Specialty Pipeline Monthly Update

Transcription

1 Specialty Pipeline Monthly Update Critical updates in an ever changing environment September 2017 New drug information Gocovri (amantadine HCl XR): The U.S. Food and Drug Administration (FDA) approved Adamas Pharma s Gocovri for the treatment of dyskinesia in patients with Parkinson s disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications. Gocovri is the first medication approved by the FDA for this indication; however, immediate-release amantadine, available generically, is used for this indication off-label. 1 The company has reported that the annual wholesale acquisition cost (WAC) should fall between $10,000 to $30,000 and will be launched in the fourth quarter of ,2 Cyltezo (adalimumab-adbm): Boehringer Ingelheim s Cyltezo, a biosimilar for AbbVie s Humira, received FDA approval for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn s disease, ulcerative colitis, and plaque psoriasis. Humira has additional indications that Cyltezo does not have including: pediatric Crohn s disease, hidradenitis suppurativa, and uveitis. Cyltezo is the second Humira biosimilar to win FDA approval, following Amgen s Amjevita which received approval in September Neither Cyltezo nor Amjevita are able to launch at this time due to ongoing patent litigation. Kymriah (tisagenlecleucel): The FDA approved Novartis Kymriah for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Kymriah is a CD19-directed genetically modified autologous T cell immunotherapy, also known as chimeric antigen receptor (CAR) T cell therapy. Novartis has priced Kymriah at a WAC of $475,000 for a course of treatment. 3 Kymriah is available for administration at limited treatment centers throughout the United States; some treatment centers are ready to administer Kymriah, while others will be ready soon. 4 Mylotarg (gemtuzumab ozogamicin): The FDA approved Pfizer s Mylotarg for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) whose tumors express the CD33 antigen and for the treatment of patients aged 2 years and older with CD33-positive AML who have experienced a relapse or who have not responded to initial treatment. Although Mylotarg originally received accelerated approval in May 2000, it was voluntarily withdrawn from the market after trials failed to verify clinical benefit and demonstrated safety concerns. 5 The current approval includes a lower recommended dose, a different schedule in combination with chemotherapy or on its own, and a new patient population. 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.

2 Specialty Pipeline Update: September 2017 Page 2 New drug information continued Mvasi (bevacizumab-awwb): Allergan/Amgen received FDA approval for Mvasi, a biosimilar to Genentech s Avastin, for the treatment of metastatic colorectal cancer, non-squamous non-small cell lung cancer (NSCLC), glioblastoma, renal cell carcinoma, and cervical cancer. Avastin has additional indications that Mvasi does not have including: recurrent epithelial ovarian, fallopian tube, and primary peritoneal cancer. Mvasi is the first biosimilar approved in the United States for the treatment of cancer. Mvasi is unable to launch at this time due to Avastin s composition-of-matter patents which are set to expire in mid Aliqopa (copanlisib): The FDA approved Bayer s Aliqopa for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies. Accelerated approval was granted for this indication based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Aliqopa is administered intravenously on an intermittent schedule. 6 New indications Yervoy (ipilimumab): The FDA expanded the indication of BMS s Yervoy for the treatment of unresectable or metastatic melanoma in pediatric patients 12 years of age and older. Prior to this approval, Yervoy was approved for the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm who have undergone complete resection, including total lymphadenectomy. Austedo (deutetrabenazine): Teva pharmaceuticals received FDA approval for Austedo to treat tardive dyskinesia. Prior to this approval, Austedo was only approved for the treatment of chorea associated with Huntington s disease. Actemra (tocilizumab): The FDA approved Genentech s Actemra for adults and pediatric patients 2 years of age and older with chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS). CRS has been identified as a potentially severe and life-threatening side effect of CAR T cell therapy. Orfadin (nitisinone): Sobi received FDA approval for once daily dosing of Orfadin for patients with hereditary tyrosinemia type 1 (HT-1) ages 5 years and older. Previously, Orfadin was only approved for twice daily dosing. Tracleer (bosentan): The FDA approved Johnson & Johnson s Tracleer for pediatric patients at least 3 years of age and older with idiopathic or congenital pulmonary arterial hypertension (PAH). This is the first approved drug for children with PAH and will be available in a scored 32 mg tablet that allows weight-based dose adjustment for younger patients and may be dispersed in water before being given orally.

