Specialty Drug Approvals 2015 Highlights + 2016 Projections

Specialty Drug Approvals

2015 HIGHLIGHTS + 2016 PROJECTIONS

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Proprietary information of Diplomat Pharmacy Inc. Subject to change without notice.

Copyright © 2015 by Diplomat Pharmacy Inc. All rights reserved.

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 December 31,

2014, 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 contained in this presentation concerning our industry and the markets in which we operate

is based on information from publicly available independent industry and research organizations and other third-

party sources, and management estimates. Management estimates are derived from publicly available information

released by independent industry and research analysts and 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

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could cause results to differ materially from those expressed in the estimates made by these third-party sources.



AT D I PLOM AT,

we blend clinical excellence

with a personal touch

—

for happier lives

and health that lasts.

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DRUG APPROVAL TRENDS* 1,2,3,4,5

FDA approvals have increased in recent years:

• 45 novel new drug approvals in 2015 + 9 biologics (as of Dec. 30, 2015)

• 41 novel new drug approvals in 2014 + 19 biologics

• 25 novel new drug approvals per year on average from 2005 to 2013

Of the 54 new drug and biologic approvals in 2015:

• Approximately half are considered specialty pharmacy–dispensed products

• Many new drugs received special FDA designations, such as breakthrough,

fast track, orphan, accelerated approval and priority review, on their way to

approval

Disease states with the greatest number of approvals in 2015:

• Rare diseases (25)

• Oncology (13)

*Numbers include novel drug approvals only. Expanded indications for previously approved drugs, new formulations, generics, biosimilars, and intravenously administered oncology products are excluded.

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IN 2015, the FDA tied the record for the most

novel new drug approvals in a year since the

creation of the Prescription Drug User Fee Act

(PDUFA). Special FDA designations—such as

breakthrough, fast track, orphan, accelerated

approval and priority review—apply to promising

drugs or those that treat diseases with an

unmet medical need. These designations have

reduced the time needed for clinical trials and/

or reduced the FDA review timeline, allowing

pipeline products to come to market more

quickly than in the past.6 Once again, specialty

drug products accounted for a sizable portion

(approximately half) of the total new drugs and

biologics approved by the FDA this year. Similar

to 2014, rare diseases and oncology saw the

most newly approved drugs in the specialty

pharmacy market. In 2015, 25 rare disease and

13 oncology products were approved, with

some drugs counting as both rare disease and

oncology approvals. 2,3, 4

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RARE DISEASES8

In the past, rare diseases were sometimes

viewed by manufacturers as less attractive

targets for product development. Due to

low patient populations, it was perhaps

understandable to think the utilization of a new

orphan drug product would be low and therefore

profits would be low as well. That thinking has

changed in recent times; treating rare diseases is

now often considered an appealing opportunity.

Orphan diseases often have little to no treatment

competition for a particular condition. These

products are typically high-cost, allowing for

potentially high earnings even if there are few

patients who may benefit. Manufacturers earn a

period of market exclusivity after FDA approval

is granted for a rare disease product, receive

certain tax incentives and may have access to

certain funding for drug development.5

Additionally, manufacturers developing an orphan

product that treats either a pediatric disease or a

neglected tropical disease have the opportunity

to earn a priority review voucher. The voucher

is redeemable with the FDA for a future priority

review for any pipeline product the manufacturer

requests, and the owner also has the option to

sell it. These vouchers have become valuable

assets. In fact, one voucher was sold for $350

million over the summer.7 The number of pipeline

orphan drug products has greatly increased in

recent years, with a high of 293 agents gaining

orphan designation in 2014 (see next page).

Prior to 2003, there were never more than

95 products earning orphan designation in a

particular year.8

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ONCOLOGY9

Oncology continues to have an active pipeline.

Key approvals in the oncology field for 2015

include Ibrance® (palbociclib) for estrogen

receptor (ER)–positive, human epidermal growth

factor receptor 2 (HER2)–negative breast

cancer; Tagrisso™ (osimertinib) for non–small

cell lung cancer in patients with T790 genetic

mutation; Farydak® (panobinostat) and Ninlaro®

(ixazomib), each for multiple myeloma, and

Lonsurf® (trifluridine and tipiracil) for colorectal

cancer. Also worth noting, Zarxio™ (filgrastim-

sndz) became the first biosimilar approved in the

U.S. It is a biosimilar of Neupogen® (filgrastim).

