Specialty Pipeline Update D Drug Insights > March 2016 New drug information Odefsey (emtricitabine, tenofovir alafenamide, rilpivirine): The Food and Drug Administration (FDA) approved Gilead s combination HIV treatment. Odefsey is similar to Complera, but contains a new tenofovir salt. Idelvion (coagulation Factor IX [recombinant], albumin fusion protein): The FDA approved CSL Behring s Idelvion for the treatment of hemophilia B. This is a long-acting drug that can be administered once every two weeks in appropriate patients. Kovaltry (octocog alfa): Bayer received FDA approval for their hemophilia factor replacement. Kovaltry is approved for the treatment of hemophilia A in children and adults. Evomela (captisol-enabled melphalan): The FDA approved Spectrum s Evomela for two indications. The first indication is for use as a high-dose conditioning treatment prior to hematopoietic stem cell transplantation (ASCT) in patients with multiple myeloma (MM). This is the first drug to receive this indication. The second indication is for the palliative treatment of patients with MM for whom oral therapy is not appropriate. New indications Ibrance (palbociclib): Pfizer received approval from the FDA for an expanded indication of Ibrance in the treatment of metastatic breast cancer. The new indication is for use in combination with fulvestrant for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer in women with disease progression following endocrine therapy. Afinitor (everolimus): The FDA approved Novartis Afinitor for treating inoperable, locally advanced or metastatic neuroendocrine tumors of gastrointestinal or lung origin. Gazyva (obinutuzumab): Genentech received FDA approval for the use of Gazyva in combination with bendamustine followed by obinutuzumab alone for the treatment of patients with follicular lymphoma who relapsed after, or are refractory to, a rituximabcontaining regimen. Xalkori (crizotinib): The FDA approved Pfizer s Xalkori for patients with the ROS-1 gene mutation. This mutation comprises about 1 percent of U.S. patients with non small cell lung cancer. Xeljanz (tofacitinib): The FDA approved Pfizer s extended-release, once-daily formulation of Xeljanz for the treatment of rheumatoid arthritis. While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner.
Specialty Pipeline > March 2016 Page 2 March news PTC Therapeutics, Inc. announced that it received yesterday evening a Refuse to File letter from the FDA regarding PTC s New Drug Application (NDA) for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmdmd). The FDA states in the Refuse to File letter that the application was not sufficiently complete to permit a substantive review. PTC first learned of the Refuse to File decision via this letter and is reviewing its content to determine the appropriate next steps. 1 Gilead Sciences has stopped six clinical trials using its drug idelalisib (Zydelig ) in combination with other cancer drugs on account of a higher rate of serious adverse events, including death, the US Food and Drug Administration (FDA) said. The announcement follows the recent decision by European Union (EU) regulators to review idelalisib in response to an increased rate of serious adverse events such as death in three clinical trials that combined the Gilead Sciences drug with other cancer drugs. 2
Specialty Pipeline > March 2016 Page 3 Specialty new product approvals in the past twelve months Generic name Brand name Manufacturer Indication(s) Route of administration Date approved* emtricitabine/tenofovir Odefsey Gilead HIV Oral March 2016 alafenamide/rilpivirine coagulation factor IX Idelvion CSL Behring Hemophilia B March 2016 octocog alfa Kovaltry Bayer Hemophilia A March 2016 tofacitinib Xeljanz XR Pfizer Rheumatoid arthritis Oral February 2016 grazoprevir/elbasvir Zepatier Merck Hepatitis C (genotypes 1 and 4) selexipag Uptravi Actelion Pulmonary arterial hypertension von Willebrand factor, recombinant Vonvendi Baxalta von Willebrand disease sebelipase alfa Kanuma Alexion/Synageva Enzyme replacement therapy asfotase alfa Strensiq Alexion Enzyme replacement therapy Subcutaneous Factor VIII, recombinant, pegylated Adynovate Baxalta Hemophilia A Oral January 2016 Oral November 2015 November 2015 mepolizumab Nucala GlaxoSmithKline Severe eosinophilic asthma Subcutaneous November 2015 elvitegravir/cobicistat/ Genvoya Gilead HIV Oral November 2015 emtricitabine/tenofovir alafenamide Factor X concentrate Coagadex BioPorducts Laboratory Factor X deficiency October 2015 uridine triacetate Xuriden Wellstat Therapeutics Hereditary orotic aciduria Oral October 2015 Factor VIII, recombinant Nuwiq Octapharma Hemophilia A September 2015 uridine triacetate Xuriden Wellstat Therapeutics Hereditary orotic aciduria Oral September 2015 dichlorphenamide Keveyis Taro Primary hyperkalemic Oral August 2015 and hypokalemic periodic paralysis. evolocumab Repatha Amgen High cholesterol Subcutaneous August 2015 daclatasvir Daklinza BMS Hepatitis C (genotype 3) Oral July 2015 ombitasvir, paritaprevir, Technivie AbbVie Hepatitis C (genotype 4) Oral July 2015 ritonavir with ribavirin alirocumab Praluent Sanofi-Genzyme High cholesterol Subcutaneous July 2015 lumacaftor/ivacaftor Orkambi Vertex Cystic fibrosis Oral July 2015 coagulation factor IX (recombinant) Ixinity Emergent BioSolutions Hemophilia B May 2015 glatiramer Glatopa Sandoz Multiple sclerosis Subcutaneous April 2015 deferasirox Jadenu Novartis Chronic iron overload Oral April 2015 *Date indicates FDA approval date only. The product launch and marketing dates are determined by the approved product s manufacturer.
