuvir)*
Gilead
Hepatitis C in pediatric patients
Oral
April 11
Ingrezza™ (valbenazine)
Mitsubishi Tanabe, Neurocrine
Tardive dyskinesia
Oral
April 21
Renflexis™ (infliximab-abda)
Samsung Bioepis
Biosimilar of Remicade®
IV
April 27
Brineura™ (cerliponase alfa)
BioMarin
CLN2 disease (Batten disease)
Intraventricular
April 27
Stivarga® (regorafenib)*
Bayer
Hepatocellular carcinoma
Oral
April 28
Alunbrig™ (brigatinib)
Ariad
Non-small-cell lung cancer, ALK+
Oral
*Expanded indication or new formulation.
Rydapt® (midostaurin)
Novartis
Acute myeloid leukemia with FLT3 genetic mutation
Oral
April 28
Tymlos™ (abaloparatide)
Radius
Osteoporosis
Sub-Q
May 5
Radicava™ (edaravone)
Mitsubishi Tanabe
Amyotrophic lateral sclerosis
IV
May 17
Kalydeco (ivacaftor)*
Vertex
Additional genetic mutations in cystic fibrosis
Oral
May 22
Actemra® (tocilizumab)*
Genentech
Giant cell arteritis
Sub-Q
May 22
Kevzara® (sarilumab)
Sanofi and Regeneron
Rheumatoid arthritis
Sub-Q
May 26
Zykadia® (ceritinib)*
Novartis
Non-small-cell lung cancer, ALK+, first-line use
Oral
May 31
Rebinyn® (coagulation factor IX (recombinant), glycoPEGylated)
Novo Nordisk
Hemophilia B
IV
June 22
Haegarda® (C1 esterase inhibitor [human])*
CSL Behring
Hereditary angioedema, new formulation of Berinert
Sub-Q
June 22
Rituxan Hycela™ (rituximab + hyaluronidase human)*
Genentech
Follicular lymphoma, diffuse large B cell lymphoma, chronic lymphocytic leukemia
Sub-Q
June 23
Tafinlar® (dabrafenib) + Mekinist® (trametinib)*
Novartis
Non-small-cell lung cancer, BRAF+
Oral
June 29
Triptodur™ (triptorelin)
Arbor
Precocious puberty
IM
June 30
Orencia® (abatacept)*
Bristol-Myers Squibb
Psoriatic arthritis
Sub-Q
Q3 2017 July 7
Endari™ (L-glutamine)
Emmaus
Sickle cell disease
Oral
July 13
Tremfya™ (guselkumab)
Janssen
Psoriasis
Sub-Q
July 17
Nerlynx™ (neratinib)
Puma
Breast cancer, HER2+
Oral
July 18
Vosevi™ (sofosbuvir + velpatasvir + voxilaprevir)
Gilead
Hepatitis C, pangenotypic
Oral
July 21
Benlysta® (belimumab)*
GlaxoSmithKline
Lupus
Sub-Q
Aug. 1
Epclusa® (sofosbuvir + velpatasvir)*
Gilead
Hepatitis C in patients co-infected with HIV
Oral
*Expanded indication or new formulation.
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April 28
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Aug. 1
Idhifa® (enasidenib)
Agios
Acute myeloid leukemia, IDH2+
Oral
Aug. 1
Imbruvica® (ibrutinib)*
Pharmacyclics
Graft-versus-host disease
Oral
Aug. 1
Nityr™ (nitisinone)*
Cycle
Hereditary tyrosinemia type-1
Oral
Aug. 3
Mavyret™ (glecaprevir + pibrentasvir)
AbbVie
Hepatitis C, pangenotypic
Oral
Aug. 17
Lynparza® (olaparib)*
AstraZeneca
Ovarian cancer, earlier-line treatment
Oral
Aug. 25
Faslodex® (fulvestrant)*
AstraZeneca
Breast cancer, HR+, HER2-, as monotherapy
IM
Aug. 29
Cyltezo™ (adalimumab-adbm)
Boehringer Ingelheim
Biosimilar of Humira®
Sub-Q
Aug. 30
Actemra® (tocilizumab)*
Genentech
Cytokine release syndrome related to CAR-T administration
IV
Aug. 30
Austedo™ (deutetrabenazine)*
Teva
Tardive dyskinesia
Oral
Aug. 30
Kymriah™ (tisagenlecleucel)
Novartis
B cell precursor acute lymphoblastic leukemia
IV
Sept. 5
Tracleer® (bosentan)*
Actelion
Pulmonary arterial hypertension in pediatric patients
Oral
Sept. 14
Privigen® (immune globulin intravenous [human] 10% liquid)*
CSL Behring
Chronic inflammatory demyelinating polyneuropathy
IV
Sept. 15
Somatuline® Depot (lanreotide)*
Ipsen
Carcinoid syndrome
Sub-Q
Sept. 28
Verzenio® (abemaciclib)
Eli Lilly
Breast cancer, HR+, HER2-
Oral
Q4 2017 Oct. 3
Glatiramer acetate; 20 and 40 mg generics
Mylan
Multiple sclerosis
Sub-Q
Oct. 13
Stelara (ustekinumab)*
Janssen
Plaque psoriasis in adolescent patients
Sub-Q
Oct. 18
Yescarta™ (axicabtagene ciloleucel)
Kite
Refractory large B cell lymphoma
IV
Oct. 20
Simponi ARIA® (golimumab)*
Janssen
Psoriatic arthritis and ankylosing spondylitis
IV
*Expanded indication or new formulation.