Zarxio™ is approved for all except one of

the same indications as Neupogen®, but the

FDA does not allow the biosimilar to be used

interchangeably with the name-brand product.

YEARNumber of Orphan Designations Granted

by the FDA8

2010 195

2011 203

2012 190

2013 260

2014 293

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HEPATITIS C9

Chronic hepatitis C (CHC), perhaps more than

any other disease state, has seen the treatment

landscape change dramatically over the last two

to three years. In 2015, we saw some additional

approvals and expanded indications to treat

some of the less common forms of the disease

in the United States. Daklinza™ (daclatasvir)

was approved for genotype 3 and Technivie™

(ombitasvir + paritaprevir + ritonavir) earned

an indication for treating genotype 4. Harvoni®

(ledipasvir + sofosbuvir) gained expanded

indications, covering HIV/CHC coinfection in

patients with CHC genotypes 1, 4, 5 and 6;

genotype 1 treatment-naïve cirrhotics; and CHC

genotypes 4, 5 and 6 regardless of treatment

experience or cirrhosis status.

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IMMUNOLOGY AND MULTIPLE SCLEROSIS9

In early 2015, we saw the arrival of Cosentyx®

(secukinumab) for psoriasis. It became the first

drug targeting interleukin (IL)-17A to earn FDA

approval. Other agents with this mechanism of

action are currently in the late-stage pipeline

as well. Also of note was the approval of

Glatopa™ (glatiramer acetate), the first generic

of Copaxone®. Glatiramer acetate is a complex

molecule that proved to be more difficult for

generic manufacturers to develop than the

average small-molecule drug, but it is not a

biosimilar.

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2015 SPECIALTY APPROVALS 2,3

Approval Date

Drug Indication RouteApproximate Population (U.S.)

Q1 2015

1/21/15Cosentyx®

(secukinumab)Psoriasis SubQ 7.5 million

1/23/15Natpara® (parathyroid hormone)

Hypoparathyroidism SubQ 60,000

1/29/15Imbruvica®* (ibrutinib)

Waldenstrom’s macroglobulinemia

Oral1,000 to 1,500 diagnoses/year

2/3/15Ibrance® (palbociclib)

Breast cancer (ER+, HER2-) Oral 1 million

2/13/15Lenvima® (lenvatinib)

Thyroid cancer Oral 600,000

2/18/15Revlimid®* (lenalidomide)

Newly diagnosed multiple myeloma

Oral 60,000

2/23/15Farydak®

(panobinostat)Multiple myeloma Oral 90,000

3/6/15Zarxio™ (filgrastim-sndz)

Biosimilar Neupogen® for neutropenia

SubQ and IV

60,000 diagnoses per year

3/17/15Cholbam™ (cholic acid)

Bile acid disorders Oral <10 in 1 million

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Q2 2015

4/16/15Glatopa™ (glatiramer acetate)

Multiple sclerosis SubQ 400,000

4/29/15Ixinity® (recombinant factor IX)

Hemophilia B IV 3,400

Q3 2015

7/2/15Orkambi™ (lumacaftor + ivacaftor)

Cystic fibrosis Oral 25,000

7/10/15Envarsus® XR (tacrolimus extended-release tablets)

Kidney transplant rejection Oral <200,000

7/11/15Rapamune®* (sirolimus)

Lymphangioleiomyomatosis Oral30,000 to 50,000 worldwide

7/13/15Iressa® (gefitinib)

Non–small cell lung cancer Oral 400,000 all NSCLC

7/24/15Praluent® (alirocumab)

Hypercholesterolemia SubQ 625,000

7/24/15Odomzo® (sonidegib)

Basal cell carcinoma Oral2.8 million diagnoses/year

*Expanded indication

[Does not include IV or other health care provider–administered oncology products]

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7/24/15Daklinza™ (daclatasvir)

Hepatitis C, genotype 3 Oral10% of HCV population in the US

7/24/15

Technivie™ (ombitasvir + paritaprevir + ritonavir)

