Specialty Drug Approvals
2015 HIGHLIGHTS + 2016 PROJECTIONS
cl inical services
Proprietary information of Diplomat Pharmacy Inc. Subject to change without notice.
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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
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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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
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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.
CLINICAL TRIAL BRIEFING
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
OTHER AGENTS IN THERAPEUTIC AREA
• 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).
CLINICAL TRIAL BRIEFING
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
OTHER AGENTS IN THERAPEUTIC AREA
• 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.
CLINICAL TRIAL BRIEFING
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
OTHER AGENTS IN THERAPEUTIC AREA
• 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.
CLINICAL TRIAL BRIEFING
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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Copyright © 2015 by Diplomat Pharmacy Inc. All rights reserved.
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
OTHER AGENTS IN THERAPEUTIC AREA
• 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].
Proprietary information of Diplomat Pharmacy Inc. Subject to change without notice.
Copyright © 2015 by Diplomat Pharmacy Inc. All rights reserved.
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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