3 Specialty Pipeline Update: September 2017 Page 3 September news The Food and Drug Administration accused the drugmaker Pfizer of failing to properly investigate reports of malfunctioning EpiPens, including incidents when patients died or became severely ill after the device failed to work. Pfizer manufactures the EpiPen, which treats allergic reactions, for the drugmaker Mylan. 7 Sarepta Therapeutics Inc. on Wednesday released promising early data from a study of a new drug to treat some patients with the rare muscle-wasting disease Duchenne Muscular Dystrophy (DMD). The drug is a follow-up to Exondys 51, another DMD treatment the FDA approved last year over the objection of some of its own scientists to treat a different subset of patients. 8 What does it really cost to bring a drug to market? The question is central to the debate over rising health care costs and appropriate drug pricing. For years, the standard figure has been supplied by researchers at the Tufts Center for the Study of Drug Development: $2.7 billion each, in 2017 dollars. Yet a new study looking at 10 cancer medications, among the most expensive of new drugs, has arrived at a much lower figure: a median cost of $757 million per drug. 9 Dupixent, the Sanofi and Regeneron drug already on a blockbuster arc in atopic dermatitis, has returned significant results in patients with severe asthma, another indication that is expected to add billions of dollars more in sales for the partners. 10 FDA commissioner Scott Gottlieb has sounded a crystal clear warning over the high and growing cost of drug development. And in a speech to regulatory execs on Monday, Gottlieb committed the FDA to backing up more efficient drug development programs with new measures to clear the regulatory path for developers barreling ahead to relatively swift pivotal data in search of an accelerated OK. 11 For the first time a medicine (Ilaris ), given just four times a year, has been shown to prevent heart attacks not by lowering cholesterol or blood pressure or preventing blood clots but by reducing inflammation. Tantalizingly, it also may have prevented deaths of non-small cell lung cancer, although everyone cautions that a lot more evidence is needed to know if that result is a fluke. 12

4 Specialty Pipeline Update: September 2017 Page 4 Specialty new product approvals in the past twelve months Generic name Brand name Manufacturer Indication(s) adalimumab-adbm Cyltezo BI RA, juvenile idiopathic arthritis, [Biosimilar to psoriatic arthritis, ankylosing Humira ] spondylitis, adult Crohn s disease, UC, and plaque psoriasis amantadine extended release Gocovri Adamas Pharma Levodopa-induced dyskinesia in Parkinson s dyskinesia glecaprevbir/pibrentasvir Mavyret AbbVie Treatment naïve and experienced HCV nitisinone Nityr Cycle sofosbuvir, velpatasivir, voxilaprevir Hereditary Tyrosinemia type-1 (HT-1) Route of administration Month approved Subcutaneous August 2017 Oral August 2017 Oral August 2017 Oral August 2017 Vosevi Gilead Sciences Previously treated HCV Oral July 2017 guselkumab Tremfya Johnson & Johnson Plaque Psoriasis Subcutaneous July 2017 L-glutamine oral powder Endari Emmaus Life Sickle cell disease Oral July 2017 Sciences fibrinogen Fibryna Octapharma Acute bleeds in congenital Intravenous July 2017 fibrinogen deficiency C1-esterase inhibitor Haegarda CSL Behring Prevention of hereditary Subcutaneous July 2017 angioedema (HAE) coagulation Factor IX Rebinyn Novo Nordisk Hemophilia B Intravenous June 2017 (recombinant), GlycoPEGylated sarilumab Kevazara Sanofi/Regeneron Rheumatoid arthritis Subcutaneous May 2017 edaravone Radicava Mitsubishi Tanabe Pharma Amyotrophic lateral sclerosis (ALS) Intravenous May 2017 abaloparatide Tymlos Radius Health Osteoporosis in Subcutaneous April 2017 postmenopausal women cerliponase alfa Brineura BioMarin CLN2 disease Intracerebroventricular infusion April 2017 infliximab-abda Renflexis (Biosimilar to Remicade ) Samsung Bioepis/ Merck valbenazine Ingrezza Neurocrine Biosciences Inc Rheumatoid arthritis, Crohn's disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis and psoriasis. Intravenous April 2017 Tardive dyskinesia Oral April 2017 deutetrabenazine Austedo Teva Chorea associated with Oral April 2017 Huntington disease ocrelizumab Ocrevus Genetech Multiple sclerosis Intravenous March 2017 dupilumab Dupixent Sanofi/Regeneron Atopic dermatitis Subcutaneous March 2017 telotristat etiprate Xermelo Lexicon Carcinoid syndrome Oral February 2017 brodalumab Siliq Valeant Plaque Psoriasis Subcutaneous February 2017 immune globulin Gammaplex 10% Bio Products Primary immunodeficiency/ chronic immune thrombocytopenic purpura Intravenous February 2017