Hepatitis C, genotype 4 Oral1% of HCV population in the US

8/7/15Keveyis™ (dichlorphenamide)

Periodic paralysis Oral 5000

8/27/15Repatha™

(evolocumab)Hypercholesterolemia SubQ 625,000

9/4/15Xuriden™ (uridine triacetate)

Hereditary orotic aciduria Oral 20 world wide

9/11/15Humira®* (adalimumab)

Hidradenitis suppurativa SubQ 155,000

9/15/15Nuwiq® (simoctocog alfa)

Hemophilia A IV 16,500

9/22/15Lonsurf® (trifluridine + tipiracil)

Colorectal cancer Oral 1.1 million

Q4 2015

10/20/15Coagadex® (coagulation factor X)

Hereditary factor X deficiency IV 300-600

10/23/15Strensiq™ (asfotase alfa)

Hypophosphatasia SubQ 1 in 100,000

11/4/15Nucala® (mepolizumab)

Eosinophilic asthma SubQ

Not well understood; 10–20% of all asthma patients have poorly controlled symptoms

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*Expanded indication

[Does not include IV or other health care provider–administered oncology products]

11/5/15

Genvoya® (elvitegravir + cobicistat + emtricitabine + tenofovir alafenamide)

HIV Oral 1.1 million

11/10/15Cotellic™

(cobimetinib)Melanoma (in combination with Zelboraf)

Oral 920,000

11/12/15Harvoni®* (ledipasvir + sofosbuvir)

HIV/HCV coinfection with genotypes 1, 4, 5 and 6; genotype 1 treatment naïve cirrhotics; and HCV genotypes 4, 5 and 6 regardless of treatment experience or cirrhosis status

Oral

Genotypes 4, 5 and 6: 3% combined of the HCV population in the U.S. 25% of HIV patients are coinfected with HCV

11/13/15Tagrisso™ (osimertinib)

Non–small cell lung cancer Oral3,000 with this particular genetic mutation (T790)

11/13/15Adynovate® (Factor VIII)

Hemophilia A IV 16,500

11/20/15Ninlaro® (ixazomib)

Multiple myeloma Oral 60,000

12/8/15Vonvendi™ (von Willebrand factor)

Von Willebrand disease IVUp to 1 in 100, no definitive diagnostic test

12/8/15Kanuma™ (sebelipase alfa)

Lysosomal acid lipase deficiency IV 1 in 525,000

12/11/15Alecensa® (alectinib)

Non–small cell lung cancer Oral 400,000 all NSCLC

12/22/15Uptravi® (selexipag)

Pulmonary arterial hypertension Oral15–50 cases in 1 million

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IN THE PIPELINE

The specialty drug pipeline continues to be

strong, with a variety of novel products for new

indications in development. Some trends are

expected to continue into 2016 and beyond,

with several approvals expected in the areas

of rare diseases and oncology. Biosimilars will

continue to be in the news as manufacturers

try to bring them to the market. However, brand

name manufacturers are expected to continue to

fight the launch of biosimilar products in court.

Some potentially high-volume biosimilars, for

products such as adalimumab (Humira®) and

etanercept (Enbrel®), have already filed with the

FDA for review. The courts will be busy with legal

arguments from both sides regarding biosimilars.

The cost of drugs—and the sustainability of

payors and patients to cover their expenses—is

expected to continue to be a major issue going

forward. Discussions about what can be done to

improve the health care system in terms of drug

access and cost are sure to continue in 2016

and for a long time thereafter.

In terms of specific drug approvals anticipated in

2016, particularly interesting products include:

• Obeticholic acid for primary biliary cirrhosis

(and later, non-alcoholic steatohepatitis)9

• Alectinib and brigatinib for non–small cell lung

cancer in patients with ALK genetic mutation9

• Velpatasvir, a new hepatitis C agent paired

with Sovaldi® (sofosbuvir) that has the

potential to treat all genotypes9

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2016 Expected Specialty Approvals9

Expected FDA

Decision DateDrug Target Indication Route

Population

(U.S.)

