Specialty Drug Pipeline Quarterly Update: September 2023

Critical updates in an ever-changing environment

September 25, 2023

This monthly pipeline wrap-up provides a review of newly approved specialty drugs, recent specialty drug launches, new indications and news of note on specialty drugs in the approval process. See separate articles for pipeline information on traditional drugs, biosimilars and gene/cell therapy.

New Drug Information

  • Aphexda (motixafortide): BioLine Therapeutics’ Aphexda was FDA approved in combination with filgrastim, a granulocyte stimulating factor) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation (ASCT) in patients with multiple myeloma (MM). Aphexda is administered subcutaneously by a health care professional 10 to 14 hours prior to beginning apheresis. In the Phase 3 GENESIS trial, 67.5% of patients treated with Aphexda achieved the stem cell collection goal within two apheresis sessions versus 9.5% of patients who received filgrastim alone, as measured by central laboratory. Mozobil® (plerixafor) and its generic versions which were launched in July 2023, share a similar indication. Launch of Aphexda is planned in September 2023 with pricing to follow.1
  • Ojjaara™ (momelotinib): GlaxoSmithKline’s oral, once-daily Ojjaara received FDA approval for the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF [postpolycythemia vera (PV) and post-essential thrombocythemia (ET)], in adults with anemia.[i] Ojjaara is a first-in-class inhibitor of Janus kinase 1 and 2 (JAK1/2) and activin A receptor type 1 (ACVR1) and first JAK inhibitor to treat myelofibrosis in patients with anemia. In the phase 3 MOMENTUM trial, momelotinib was superior to danazol in the primary endpoint of the proportion of patients achieving a ≥ 50% reduction from baseline in the Myelofibrosis Symptom Assessment Form Total Symptom Score (MSA TSS; 25% versus 9%, respectively; p=0.0095).2 The wholesale acquisition cost (WAC) for Ojjaara will be $322,800. Ojjaara will compete for market share with: Jakafi (ruxolitinib), Inrebic (fedrantinib), and Vonjo (pancritinib). Ojjaara is the only one approved for both newly diagnosed and previously treated myelofibrosis patients with anemia; however, in a Phase 3 trial, SIMPLIFY-1, Ojjaara was non-inferior to Jakafi in the primary endpoint (splenic volume reduction) but failed to improve disease-associated symptoms compared with Jakafi.3

 New Indications

  • Reblozyl® (luspatercept-aamt): The FDA approved a new indication for Bristol-Myers Squibb’s Reblozyl for the treatment of anemia without previous use of an erythropoiesis stimulating agent use (ESA-naïve) in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions.
  • Mekinist® (trametinib) plus Tafinlar® (dabrafenib): The FDA expanded the indications for Novartis’ Mekinist and Tafinlar for their combined use to treat patients with unresectable or metastatic solid tumors with BRAF V600E mutation that has progressed following prior treatment and with no satisfactory alternative treatment options to include patients 1 to 5 years of age. The indications were approved under Accelerated Approval based on overall response rate and duration of response; continued approval may depend upon results from confirmatory trial(s).
  • Temodar® (temozolomide): The FDA approved Merck’s Temodar for a new indication for adjuvant treatment of adults with newly diagnosed anaplastic astrocytoma. The FDA also revised the existing refractory anaplastic astrocytoma indication to remove the requirement that patients had experienced disease progression while on a procarbazine- and nitrosourea-containing drug regimen.