Specialty Pipeline > March 2016 Page 4 New indications for approved specialty products Generic name Brand name Manufacturer New indication Date approved* ledipasvir/sofosbuvir Harvoni Gilead For the treatment of liver transplant recipients February 2016 with genotype 1 or 4 infection without cirrhosis or with compensated cirrhosis, and for genotype 1-infected patients with decompensated cirrhosis daclatasvir Daklinza Bristol-Myers Squibb For use in combination with Sovaldi February 2016 (sofosbuvir) for the treatment of chronic hepatitis C virus (HCV) in patients co-infected with HIV and for HCV patients with advanced cirrhosis, including decompensated cirrhosis and patients with post-liver transplant recurrence of HCV genotype 1 infection onabotulinumtoxina Botox Allergan For the treatment of adults with lower limb January 2016 spasticity secukinumab Cosentyx Novartis For the treatment of adult patients with active January 2016 ankylosing spondylitis and active psoriatic arthritis ledipasvir/sofosbuvir Harvoni Gilead For use in patients with genotype 4, 5 and November 2015 6 chronic HCV infection and in patients co infected with HIV ambrisentan Letairis Gilead For use in combination with tadalafil for the October 2015 treatment of pulmonary arterial hypertension adalimumab Humira AbbVie For the treatment of moderate to severe September 2015 hidradenitis suppurativa cysteamine Procysbi Raptor Pharmaceuticals Pediatric nephropathic cystinosis in patients August 2015 2 6 years old abobotulinumtoxina Dysport Ipsen Treatment of upper limb spasticity in adults July 2015 sirolimus Rapamune Pfizer To treat lymphangioleiomyomatosis May 2015 aflibercept Eylea Regeneron To treat diabetic retinopathy April 2015 onabotulinumtoxina Botox Allergan Treatment of upper limb spasticity in adults April 2015 *Date indicates FDA approval date only. The product launch and marketing dates are determined by the approved product s manufacturer.
Specialty Pipeline > March 2016 Page 5 Oncology product approvals in the past twelve months Generic name Brand name Manufacturer Indication(s) captisol-enabled melphalan Evomela Spectrum For use as a high-dose conditioning treatment prior to hematopoietic stem cell transplantation (ASCT) in patients with multiple myeloma (MM) and for the palliative treatment of patients with MM for whom oral therapy is not appropriate Route of administration Date approved* March 2016 alectinib Alecensa Genentech Non-small cell lung cancer Oral elotuzumab Empliciti Bristol-Myers Squibb/ AbbVie Multiple myeloma bendamustine Bendeka Teva Chronic lymphocytic leukemia and indolent B-cell non-hodgkin lymphoma necitumumab Portrazza Lilly Non-small cell lung cancer November 2015 ixazomib Ninlaro Millennium Multiple myeloma Oral November 2015 Pharmaceuticals/ Takeda daratumumab Darzalex Janssen Multiple myeloma November 2015 osimertinib Tagrisso AstraZeneca Non-small cell lung cancer Oral November 2015 irinotecan liposome Onivyde Merrimack Pharmaceuticals Pancreatic cancer November 2015 trabectedin Yondelis Janssen Soft tissue sarcoma November 2015 cobimetinib Cotellic Genentech Metastatic melanoma Oral November 2015 talimogene Imlygic Amgen Metastatic melanoma Injection October 2015 laherparepvec trifluridine/tipiracil Lonsurf Otsuka Colorectal cancer Oral September 2015 gefitinib Iressa AstraZeneca Non-small cell lung cancer Oral July 2015 sonidegib Odomzo Novartis Basal cell carcinoma Oral July 2015 *Date indicates FDA approval date only. The product launch and marketing dates are determined by the approved product s manufacturer.