Q1 2016

Early Q1 2016 DrisapersenDuchenne muscular dystrophy

SubQ 1–2 in 10,000

Early 2016 N9-GP/NN-7999 Hemophilia B IV 3,400

Early 2016 Daclizumab Multiple sclerosis SubQ 400,000

Possibly Q1 2016Remune™

(HIV-1 immunogen)HIV Injection 1.1 million

Possibly Q1 2016Kalydeco®*

(ivacaftor)

Cystic fibrosis additionalgene mutation (Non-G551D gating)

Oral

25,000 entire CFpopulation; Non-G551D mutationabout 2% of this

Possibly Q1 2016Factor IX +

recombinant albuminHemophilia B IV 3,400

Possibly Q1 2016Epoetin alfa

biosimilarAnemia SubQ and IV

35–40% of cancer patients

1/28/16Grazoprevir +

elbasvirHepatitis C, genotypes 1, 4 and 6

OralGenotype 1: 70%;Genotypes 4 and 6: 2% combined

2/26/16 EteplirsenDuchenne muscular dystrophy

IV1–2 in 10,000 males

3/15/16Imbruvica®*

(ibrutinib)First-line treatment for CLL

Oral16,000 new diagnoses yearly

3/31/16 Reslizumab Eosinophilic asthma IV

Not well understood; 10–20% of all asthma patients have poorly controlled symptoms

3/31/16 DefibrotideVeno-occlusive disease of the liver

Oral 1–5 in 10,000

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*Expanded indication [Does not include IV or other health care provider–administered oncology products]

Q2 2016

4/6/16

Daklinza®

(daclatasvir)

+ Sovaldi®*

(sofosbuvir)

HCV/HIV coinfection, HCV with advanced cirrhosis, and post-transplant HCV recurrence

Oral

25% of HIV patients are coinfected with HCV; post-transplant recurrence: 1/3 of transplant patients

4/15/16Mycapssa™

(octreotide)Acromegaly Oral 20,000

4/15/16Xalkori®*

(crizotinib)

Non–small cell lung cancer with ROS1 mutation

Oral1% of NSCLC patients have ROS1 mutation

4/30/16 Ixekizumab Psoriasis SubQ 7.5 million

5/29/16 Obeticholic acid Primary biliary cirrhosis Oral 40 in 100,000

6/25/16Gilotrif®*

(afatinib)Lung squamous cell carcinoma

Oral25–30% of lung cancers

6/28/16 RociletinibNon–small cell lung cancer with T790 mutation

Oral3,000 with this particular genetic mutation (T790)

Possibly Q2 2016

Firdapse®

(amiphampridine

phosphate)

Lambert-Eaton myasthenic syndrome

Oral 2–3 in 1 million

Possibly Q2 2016 BrigatinibNon–small cell lung cancer

Oral400,000 (all NSCLC)

Possibly Q2 2016

Etanercept

(Enbrel® biosimilar

made by Sandoz)

Same as Enbrel® SubQ Same as Enbrel®

Possibly Q2 2016 Deutetrabenazine Huntington’s disease Oral 30,000

Possibly Q2 2016 Cabozantinib* Renal cell carcinoma Oral17,000 for second line or later RCC

Possibly Q2 2016Velpatasvir +

Sovaldi®Hepatitis C, pan-genotypic

Oral 4.4 million

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MIKE | DIPLOMAT PHARMACIST

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192015 Key

Approvals

Cosentyx®

Ibrance®

Praluent®

Repatha™

Tagrisso™

20

22

24

26

28



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Cosentyx® (secukinumab)10 Psoriasis

INDICATION

Treatment of adult patients with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy.