Soliris® (eculizumab)*
Alexion
Myasthenia gravis
IV
Oct. 31
Calquence® (acalabrutinib)
AstraZeneca
Mantle cell lymphoma
Oral
Nov. 6
Alecensa® (alectinib)*
Genentech
Non-small-cell lung cancer, ALK+, first-line setting
Oral
Nov. 6
Zelboraf® (vemurafenib)*
Genentech
Erdheim-Chester disease
Oral
Nov. 9
Sprycel® (dasatinib)*
Bristol-Myers Squibb
Pediatric patients with Ph+ chronic myeloid leukemia
Oral
Nov. 14
Fasenra™ (benralizumab)
AstraZeneca
Severe eosinophilic asthma, as an add on maintenance treatment
Sub-Q
Nov. 15
Mepsevii™ (vestronidase alfa)
Ultragenyx
Mucopolysaccharidosis type VII
IV
Nov. 16
Hemlibra® (emicizumab-kxwh)
Genentech
Hemophilia A in patients with inhibitors
Sub-Q
Nov. 16
Sutent® (sunitinib malate)*
Pfizer
Adjuvant treatment for patients at high risk of recurrent renal cell carcinoma
Oral
Nov. 21
Juluca® (dolutegravir + rilpivirine)*
Viiv
HIV, as maintenance treatment for virologically suppressed patients
Oral
Nov. 22
Isentress® (raltegravir)*
Merck
HIV in newborns
Oral
Dec. 1
Repatha® (evolocumab)*
Amgen
Reduction of risk for heart attack, stroke, and coronary revascularization in patients with cardiovascular disease
Sub-Q
Dec. 1
Taltz® (ixekizumab)*
Eli Lilly
Psoriatic arthritis
Sub-Q
Dec. 13
Ixifi™ (infliximab-qbtx)
Pfizer
Biosimilar of Remicade®
IV
Dec. 19
Bosulif® (bosutinib)*
Pfizer
Ph+ chronic myeloid leukemia, first-line setting
Oral
Dec. 19
Cabometyx® (cabozantinib)*
Exelixis
Renal cell carcinoma, first-line setting
Oral
Dec. 19
Luxturna™ (voretigene neparvovec-rzyl)
Spark
Vision loss due to biallelic RPE65mutation-associated retinal dystrophy
IV
*Expanded indication or new formulation.
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Oct. 23
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SPECIALTY DRUG PIPELINE15,16,17 In 2018, certain pipeline trends are expected to continue. Oncology and rare diseases will still be a focus of drug development for many manufacturers. Gene and CAR-T therapies will make headlines as more study results become available from clinical trials. While these treatments were first approved in 2017 and currently have limited FDA-approved indications, they will expand their scope in 2018 to reach more disease states and patient populations. The cost of CAR-T and gene therapies is a major issue that will need to be addressed and might serve as a limiting factor for how widespread their use becomes. The cost for the two currently approved CAR-T treatments is approximately $373,000–475,000. The price for voretigene neparvovecrzyl has not yet been released, but some analysts predict it will be in the ballpark of $1 million. While these are high-cost products, each is given as a one-time dose, unlike most drugs. Another area of interest in 2018 is new agents to treat migraine headache. Erenumab by Amgen and Novartis; fremanezumab by Teva; and galcanezumab from Eli Lilly are subcutaneously administered drugs. Each have filed with the FDA to treat this debilitating condition that might impact up to 12 percent of the population. All three products target calcitonin-gene-related peptide (CGRP), a new mechanism of action for migraines that has shown promise in clinical trials.
