Diplomat 2017 Highlights & 2018 Projections

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.

we blend clinical excellence with a personal touch — for happier lives and health that lasts.

Diplomat Pharmacy Services | Specialty Drug Approvals

AT D I P L O M AT,

Diplomat Pharmacy Services | Specialty Drug Approvals

2

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.

3

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

Diplomat Pharmacy Services | Specialty Drug Approvals

4

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.

Diplomat Pharmacy Services | Specialty Drug Approvals

5

Diplomat Pharmacy Services | Specialty Drug Approvals

6

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)

7

Diplomat Pharmacy Services | Specialty Drug Approvals

2017 SPECIALTY DRUGS

8

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.

9

Diplomat Pharmacy Services | Specialty Drug Approvals

April 28

Diplomat Pharmacy Services | Specialty Drug Approvals

10

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.

11

Diplomat Pharmacy Services | Specialty Drug Approvals

Oct. 23

Diplomat Pharmacy Services | Specialty Drug Approvals

12

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

Diplomat Pharmacy Services | Specialty Drug Approvals

13

14

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

15

Diplomat Pharmacy Services | Specialty Drug Approvals

Damoctocog alfa (BAY 94-9027) Bayer

Diplomat Pharmacy Services | Specialty Drug Approvals

16

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

17

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

Diplomat Pharmacy Services | Specialty Drug Approvals

18

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

9.

10.

11.

12.

13.

14.

15.

16.

17.

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.

19

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

20

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

GREAT LAKES DISTRIBUTION CENTER DIPLOMAT HEADQUARTERS

CALL TOLL-FREE

877.977.9118

OR VISIT diplomatpharmacy.com

@diplomatrx DISCLAIMER The information herein is for educational purposes only and may not be construed as medical advice. Diplomat Pharmacy Inc. takes no responsibility for the accuracy or validity of the information herein, nor the claims or statements of any manufacturer.

Copyright Š 2018 by Diplomat Pharmacy Inc. Diplomat is a registered trademark of Diplomat Pharmacy Inc. All rights reserved. OPS-151300-1801