5 Specialty Pipeline Update: September 2017 Page 5 Specialty new product approvals in the past twelve months continued Generic name Brand name Manufacturer Indication(s) deflazacort Emflaza Marathon Duchenne Muscular Dystrophy (DMD) Route of administration Month approved Oral February 2017 etelcacetide Parsabiv Amgen Secondary hyperparathyroidism Intravenous February 2017 nusinersen Spinraza Ionis/Biogen Spinal muscular atrophy (SMA) Intrathecal December 2016 bezlotoxumab Zinplava Merck Reduce recurrence of Clostridium difficile infection adalimumab-otto Amjevita [Biosimilar to Humira ] Amgen New indications for approved specialty products RA, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn s disease, UC, and plaque psoriasis Intravenous November 2016 Subcutaneous October 2016 Generic name Brand name Manufacturer New indication(s) Date approved bosentan Tracleer Johnson & Johnson For pediatric patients at least 3 years old and September 2017 older with idiopathic or congenital pulmonary arterial hypertension (PAH) nitisinone Orfadin Sobi Once daily dosing for HT-1 September 2017 tocilizumab Actemra Genentech Adults and pediatric patients 2 years of age August 2017 and older with chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS) deutetrabenazine Austedo Teva Tardive dyskinesia August 2017 sofosbuvir/valpatasvir Epclusa Gilead HCV co-infected with HIV August 2017 ibrutinib Imbruvica AbbVie/Janssens Chronic graft-versus-host-disease August 2017 ivacaftor Kalydeco Vertex Cystic fibrosis in patients 2 years and older with 1 of 5 splice mutations in the CFTR gene August 2017 abatacept Orencia BMS Psoriatic arthritis July 2017 abobotulinumtoxin A Dysport Ipsen Spasticity in adults from in the lower limbs June 2017 tocilizumab Actemra Roche Giant cell arteritis May 2017 ivacaftor Kalydeco Vertex To treat additional mutations in cystic fibrosis, May 2017 bringing the number of treatable CFTR gene mutations to 33 ranibizumab Lucentis Genetech (Roche) Diabetic retinopathy in patients without April 2017 diabetic macular edema (DME) ledipasvir/sofosbuvir Harvoni Gilead Hepatitis C in children April 2017 sofosbuvir Sovaldi Gilead Hepatitis C in children April 2017 ranibizumab Lucentis Genentech Myopic choroidal neovascularization (mcnv) February 2017 Factor VIII, recombinant, pegylated Adynovate Shire Pediatric hemophilia A patients who are 12 years old and younger, as well as for use in surgical settings for children and adults. December 2016

6 Specialty Pipeline Update: September 2017 Page 6 New indications for approved specialty products continued Generic name Brand name Manufacturer New indication(s) Date approved etanercept Enbrel Amgen Pediatric patients 4 years or older with November 2016 chronic moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy canakinumab Ilaris Novartis Tumor Necrosis Factor-Receptor October 2016 Associated Periodic Syndrome (TRAPSS), Hyperimmunoglobulin D Syndrome (HIDS)/ Mevalonate Kinase Deficiency (MKD), and Familial Mediterranean Fever (FMF) lumacaftor/ivacaftor Orkambi Vertex Treatment of cystic fibrosis in patients 6 years and older who are homozygous for the F508del mutation in the CFTR gene. October 2016 Oncology product approvals in the past twelve months Generic name Brand name Manufacturer Indication(s) Route of administration Date approved copanlisib Aliqopa Bayer Follicular lymphoma Intravenous September 2017 bevacizumab-awwb Mvasi (Biosimilar to Avastin ) Allergan/Amgen Colorectal cancer, NSCLC, glioblastoma, renal cell carcinoma, and cervical cancer Intravenous September 2017 gemtuzumab ozogamicin Mylotarg Pfizer AML Intravenous September 2017 tisagenlecleucel Kymriah Novartis r/r ALL (patients up to 25 years Intravenous August 2017 of age) inotuzumab ozogamicin Besponsa Pfizer r/r B-cell precursor ALL Intravenous August 2017 cytarabine/daunorubicin Vyxeos Jazz t-aml and AML-MRC Intravenous August 2017 liposome enasidenib Idhifa Celgene/Agios r/r AML with IDH2 mutation Oral August 2017 neratinib Nerlynx Puma Biotechnology Breast cancer Oral July 2017 rituximab/hyaluronidase human ribociclib/letrozole Rituxan Hycela Kisqali and Femara CoPack Genetech/Halozyme Therapeutics Follicular lymphoma, DLBCL and CLL Subcutaneous July 2017 Novartis Breast cancer Oral May 2017 brigatinib Alunbrig ARIAD ALK+ NSCLC resistant to Xalkori Oral April 2017 durvalumab Imfinzi AstraZeneca Bladder cancer IV April 2017 midostaurin Rydapt Novartis FLT3-mutated AML or systemic Oral April 2017 mastocytosis niraparib Zejula Tesaro Ovarian, fallopian tube, or Oral March 2017 primary peritoneal cancer avelumab Bavencio Merck KgaA/Pfizer Metastatic Merkel cell IV March 2017 carcinoma ribociclib Kisqali Novartis Breast cancer Oral March 2017 rucaparib Rubraca Clovis Ovarian cancer Oral December 2016 oloaratumab Lartruvo Lilly Soft tissue sarcoma Intravenous November 2016