CLINICAL TRIAL BRIEFING

Secukinumab was evaluated for the treatment of psoriasis in four multicenter, randomized, double-blind, placebo-controlled trials composed of a total of 2403 patients. Patients enrolled in the studies received either 150 or 300 mg of secukinumab, placebo, or in certain studies, a biologic active control. Dosing was completed on weeks zero, one, two, three and four, and every four weeks thereafter. For certain studies, patients receiving placebo who were non-responders were crossed over and received secukinumab weekly during weeks 12 to 16, and then every four weeks thereafter. Two studies were 12 weeks in duration and two were 52 weeks in length. For all studies, the primary endpoint was the proportion of patients achieving a ≥75 percent reduction in Psoriasis Area Severity Index (PASI) at week 12 compared to baseline and a rating of clear or almost clear on the Investigator’s Global Assessment (IGA). Secondary endpoints included proportion of patients achieving ≥90 percent PASI at week 12 compared to baseline, maintenance of efficacy at week 52, and improvement in itching, pain and scaling at week 12 based on Psoriasis Symptom Diary. Across all four trials, PASI 75 ranged from, 67–71 percent for patients given 150 mg of secukinumab, 75–87 percent for patients given 300 mg of secukinumab, and 0–5 percent for patients given placebo. IGA of clear or almost clear was achieved by 51–53 percent of patients given 150 mg of secukinumab, 62–73 percent of patients given 300 mg of secukinumab, and 0–3 percent of patients given placebo.

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APPROVAL DATE: Jan. 21, 2015MANUFACTURER: Novartis PharmaceuticalsCLASS: Interleukin-17A antagonistSTORAGE: 2°C to 8°C (36°F to 46°F); protect from lightHOW SUPPLIED: 150 mg/mL solution in a prefilled pen or syringe or a single-use vial containing 150 mg of lyophilized powder for reconstitution

DOSING

Administer 300 mg by subcutaneous injection at weeks zero, one, two, three and four, then every four weeks thereafter.

SAFETY

Common Adverse Events: Nasopharyngitis, diarrhea and upper respiratory tract infection Serious Adverse Events: Infections, tuberculosis, Crohn’s disease exacerbations and hypersensitivity reactions

OTHER AGENTS IN THERAPEUTIC AREA

• Cimzia® (certolizumab pegol)

• Enbrel® (etanercept)

• Humira® (adalimumab )

• Otezla® (apremilast)

• Remicade® (infliximab)

• Simponi® (golimumab)

• Stelara® (ustekinumab)

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Ibrance® (palbociclib)11

Oncology

INDICATION

Treatment of postmenopausal women with estrogen receptor (ER)–positive, human epidermal growth factor receptor 2 (HER2)–negative advanced metastatic breast cancer. Palbociclib is used in combination with letrozole.


Palbociclib was evaluated for the treatment of breast cancer in a randomized, open-label, multicenter study composed of 165 patients with ER-positive, HER2 negative advanced breast cancer who had not previously received systemic treatment. Patients on study received palbociclib plus letrozole or letrozole alone. Palbociclib was administered daily for 21 consecutive days, followed by seven days off. Dosing continued with this cycle until disease progression, unmanageable toxicity or consent withdrawal occurred. The primary endpoint of the study was progression-free survival (PFS) as measured by the Response Evaluation Criteria in Solid Tumors (RECIST). Additionally, overall response rate (ORR) was determined. Median PFS was 20.2 months for the palbociclib-plus-letrozole group (95 percent CI: 13.8, 27.5) and 10.2 months for the letrozole-only group (95 percent CI: 5.7, 12.6). ORR was 55.4 percent for the palbociclib-plus-letrozole group and 39.4 percent for the letrozole-only group. Overall survival (OS) was not available at the time of analysis. Approval was granted under the FDA accelerated approval program.

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APPROVAL DATE: Feb. 3, 2015MANUFACTURER: Pfizer Inc.CLASS: Kinase inhibitorSTORAGE: 20°C to 25°C (68°F to 77°F); excursions permitted from 15°C to 30°C (59°F to 86°F)HOW SUPPLIED: 75, 100, and 125 mg capsules

DOSING

Administer 125 mg of palbociclib orally once daily with food for 21 consecutive days followed by seven days off for repeated 28-day cycles. Take in combination with 2.5 mg of letrozole dosed once daily continuously for 28 days during the cycles. Doses of palbociclib may be reduced if adverse events occur. See prescribing information for details.

SAFETY

Common Adverse Events: Neutropenia, leukopenia, fatigue, anemia, upper respiratory infection, nausea, stomatitis, alopecia, diarrhea, thrombocytopenia, decreased appetite, vomiting, asthenia, peripheral neuropathy and epistaxis

Serious Adverse Events: Neutropenia and infections


• Afinitor® (everolimus)

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Praluent® (alirocumab)12

Hypercholesterolemia

INDICATION

Treatment as an adjunct to diet and maximally tolerated statin therapy for adult patients with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease, who require additional lowering of low-density lipoprotein cholesterol (LDL-C).