2018 SPECIALTY DRUGS
Expected FDA Decisions
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SPECIALTY DRUG PIPELINE AGENTS16,17 Drug Name Manufacturer(s)
Proposed Indication(s)
Drug Class Mechanism
Route
Upcoming Events
Diplomat Pharmacy Services | Specialty Drug Approvals
ONCOLOGY Apalutamide Janssen
Non-metastatic castration resistant prostate cancer
Androgen receptor antagonist
Oral
Estimated PDUFA: Aug. 11, 2018
Binimetinib + Encorafenib Array
Melanoma, BRAF+
MEK inhibitor + BRAF kinase inhibitor
Oral
PDUFA: June 30, 2018
Duvelisib Verastem
Chronic lymphocytic leukemia/small lymphocytic lymphoma, follicular lymphoma
PI3K inhibitor
Oral
FDA decision: late 2018
Filgrastim biosimilar Adello
Same indications as NeupogenÂŽ
Leukocyte growth factor
SubQ
FDA decision: early 2018
Fostamatinib Rigel
Immune thrombocytopenic purpura
SYK inhibitor
Oral
PDUFA: April 17, 2018
Ivosidenib Agios
Acute myeloid leukemia, IDH1+
IDH1 inhibitor
Oral
FDA decision: late 2018
Larotrectinib Loxo
TRK+ cancers
TRK inhibitor
Oral
FDA decision: mid- to late-2018
Talazoparib BioMarin, Pfizer
Breast cancer, BRCA+
PARP inhibitor
Oral
FDA decision: late 2018
RARE DISEASE Arikayce™ (liposomal amikacin) Insmed
Non-tuberculosis mycobacterium infection of the lung
Antibiotic
Inhalant
FDA decision: mid- to late-2018
Burosumab Ultragenyx
X-linked hypophosphatemia
FGF inhibitor
Sub-Q
PDUFA: April 17, 2018
Hemophilia A
Pegylated recombinant coagulation factor VIII
IV
FDA decision: mid-2018
Epidiolex® (cannabidiol) GW
Dravet syndrome, Lennox-Gastaut syndrome
Cannabinoid receptor antagonist
Oral
FDA decision: mid-2018
Eptacog beta (LR769) LFB
Hemophilia A and B
Factor VIIa
IV
FDA decision: early 2018
Inotersen Ionis
Amyloidosis
TTR inhibitor
Sub-Q
Estimated PDUFA: May 6, 2018
Lanadelumab Shire
Hereditary angioedema
KLK inhibitor
Sub-Q
FDA decision: mid-2018
Linhaliq™ (ciprofloxacin) Aradigm
Non-cystic fibrosis bronchiectasis
Antibiotic
Inhaled
PDUFA: Jan. 26, 2018
Migalastat Amicus
Fabry disease
Galactose-alpha 1 binder
Oral
FDA decision: mid- to late-2018
Patisiran Alnylam
Amyloidosis
TTR inhibitor
IV
PDUFA: May 16, 2018
Pegvaliase Biomarin
Phenylketonuria
Enzyme substitute
Sub-Q
PDUFA: May 28, 2018
Plasminogen Prometic
Hypoplasminogenemia
Plasminogen replacement
IV
PDUFA: April 14, 2018
Stannsoporfin Mallinckrodt
Severe hyperbilirubinemia
Heme oxygenase inhibitor
IM
FDA decision: mid-2018
Tezacaftor + Kalydeco® (ivacaftor) Vertex
Cystic fibrosis
CFTR corrector + CFTR potentiator
Oral
PDUFA: Feb. 28, 2018
Volanesorsen Ionis
Familial chylomicronemia syndrome
Apolipoprotein CIII inhibitor
Sub-Q
PDUFA: Aug. 30, 2018
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Diplomat Pharmacy Services | Specialty Drug Approvals
Damoctocog alfa (BAY 94-9027) Bayer
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IMMUNOLOGY & MULTIPLE SCLEROSIS Adalimumab biosimilar (M923) Momenta
Same indications as Humira®
TNF blocker
Sub-Q
FDA decision: early 2018; delayed product launch likely
Baricitinib Incyte, Eli Lilly
Rheumatoid arthritis
JAK inhibitor
Oral
FDA decision: mid- to late-2018
Ozanimod Celgene, Receptos
Multiple sclerosis
S1P modulator
Oral
FDA decision: late 2018
Tildrakizumab Sun, Merck
Psoriasis
IL-23 antagonist
Sub-Q
Estimated PDUFA: March 24, 2018
OTHER Avatrombopag Dova
Thrombocytopenia
Thrombopoietin agonist
Oral
PDUFA: May 21, 2018
Bictegravir + Emtriva® (emtricitabine) + Vemlidy® (tenofovir alafenamide) Gilead
HIV
INSTI + NRTI + NRTI
Oral
PDUFA: Feb. 12, 2018
Erenumab Amgen, Novartis
Migraine headache
CGRP inhibitor
Sub-Q
PDUFA: May 17, 2018
Fremanezumab Teva
Migraine headache
CGRP inhibitor
Sub-Q
PDUFA: Aug. 17, 2018
Galcanezumab Eli Lilly
Migraine headache
CGRP inhibitor
Sub-Q