Specialty Pipeline > March 2016 Page 6 New indications for approved oncology drugs Generic name Brand name Manufacturer New indication Date approved* crizotinib Xalkori Pfizer For patients with the ROS-1 gene mutation March 2016 obinutuzumab Gazyva Genentech For use in combination with bendamustine followed by March 2016 obinutuzumab alone for the treatment of patients with follicular lymphoma everolimus Afinitor Novartis For treating inoperable, locally advanced or metastatic March 2016 neuroendocrine tumors palbociclib Ibrance Pfizer For use in combination with fulvestrant for the treatment March 2016 of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer nivolumab Opdivo Bristol-Myers Squibb To be used in combination with Yervoy as a treatment January 2016 for patients with BRAF V600 wild-type and BRAF V600 mutation-positive unresectable or metastatic melanoma eribulin mesylate Halaven Eisai For the second-line treatment of liposarcoma January 2016 pembrolizumab Keytruda Merck First-line treatment for unresectable or metastatic melanoma and as a treatment for patients with ipilimumab (Yervoy )-refractory melanoma nivolumab Opdivo Bristol-Myers Squibb For the treatment of BRAF V600 wild-type melanoma ipilimumab Yervoy Bristol-Myers Squibb Adjuvant therapy for patients with stage III melanoma November 2015 nivolumab Opdivo Bristol-Myers Squibb To be used in combination with Yervoy (ipilimumab) for the treatment of patients with unresectable or metastatic melanoma and for the treatment of patients with metastatic nonsquamous, non-small-cell lung cancer (NSCLC) whose disease progressed during or after platinum-based chemotherapy brentuximab vedotin Adcetris Seattle Genetics To treat patients at high risk of relapse immediately after an autologous hematopoietic stem cell transplant (HSCT) to help prevent progression of the disease October 2015 August 2015 carfilzomib Kyprolis Onyx/Amgen To be used in combination with lenalidomide and July 2015 dexamethasone for the treatment of patients with relapsed multiple myeloma who have received one to three prior lines of therapy ramucirumab Cyramza Eli Lilly For use as a second-line treatment option for metastatic colorectal cancer April 2015 *Date indicates FDA approval date only. The product launch and marketing dates are determined by the approved product s manufacturer.
Specialty Pipeline > March 2016 Page 7 Pipeline watch Generic name Brand name Manufacturer Indication(s) Route of administration ixekizumab N/A Lilly Plaque psoriasis Subcutaneous pegfilgrastim N/A Apotex Biosimilar to Neulasta filgrastim Grastofil Apotex Biosimilar to Neupogen reslizumab Cinqair Teva Severe eosinophilic asthma Subcutaneous / intravenous daclizumab high-yield process Zinbryta Biogen/AbbVie Multiple sclerosis Subcutaneous defibrotide N/A Jazz Pharmaceuticals hepatic veno-occlusive disease infliximab Remsima Celltrion/Pfizer Biosimilar to Remicade; seeking all indications emtricitabine/tenofovir alafenamide Anticipated FDA decision date* 1st half 2016 1st half 2016 1st half 2016 1Q2016 March April 2016 March 2016 April 2016 N/A Gilead HIV Oral April 2016 octreotide Mycapssa Chiasma Acromegaly Oral April 2016 recombinant factor VIII single chain, rfviii- SingleChain N/A CSL Behring Hemophilia A May 2016 eteplirsen N/A Sarepta Therapeutics Duchenne muscular dystrophy May 2016 obeticholic acid N/A Intercept Primary biliary cirrhosis Oral May 2016 etanercept N/A Sandoz Biosimilar to Enbrel Subcutaneous June 2016 rociletinib N/A Clovis Oncology/ Non-small cell lung cancer Oral June 2016 Celgene sofosbuvir/velpatasvir N/A Gilead Hepatitis C (all genotypes) Oral June 2016 venetoclax N/A Roche/AbbVie Chronic lymphocytic leukemia Oral June 2016 *Expected launch dates are predictions made by Prime Therapeutics based on industry information.
Specialty Pipeline > March 2016 Page 8 Watch list Criteria for inclusion on the Watch List include: Drug is submitted to the FDA and is anticipated to have material impact to trend and/or material impact to preferred product strategies (medical or pharmacy). Brand (generic)/ manufacturer eteplirsen/sarepta Therapeutics obeticholic acid Proposed indication/ route of administration Duchenne s muscular dystrophy (IV) Primary biliary cirrhosis (PBC); off-label use for non-alcoholic steatohepatitis (NASH) Anticipated approval (PDUFA date) Similar products (by indication) Spend* Net new impact to PMPM* pharmacy benefit 5/26/2016 None $ none $ 5/29/2016 ursodiol $ $$ (PBC); $$$$ (NASH) $ $$ (PBC); $$$$ (NASH) *Notations: $ < $0.08 per member per month (PMPM), $$ = $0.08 $0.39 PMPM, $$$ = $0.40 $2.00 PMPM, $$$$ > $2.01 PMPM Net new impact to PMPM* medical benefit None References 1 http://www.fiercebiotech.com/press-releases/ptc-receives-refuse-file-letter-fda-translarna-ataluren?utm_medium=nl&utm_source=internal&mkt_ tok=3rkmmjwwff9wsronvqjneu%2fhmjteu5z17ekkwqe0lmi%2f0er3fovrpufgji4armbmnq%2btfawtg5toziv8r7lmkm1ty9mqwxtk 2 http://www.medscape.com/viewarticle/860372?nlid=102203_3901&src=wnl_newsalrt_160314_mscpedit&uac=92335bg&impid=1022582&faf=1 0X00XXXX 2992-B1 Prime Therapeutics LLC 03/16