References

  1. BioLineRx’s Aphexda receives FDA approval for stem cell mobilisation (msn.com)
  2. https://www.formularywatch.com/view/fda-approves-gsk-s-myelofibrosis-drug
  3. https://ashpublications.org/ashclinicalnews/news/3197/Mixed-Results-in-Trials-of-Momelotinib-Versus

 

 

Specialty New Product Approvals in the Past Twelve Months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved
motixafortide Aphexda BioLine Therapeutics Mobilize hematopoietic stem cells SC September 2023
aflibercept Eylea HD Regeneron Diabetic macular edema Intravitreal August 2023
pozelimab-bbfg Veopoz Regeneron CD55-deficient IV/SC August 2023
palovarotene Sohonos Ipsen Fibrodysplasia ossificans Oral August 2023
Avacincaptad pegol Izervay Astellas/Iveric Dry age-related macular degeneration Intravitreal August 2023
Nirsevimab-alip Beyfortus AstraZeneca/Sanofi Prevention of RSV IM July 2023
Somatrogon-ghla Ngenla Pfizer/OPKO Pediatric growth hormone deficiency SC June 2023
rozanolixizumab-noli Rystiggo® UCB Myasthenia gravis SC June 2023
ritlecitinib Litfulo® Pfizer Alopecia areata Oral June 2023
efgartigimod alfa and hyaluronidase-qvfc Vyvgart® Hytrulo Argenx Generalized myasthenia gravis (gMG) SC- New formulation June 2023
pegunigalsidase alfa- iwxj Elfabrio® Chiesi Global Rare Diseases/ Protalix Fabry disease IV May 2023
fecal microbiota spores live-brpk Vowst® Seres Therapeutics Recurrent C difficile infection Oral April 2023
tofersen Qalsody® Biogen Superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis Intrathecal April 2023
elexacaftor/tezacaftor/ivacaftor and ivacaftor Trikafta® Vertex Pharmaceuticals Cystic fibrosis 2 through 5 years of age with at least one F508del mutation or a mutation that is responsive based on in vitro data Oral April 2023
leniolisib Joenja® Pharming Activated phosphoinositide 3-kinase delta syndrome (APDS) Oral March 2023
trofinetide Daybue® Acadia Pharmaceuticals Inc. Rett syndrome Oral March 2023
omaveloxolone Skyclarys™ Reata Pharmaceuticals Friedreich’s ataxia Oral February 2023
deutetrabenazine Austedo™ XR Teva Tardive dyskinesia and chorea associated with Huntington disease Oral February 2023
antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl Altuviiio™ Sanofi/ Sobi Hemophilia A IV February 2023
sparsentan Filspari Travere Therapeutics  IgA nephropathy (IgAN) Oral February 2023
velmanase alfa Lamazede™ Chiesi Global Rare Diseases Alpha-mannosidosis IV February 2023
pegcetacoplan Syfovre™ Apellis Pharmaceuticals Geographic atrophy secondary to AMD Intravitreal February 2023
daprodustat Jesduvroq™ tablets GlaxoSmithKline Oral treatment for anemia from chronic kidney disease Oral February 2023
lecanemab-irmb Leqembi™ Eisai Alzheimer’s disease IV January 2023
anacaulase-bcdb NexoBrid™ MediWound Burn tissue Topical gel January 2023
sodium phenylbutyrate Olpruva™ Acer Therapeutics/ Relief Therapeutics Urea cycle disorders Oral January 2023
ublituximab-xiiy Briumvi™ TG Therapeutics Relapsing multiple sclerosis IV December 2022
fecal microbiota, live-jslm Rebyota™ Ferring (Rebiotix) Clostridioides difficile Rectal November 2022
teplizumab-mzwv Tzield™ Provention Bio Delay of certain forms of diabetes IV November 2022
furosemide Furoscix™ ScPharmaceuticals Diuresis in patients with worsening heart failure SC minipump October 2022

 

New Indications for Approved Specialty Products
Generic Name Brand Name Manufacturer New Indication(s) Date Approved
luspatercept-aamt Reblozyl BMS/Celgene Anemia due to ….. August 2023
odevixibat Bylvay® Albireo Alagille syndrome June 2023
upadacitinib Rinvoq® AbbVie Moderately to severely active Crohn’s disease May 2023
ivacaftor Kalydeco® Vertex Cystic fibrosis patients with CFTR gene mutations May 2023
somapacitan-beco Sogroya® Novo Nordisk Endocrine gland diseases April 2023
elexacaftor/tezacaftor/ivacaftor and ivacaftor Trikafta® Vertex Cystic fibrosis 2 through 5 years of age with at least one F508del mutation or a mutation that is responsive based on in vitro data April 2023
immune globulin infusion; recombinant human hyaluronidase HyQvia® Takeda Primary immunodeficiency April 2023
maralixibat Livmarli® Mirum Pharmaceuticals Expanded pediatric indication to lower the one-year and older age threshold for treatment of cholestatic pruritis in Alagille syndrome (ALGS) to patients aged three months of age and older March 2023
evinacumab-dgnb Evkeeza® Regeneron Adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia

 

March 2023
sildenafil citrate oral suspension Revatio® oral suspension Pfizer Pulmonary arterial hypertension March 2023
sarilumab Kevzara® Sanofi/ Regeneron Polymyalgia rheumatica (PMR) who had an inadequate response to corticosteroids or cannot tolerate corticosteroid taper February 2023
aflibercept Eylea® Regeneron Retinopathy of prematurity February 2023
abrocitinib Cibinqo® Pfizer Adolescents (12 to <18 years) with refractory, moderate to severe atopic dermatitis (AD) not adequately controlled with other systemic products, including biologics, or when those therapies is inadvisable February 2023
lanadelumab-flyo Takhzyro® Shire Expanded pediatric indication for prophylaxis to prevent attacks of hereditary angioedema (HAE) in children 2 to <12 years of age February 2023
tezepelumab-ekko Tezspire® AstraZeneca/ Amgen Severe asthma February 2023
tocilizumab Actemra® IV Genentech (Roche) COVID-19 in hospitalized adults who are receiving systemic corticosteroids and require supplemental oxygen, mechanical ventilation, or extracorporeal membrane oxygenation (ECMO) December 2022
abaloparatide Tymlos® Radius Health Men with osteoporosis at high risk for fracture December 2022
tildrakizumab-asmn Ilumya® Sun/ Merck &Co Plaque psoriasis of the scalp December 2022
upadacitinib Rinvoq® AbbVie Active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation who have responded inadequately to NSAIDs October 2022
lumasiran Oxlumo® Alnylam Primary hyperoxaluria type 1 (PH1) to include reduction of plasma oxalate in patients with advanced PH1, including patients with end-stage kidney disease on dialysis October 2022
Oncology Product Approvals in the Past Twelve Months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Date Approved
momelotinib Ojjaara GSK Myelofibrosis with anemia Oral September 2023
melphalan Hepzato Kit Delcath Uveal melanoma Injectable August 2023
elranatamab-bcmm Elrexfio Pfizer Multiple myeloma SC August 2023
abiraterone acetate/niraparib Akeega Janssen Prostate cancer Oral August 2023
talquetamab-tgvs Talvey Janssen Multiple myeloma SC August 2023
quizartinib Vanflyta Daiichi Sankyo AML Oral July 2023
glofitamab-gxbm Columvi® Roche Relapsed or refractory large B-cell lymphoma IV June 2023
epcoritamab-bysp Epkinly® Genmab A/S Relapsed/refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy IV May 2023
dabrafenib + trametinib Tafinlar® + Mekinist® Novartist/GSK Pediatric patients with low-grade glioma (LGG) with a BRAF V600E mutation Oral solution March 2023
retifanlimab-dlwr Zynyz® Incyte Corporation Adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) IV March 2023
pirtobrutinib Jaypirca® Lilly (Loxo) Certain forms of relapsed or refractory mantle cell lymphoma (MCL) Oral January 2023
elacestrant Orserdu® Radius Health/ Menarini ER+/ HER2- advanced or metastatic breast cancer Oral January 2023
mosunetuzumab-axgb Lunsumio® Genentech Certain forms of relapsed or refractory follicular lymphoma IV December 2022
adagrasib Krazati® Mirati Therapeutics Second-line treatment of advanced non-small cell lung cancer (NSCLC) Oral December 2022
olutasidenib Rezlidhea® Forma/ Rigel Relapsed or refractory acute myeloid leukemia Oral December 2022
docetaxel Docetaxel® (Meridian) Meridian Laboratories Certain forms of cancer IV November 2022
tremelimumab-actl Imjudo® AstraZeneca MedImmune Certain forms non-small cell lung cancer IV November 2022
mirvetuximab soravtansine-gynx Elahere® ImmunoGen Certain forms of folate receptor alpha-high platinum-resistant ovarian cancer IV November 2022
teclistamab-cqyv Tecvayli® Johnson & Johnson (Janssen) Relapsed or refractory multiple myeloma IV or SC October 2022
tremelimumab-actl Imjudo® AstraZeneca Certain forms of unresectable hepatocellular carcinoma IV October 2022
New Indications for Approved Oncology Drugs
Generic Name Brand Name Manufacturer New Indication Date Approved
temozolomide Temodar Merck Newly diagnosed anaplastic astrocytoma/ Existing refractory anaplastic astrocytoma September 2023
luspartercept-aamt Reblozyl BMS ESA-naïve anemia due to MDS August 2023
trametinib/dabrafenib Mekinist/Tafinlar Novartis Solid tumors August 2023
Tipiracil hydrochloride Lonsurf Taiho Colonrectal cancer Oral
Dostarlimab-gxly Jemperli GSK/Tesaro Endometrial cancer July 2023
talazoparib tosylate Talzenna Medivation/