7 Specialty Pipeline Update: September 2017 Page 7 New indications for approved oncology drugs Generic name Brand name Manufacturer Indication(s) Date approved ipilimumab Yervoy BMS Unresectable or metastatic melanoma in pediatric patients August years of age and older olaparib Lynparza AstraZeneca Accelerated approval to full approval for ovarian cancer August 2017 (new formulation of tablets) nivolumab Opdivo BMS chl, dmmr, and MSI-H colorectal cancer August 2017 blinatumomab Blincyto Amgen r/r B-cell precursor ALL July 2017 panitumumab Vectibix Amgen Wild-type RAS metastatic colorectal cancer July 2017 dabrafenib + trametinib Tafinlar + Mekinist Novartis Metastatic NSCLC with BRAF V600E mutation July 2017 daratumumab Darzalex Johnson & Johnson/ Genmab Used in combination with Pomalyst and dexamethasone for the treatment of patients with r/r multiple myeloma who have received at least 2 prior therapies, with a proteasome inhibitor and an immunomodulator. June 2017 certinib Zykadia Novartis ALK inhibitor for first-line treatment of patients with ALK+ NSCLC June 2017 pembrolizumab Keytruda Merck Unresectable or metastatic solid tumor associated with the June 2017 genetic abnormality microsatellite instability. pembrolizumab Keytruda Merck Bladder cancer May 2017 pembrolizumab Keytruda Merck Metastatic non-squamous non-small lung cancer May 2017 avelumab Bavencio Merck/Pfizer Bladder cancer May 2017 May 2017 regorafenib Stivarga Bayer Hepatocellular carcinoma in patients who were previously treated with Nexavar atezolizumab Tecentriq Genetech Bladder cancer April 2017 pembrolizumab Keytruda Merck Refractory classical Hodgkin s lymphoma March 2017 lenalidomide Revlimid Celgene Maintenance treatment of newly diagnosed multiple myeloma February 2017 patients after receiving autologous stem cell transplant (ASCT) nivolumab Opdivo Bristol Patients with locally advanced unresectable or metastatic February 2017 urothelial carcinoma following progression on a platinumcontaining therapy. ibrutinib Imbruvica Janssen Marginal zone lymphoma (MZL) who require systemic therapy January 2017 bevacizumab Avastin Genentech In combination with chemotherapy, followed by Avastin alone, for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer. daratumumab Darzalex Janssen In combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy. nivolumab Opdivo BMS To treat metastatic or recurrent squamous cell carcinoma of the head and neck following disease progression on or after platinum-based therapy. December 2016 November 2016 November 2016