Estimated PDUFA: Oct. 11, 2018
Ibalizumab TaiMed, Theratechnologies
HIV
HIV-1 entry inhibitor
IV
PDUFA: Jan. 3, 2018
Ulipristal acetate Allergan
Uterine fibroid tumors
Progesterone receptor modulator
Oral
FDA decision: mid-2018
SPECIALTY DRUG NEW INDICATIONS, COMBINATIONS, & FORMULATIONS16,17 Drug Name Manufacturer(s)
Proposed Current Indication(s) Expanded Indication(s)
Route
Upcoming Events
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Gilotrif (afatinib) Boehringer Ingelheim
Non-small-cell lung cancer, certain EGFR exon 19 or 21 genetic mutations
Non-small-cell lung cancer, certain other EGFR exon 21 genetic mutations
Oral
Estimated PDUFA: April 13, 2018
Lenvima® (lenvatinib) Eisai
Hepatocellular carcinoma, second-line or later
Hepatocellular carcinoma, first-line setting
Oral
Estimated PDUFA: March 26, 2018
Lynparza® (olaparib) AstraZeneca
Ovarian cancer
Breast cancer, BRCA+, HER2-
Oral
FDA decision: early 2018
Rubraca® (rucaparib) Clovis
Ovarian cancer, BRCA+, after two or more previous treatments
Ovarian cancer, second-line or later as maintenance treatment
Oral
PDUFA: April 6, 2018
Tafinlar® (dabrafenib) + Mekinist® (trametinib) Novartis
Unresectable or metastatic melanoma and non-small cell lung cancer, BRAF+
Melanoma, BRAF+ in an adjuvant setting
Oral
FDA decision: mid- to late-2018
Tagrisso® (osimertinib) AstraZeneca
Non-small-cell lung cancer, T790M+, after previous treatment
Non-small-cell lung cancer, EGFR+, first-line setting
Oral
FDA decision: early- to mid-2018
Verzenio™ (abemaciclib) Eli Lilly
Breast cancer, HR+, HER2-, after prior treatment
Breast cancer, HR+, HER2-, first-line setting
Oral
Estimated PDUFA: April 12, 2018
Xgeva® (denosumab) Amgen
Skeletal related events in patients with bone metastasis associated with solid tumors
Skeletal related events in patients with bone metastasis associated with multiple myeloma
Sub-Q
PDUFA: Feb. 3, 2018
Yonsa™ (abiraterone acetate) Churchill
Prostate cancer
Prostate cancer, new formulation
Oral
PDUFA: March 19, 2018
Zytiga® (abiraterone acetate) Janssen
Metastatic castration-resistant prostate cancer
Prostate cancer, high risk-metastatic hormone treatment-naïve
Oral
FDA decision: mid-2018
Diplomat Pharmacy Services | Specialty Drug Approvals
ONCOLOGY
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IMMUNOLOGY & MULTIPLE SCLEROSIS Cimzia® (certolizumab) UCB
Crohn's disease, rheumatoid arthritis, psoriatic arthritis, and ankylosing spondylitis
Psoriasis
Sub-Q
Estimated PDUFA: May 25, 2018
Xeljanz® (tofacitinib) Pfizer
Rheumatoid arthritis
Ulcerative colitis
Oral
Estimated PDUFA: March 2018
OTHER Prezista® (darunavir) + Tybost® (cobicistat) + Emtriva® (emtricitabine) + Vemlidy® (tenofovir alafenamide) Johnson & Johnson, Gilead
Components of HIV treatment as individual products
HIV combination treatment
Prolia® (denosumab) Amgen
Osteoporosis
Osteoporosis related to glucocorticoid use
Sub-Q
PDUFA: May 28, 2018
Samsca® (tolvaptan) Otsuka
Hyponatremia
Autosomal dominant polycystic kidney disease
Oral
PDUFA: April 24, 2018
Oral
FDA decision: mid-2018
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FDA approval brings first gene therapy to the United States. U.S. Food & Drug Administration Website. fda. gov/NewsEvents/Newsroom/PressAnnouncements/ ucm574058.htm. Accessed Dec. 18, 2017. FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma. U.S. Food & Drug Administration Website. fda.gov/NewsEvents/ Newsroom/PressAnnouncements/ucm581216.htm. Accessed Dec. 18, 2017. FDA approves new combination treatment for acute myeloid leukemia. U.S. Food & Drug Administration Website. fda.gov/NewsEvents/Newsroom/ PressAnnouncements/ucm555778.htm. Accessed Dec. 18, 2017. FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia. fda.gov/ NewsEvents/Newsroom/PressAnnouncements/ ucm569421.htm. Accessed Dec. 18, 2017. FDA approves new eczema drug Dupixent. U.S. Food & Drug Administration Website. fda.gov/NewsEvents/ Newsroom/PressAnnouncements/ucm549078.htm. Accessed Dec. 18, 2017. Janssen announces U.S. FDA approval of Tremfya™ (guselkumab) for the treatment of moderate to severe plaque psoriasis. Johnson & Johnson Website. jnj. com/media-center/press-releases/janssen-announcesus-fda-approval-of-tremfya-guselkumab-for-thetreatment-of-moderate-to-severe-plaque-psoriasis. Accessed Dec. 18, 2017. Raising money for migraine research. Migraine Research Foundation Website. migraineresearchfoundation.org/about-migraine/ migraine-facts/. Accessed Dec. 18, 2017. BioPharm Insight. Infinata. biopharminsight.com. Accessed December 2017. PDUFA Calendar. Biopharmacatalyst.com Website. biopharmcatalyst.com/calendars/pdufa-calendar. Accessed December 2017.
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Diplomat Pharmacy Services | Specialty Drug Approvals
REFERENCES 1. Novel Drug Approvals for 2017. U.S. Food & Drug Administration Website. fda.gov/Drugs/ DevelopmentApprovalProcess/DrugInnovation/ ucm537040.htm. Accessed Dec. 22, 2017. 2. 2017 Biological License Application Approvals. U.S. Food & Drug Administration Website. fda.gov/BiologicsBloodVaccines/ DevelopmentApprovalProcess/ BiologicalApprovalsbyYear/ucm547553.htm. Accessed Dec. 22, 2017. 3. Chimeric Antigen Receptor (CAR) T-cell Therapy. Leukemia & Lymphoma Society Website. lls.org/ treatment/types-of-treatment/immunotherapy/ chimeric-antigen-receptor-car-t-cell-therapy. Accessed Dec. 15, 2017. 4. FDA approves new drug to treat multiple sclerosis. U.S. Food & Drug Administration Website. fda.gov/ NewsEvents/Newsroom/PressAnnouncements/ ucm549325.htm. Accessed Dec. 15, 2017. 5. ALS association applauds FDA for speedy approval of new ALS drug Radicava (edaravone). ALS Association Website. alsa.org/news/media/press-releases/ approval-of-new-als-drug-050417.html. Accessed Dec. 15, 2017. 6. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. U.S. Food & Drug Administration Website. Accessed Dec. 22, 2017. 7. FDA approves new treatment to prevent bleeding in certain patients with hemophilia A. U.S. Food & Drug Administration Website. fda.gov/NewsEvents/ Newsroom/PressAnnouncements/ucm585567.htm. Accessed Dec. 18, 2017. 8. FDA approves drug to treat ALS. U.S. Food & Drug Administration Website. U.S. Food & Drug Administration Website. fda.gov/NewsEvents/ Newsroom/PressAnnouncements/ucm557102. htm?source=govdelivery&utm_medium=email&utm_ source=govdeliveryfda.gov/NewsEvents/ Newsroom/PressAnnouncements/ucm557102. htm?source=govdelivery&utm_medium=email&utm_ source=govdelivery. Accessed Dec. 18, 2017.
Diplomat Pharmacy Services | Specialty Drug Approvals
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Lead Author RYAN CHANDANAIS As an emerging therapeutics analyst, Ryan Chandanais gathers, analyzes, and presents pipeline intelligence about specialty drug products. Before joining Diplomat, he served as a research associate and report coordinator at a contract research organization for preclinical drug studies. Ryan earned a Master of Science in integrative pharmacology from Michigan State University and a Bachelor of Science in education from Central Michigan University. He is certified as a pharmacy technician by the Pharmacy Technician Certification Board and as a laboratory animal technologist by the American Association for Laboratory Animal Science. Ryan can be contacted at rchandanais@diplomat.is.
Diplomat Pharmacy Services | Specialty Drug Approvals
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