Alirocumab was evaluated in five multicenter, double-blind, placebo-controlled trials composed of 3,499 total patients. Patients on study had HeFH and/or clinical atherosclerotic cardiovascular disease and were receiving a maximally tolerated dose of a statin. Some patients were also receiving other lipid-modifying therapies. For three studies, the initial dose was 75 mg given every two weeks. If insufficient response in LDL-C was observed at week eight, dosing was increased to 150 mg every two weeks for week 12 and beyond. In the other two studies, only the 150 mg dose was administered. Selected patients received placebo in all studies. All studies were 52 weeks or greater in duration. The primary endpoint in all studies was mean percent change from baseline in LDL-C at week 24. Across the five trials, the mean treatment differences in LDL-C between alirocumab and placebo ranged from -36 percent (95 percent CI: -49, -24; p<0.0001) to -58 percent (95 percent CI: -61, -56; p<0.0001). Additional analyses related to the reduction of cholesterol and apolipoprotein were also performed.

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APPROVAL DATE: July 24, 2015MANUFACTURER: Sanofi-Aventis and RegeneronCLASS: PCSK9 inhibitorSTORAGE: 36°F to 46°F (2°C to 8°C)HOW SUPPLIED: 75 and 150 mg/mL pens and syringes PATIENT POPULATION: HeFH - 1 in 500; clinical atherosclerotic cardiovascular disease, over 25 million patients in the U.S.

DOSING

Administer subcutaneously once every two weeks. The recommended starting dose is 75 mg. Dosing may be increased to a maximum of 150 mg if LDL-C response is insufficient at the initial dose level.

SAFETY

Common Adverse Events: Nasopharyngitis, injection site reactions and influenza

Serious Adverse Events: Hypersensitivity reactions


• Repatha™ (evolocumab)

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Repatha™ (evolocumab)13

Hypercholesterolemia

INDICATION

Treatment as an adjunct to diet and maximally tolerated statin therapy for adult patients with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (CVD) who require additional lowering of low-density lipoprotein cholesterol (LDL-C). Additionally indicated for homozygous familial hypercholesterolemia (HoFH) when used in combination with other LDL-lowering therapies.


Evolocumab was evaluated in four multicenter, double-blind, randomized, placebo-controlled trials composed of 813 total patients. Patients on study had hypercholesterolemia, including CVD with primary hyperlipidemia, HeFH or HoFH. Patients received statins and/or other lipid-modifying therapies in addition to evolocumab or placebo. Evolocumab was given 140 mg every two weeks or 420 mg monthly for 12 weeks in three of the trials and 52 weeks in the other trial. The primary endpoint of the studies was mean difference from placebo in LDL-C. Across the four trials, the mean treatment differences in LDL-C between evolocumab and placebo ranged from -31 percent (95 percent CI: -44, -18) to -71 percent (95 percent CI: -81, -61). Additional analyses related to the reduction of cholesterol and apolipoprotein were also performed.

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APPROVAL DATE: Aug. 27, 2015MANUFACTURER: AmgenCLASS: PCSK9 inhibitorSTORAGE: 36°F to 46°F (2°C to 8°C). May be stored at room temperature for up to 30 days. HOW SUPPLIED: 140 mg/mL prefilled pens and syringes PATIENT POPULATION: HeFH: 1 in 500; HoFH: 1 in 1 million; clinical atherosclerotic cardiovascular disease: over 25 million in the U.S.

DOSING

Administer by subcutaneous injection.

For primary hyperlipidemia with CVD or HeFH: 140 mg every two weeks or once monthly, given as three consecutive 140 mg injections.

For HoFH: 420 mg once monthly.

SAFETY

Common Adverse Events: Nasopharyngitis, upper respiratory tract infection, influenza, back pain and injection site reactions

Serious Adverse Events: Allergic reactions


• Praluent® (alirocumab)

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Tagrisso™ (osimertinib)14 Oncology

INDICATION

Treatment of metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non–small cell lung cancer (NSCLC) in patients who have progressed on or after EGFR TKI therapy.