Pfizer

Prostate cancer June 2023
olaparib Lynparza® AstraZeneca In combination with abiraterone and prednisone or prednisolone for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC) June 2023
polatuzumab vedotin-piiq Polivy® Genentech (Roche) Previously untreated diffuse large B-cell lymphoma (DLBCL) April 2023
enfortumab vedotin-ejfv Padcev® Astellas/ Seagen First-line treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are ineligible for cisplatin chemotherapy April 2023
pembrolizumab Keytruda® Merck Advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation March 2023
abemaciclib Verzenio® Lilly Certain forms hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-), node-positive, early breast cancer (EBC) at a high risk of recurrence March 2023
dostarlimab-gxly Jemperli® GlaxoSmithKline (Tesaro)/ AnaptysBio Recurrent or advanced mismatch repair deficient (dMMR) endometrial cancer, as determined by an FDA-approved test, who have progressed on or after platinum-based regimens and are not candidates for curative surgery or radiation February 2023
sacituzumab govitecan-hziy Trodelvy® Gilead HR+, HER2- breast cancer patients who have received endocrine-based therapy and at least 2 other systemic therapies for metastatic cancer. February 2023
pembrolizumab Keytruda® Merck Adjuvant treatment of patients with stage IB, II or IIIA non-small cell lung cancer (NSCLC) following complete surgical resection January 2023
tucatinib Tukysa® Seagen (formerly Seattle Genetics) HER2+ colorectal cancer who have received at least one prior treatment regimen for unresectable or metastatic disease January 2023
zanubrutinib Brukinsa® BeiGene Adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) January 2023
rucaparib camsylate Rubraca® Clovis Oncology Peritoneal cancer December 2022
atezolizumab Tecentriq® Genentech Alveolar soft part sarcoma December 2022
pemetrexed Pemfexy® Eagle Pharmaceuticals First-line therapy for non-squamous non-small cell lung cancer December 2022
palbociclib Ibrance® Pfizer Hormone receptor positive breast cancer December 2022
asparaginase erwinia chrysanthemi (recombinant)- rywn Rylaze® Jazz Pharmaceuticals Acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who have developed hypersensitivity to E coli-derived asparaginase November 2022
tremelimumab-actl Imjudo® AstraZeneca Metastatic non-small cell lung cancer (NSCLC) with no sensitizing EGFR mutation or anaplastic lymphoma kinase (ALK) genomic tumor aberrations November 2022
durvalumab Imfinzi® AstraZeneca NSCLC with no sensitizing EGFR mutation or anaplastic lymphoma kinase (ALK) genomic tumor aberrations November 2022
cemiplimab-rwlc Libtayo® IV Regeneron/ Sanofi Certain forms of patients with advanced NSCLC with no EGFR, ALK or ROS1 aberrations November 2022
brentuximab vedotin Adcetris® Seagen Pediatric patients with previously untreated high risk classical Hodgkin lymphoma (cHL) November 2022
cemiplimab-rwlc Libtayo® Regeneron NSCLC with no EGFR, ALK, or ROS1 aberrations November 2022
cobimetinib Cotellic® Genentech (Roche) Adults with histiocytic neoplasm October 2022
Specialty Pipeline
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Approval date*
toripalimab Tuoyi Coherus Nasopharyngeal carcinoma IV 3Q2023
cipaglucosidase alfa + miglustat N/A Amicus Pompe disease IV 3Q2023
tislelizumab BeiGene/