8 Specialty Pipeline Update: September 2017 Page 8 New indications for approved oncology drugs continued Generic name Brand name Manufacturer Indication(s) Date approved pembrolizumab Keytruda Merck For the treatment of patients with metastatic NSCLC whose November 2016 tumors express PD-L1 as determined by an FDA-approved test. This approval also expands the indication in second-line treatment of lung cancer to include all patients with PD-L1- expressing NSCLC. erlotinib Tarceva Genetech For metastatic non-small cell lung cancer (NSCLC) to limit use to November 2016 patients whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by a FDA-approved test receiving firstline, maintenance, or second or greater line treatment after progression following at least 1 prior chemotherapy regimen. atezolizumab Tecentriq Roche Treatment of patients with metastatic non-small cell lung cancer (NSCLC) patients who have progressed during or following platinum-containing chemotherapy. October 2016 Pipeline watch Route of Anticipated Generic name Brand name Manufacturer Indication(s) administration approval date* N/A Hemophilia A or B with inhibitors Intravenous September 2017 eptacog beta, activated (Factor VIIa) Myl (trastuzumab) MYL-1401H (pegfilgrastim) N/A [Biosimilar to Herceptin ] N/A [Biosimilar to Neulasta ] revo Biologic/ LFB Mylan/Biocon HER2-positive breast and gastric Intravenous September 2017 cancers Mylan/Biocon Neutropenia Subcutaneous October 2017 benralizumab N/A AstraZeneca Asthma Subcutaneous December 2017 abemaciclib N/A Lilly Breast cancer Oral January 2018 acalabrutinib N/A AstraZeneca (Acerta Pharma) CT-P10 (rituximab) Truxia [Biosimilar Celltrion/Teva to Rituxan ] tildrakizumab N/A Sun Pharmaceutical Industries CT-P6 (trastuzumab) N/A [Biosimilar to Celltrion/Teva Herceptin ] abiraterone acetate Yonsa Churchill *Anticipated approval dates are predictions made by Prime Therapeutics based on industry information. Mantle cell lymphoma Oral February 2018 NHL, CLL, RA, granulomatosis with polyangiitis, and microscopic polyangiitis Intravenous February 2018 Plaque psoriasis Subcutaneous March 2018 HER2-positive breast and gastric Intravenous March 2018 cancers Prostate cancer Oral March 2018

9 Specialty Pipeline Update: September 2017 Page 9 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 Translarna (ataluren)/ PTC Therapeutics axicabtagene ciloleucel (KTE-C19)/ Kite Pharma & Gilead Luxturna (voretigene neparvovec)/sparks Therapeutics Ryplazim (human plasma-derived plasminogen, purified)/prometic Life Sciences, Inc ibalizumab/taimed Biologics and Theratechnologies Proposed indication/ route of administration Nonsense mutation Duchenne muscular dystrophy (nmdmd)/oral r/r B-cell NHL/IV Retinal dystrophy caused by mutations in the RPE65 gene/subretinal injection Congenital plasminogen deficiency/iv Multidrug resistant HIV-1 infection/iv Anticipated approval (PDUFA date) Similar products Spend* Net new impact to PMPM* pharmacy benefit Net new impact to PMPM* medical benefit October 2017 None $ $$ $ $$ None November Kymriah $$ $$$ None $$ $$$ 2017 January 2018 None $ $$ None $ $$ January 2018 None $ $$ None $ $$ January 2018 Treatment depends on resistance testing $$ None $$ Drugs to be evaluated for Watch List Tavalisse (fostamatinib)/rigel Chronic and persistent April 2018 CS, IVIG, Nplate, TBD TBD None immune thrombocytopenia/oral Promacta, Rituxan, anti-d emicizumab/genentech Prophylaxis of bleeds February 2018 NovoSeven, FEIBA TBD TBD None in hemophilia A with inhibitors/sc Lutathera (177Lu-DOTAO-Tyr3- GEP-NETS/IV February 2018 Sandostatin TBD None TBD octreotate)/advanced Accelerator Applications LAR Depot Pegvaliase (PEGylated recombinant phenylalanine ammonia lyase enzyme)/biomarin Reduce phenylalanine levels in patients with phenylketonuria/sc February 2018 Kuvan TBD TBD None burosumab/ultragenyx X-linked hypophosphatemia/iv April 2018 Phosphate supplements and high dose calcitriol Aimovig (erenumab) Prevention of migraines/sc May 2018 Beta-blockers, antidepressants, anticonvulsants TBD None TBD TBD TBD None

10 Specialty Pipeline Update: September 2017 Page 10 Orphan drugs, evaluated for Watch List, do not meet criteria Recombinant human MPS 7/IV November None <$0.08 PMPM beta-glucuronidase (rhgus)/ultragenyx 2017 sirolimus (DE-109)/Santeen Non-infectious uveitis, posterior segment December 2017 Humira Triesence Switch therapy: no net new trend. (NIU-PS)/Intravitreal injection Ozurdex Linhaliq (ciprofloxacin)/aradigm Non-cystic fibrosis bronchiectasis January 2018 [Off -Label] Cayston Switch therapy: no net new trend. patients with chronic lung infections with Pseudomonas aeruginosa/ inhaled (aztronam), tobramycin for inhalation tezacaftor & ivacaftor/vertex Cystic fibrosis/oral February 2018 Kalydeco Orkambi Switch therapy: no net new trend. *Notations: $ < $0.08 per member per month (PMPM), $$ = $0.08 $0.39 PMPM, $$$ = $0.40 $2.00 PMPM, $$$$ > $2.01 PMPM References All brand names are property of their respective owners B1 Prime Therapeutics LLC 09/17