Osimertinib was evaluated for the treatment of T790M mutation-positive NSCLC in two multicenter, single-arm, open-label trials composed of 411 total patients. Patients on study had progressed on a prior EGFR TKI treatment and received 80 mg of osimertinib once daily. The primary endpoint of both studies was objective response rate (ORR) according to RECIST criteria as evaluated by a blinded independent central review. Additionally, duration of response (DUR) was measured. The ORR for the combined studies was 59 percent (95 percent CI: 54, 64). Fifty-nine percent of responses were classified as partial and 0.5 percent were complete responses. DUR ranged from 1.1 to 5.6 months after a median duration of follow-up for 4.0 months in one study and 4.2 months in the other.

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APPROVAL DATE: Nov. 13, 2015MANUFACTURER: AstraZenecaCLASS: Kinase inhibitorSTORAGE: 25°C (77°F) with excursions permitted from 15°C to 30°C (59°F to 86°F)HOW SUPPLIED: 40 and 80 mg tablets PATIENT POPULATION: Approximately 60 percent of patients treated with an EGFR inhibitor develop T790M mutation

DOSING

Administer 80 mg orally once daily, with or without food.

SAFETY

Common Adverse Events: Diarrhea, rash, dry skin and nail toxicity

Serious Adverse Events: Interstitial lung disease/pneumonitis, QTc interval prolongation, cardiomyopathy and embryo-fetal toxicity


• Gilotrif® (afatinib)

• Iressa® (gefitinib)

• Tarceva® (erlotinib)

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About the Author

RYAN CHANDANAIS, LEAD AUTHOR Ryan Chandanais is an emerging therapeutics analyst at Diplomat

in Flint, Michigan. His job is to gather, analyze and present pipeline

intelligence involving specialty drug products. He has additional

drug development–related experience at a contract research

organization for pre-clinical drug studies, where he served as

a research associate and report coordinator. He has earned a

Master of Science in Integrative Pharmacology from Michigan

State University and a Bachelor of Science in Education from

Central Michigan University. He has acquired certifications as

a pharmacy technician (CPhT) from the Pharmacy Technician

Certification Board, and as a laboratory animal technologist

(LATG) from the American Association for Laboratory Animal

Science. He can be contacted at [email protected].



REFERENCES:1. U.S. Food and Drug Administration Center for Drug Evaluation and Research. “Novel New Drugs 2014 Summary” January

2015.2. U.S. Food and Drug Administration Website. “New Molecular Entity and New Therapeutic Biological Product Approvals

for 2015” http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm430302.htm (accessed December 8, 2015).

3. U.S. Food and Drug Administration Website. “2015 Biological License Application Approvals” http://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/ucm434961.htm (accessed December 8, 2015).

4. U.S. Food and Drug Administration Website. “Search Orphan Drug Designations and Approvals” http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm?Index_Number=309410 (accessed December 9, 2015).

5. Fast track, accelerated approval and priority review. Food and Drug Administration Website. http://www.fda.gov/forpatients/approvals/fast/ucm20041766.htm (accessed December 8, 2015).

6. Developing products for rare disease & conditions. U.S. Food and Drug Administration (FDA) Website. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm (accessed December 1, 2015).

7. Priorityreviewvoucher.org. http://priorityreviewvoucher.org (accessed December 8, 2015).8. FDA Law Blog – Hyman, Phelps, and McNamara, P.C. http://www.fdalawblog.net/fda_law_blog_hyman_phelps/orphan-

drugs (accessed December 4, 2015).9. BioPharm Insight. Boston, MA: Infinata; 2013. http://www.infinata.com/biopharma-solution/by-product/biopharm-insight.

html (accessed December 2015). http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm (accessed December 1, 2015).

10. Cosentyx® [package insert]. East Hanover, NJ: Novartis; 2015.11. Ibrance® [package insert]. New York, NY: Pfizer; 2015. 12. Praluent® [package insert]. Bridgewater, NJ: Sanofi-Aventis U.S.; 2015.13. Repatha™ [package insert]. Thousand Oaks, CA: Amgen; 2015.14. Tagrisso™ [package insert]. Wilmington, DE: AstraZeneca; 2015.

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Specialty Drug Approvals 2015 Highlights + 2016 Projections