Novartis

Novartis Esophageal squamous cell carcinoma IV 4Q2023
vedolizumab Entyvio SC Takeda Ulcerative colitis SC 4Q2023
eflornithine   US World Meds Pediatric neuroblastoma Oral 4Q2023
efbemalenograstim Ryzneuta Evive Biotech Chemotherapy-induced neutropenia SC 2023
nirogacestat Ogsiveo SpringWorks Therapeutics Desmoid tumors Oral November 2023
nedosiran N/A Novo Nordisk (Dicerna) Primary hyperoxaluria SC September 2023
zilucoplan N/A UCB Generalized myasthenia gravis SC September 2023
lebrikizumab N/A Lilly Atopic dermatitis SC September 2023
I/Ontak (denileukin diftitox) N/A Citius Pharmaceuticals Recurrent cutaneous T-cell lymphoma (CTCL) IV September 2023
etrasimod N/A Pfizer Ulcerative colitis Oral October 2023
CTP13 SC (remsima SC) N/A Celltrion Crohn’s disease SC October 2023
vamorolone N/A Santhera Pharmaceuticals /ReveraGen BioPharma Duchenne muscular dystrophy (DMD) Oral October 2023
repotrectinib N/A BMS NSCLC Oral November 2023
TAK-755 (apadamtase alfa) N/A Takeda Congenital thrombotic thrombocytopenic purpura IV November 2023
fruquintinib N/A Hutchmed and Takeda Certain forms of metastatic colorectal cancer Oral November 2023
eplontersen N/A Ionis Pharmaceuticals Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) SC December 2023
capivasertib N/A AstraZeneca/Astex Hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer

 

Oral December 2023
mirikizumab Omvoh Lilly Ulcerative colitis IV/SC December 2023
iptacopan N/A Novartis PNH Oral December 2023
givinostat N/A Italfarmaco DMD Oral December 2023
eplontersen N/A Ionis Hereditary transthyretin-mediated amyloid polyneuropathy SC December 2023
cosibelimab N/A Checkpoint Therapeutics Metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC who are not candidates for curative surgery or radiation IV January 2024
zolbetuximab N/A Astellas HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma IV January 2024
bimekizumab Bimzelx® UCB Pharma Plaque psoriasis SC 2024
danicopan N/A Alexion Paroxysmal nocturnal hemoglobinuria (PNH) with extravascular hemolysis (EVH) Oral January to February 2024
Immune globulin IV human, 10% liquid N/A GC Biopharma Primary humoral immunodeficiency (PI) to reduce serious bacterial infections IV January 2024
donanemab N/A Lilly Early symptomatic Alzheimer’s disease IV January 2024
resmetirom N/A Madrigal Nonalcoholic steatohepatitis Oral March 2024
camrelizumab N/A Hengrui Pharma (Jiangsu Hengrui) Unresectable hepatocellular carcinoma (uHCC) IV May 2024
rivoceranib N/A Elevar Therapeutics uHCC Oral May 2024
imetelstat N/A Geron Myelodysplastic syndromes IV June 2024
crovalimab N/A Genentech PNH SC June 2024

* Anticipated approval dates are predictions made by Prime Therapeutics based on industry information.

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