Specialty Drug Pipeline Quarterly Update: June 2023

Critical updates in an ever-changing environment

June 22, 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

  • Columvi® (glofitamab-gxbm): The U.S. Food and Drug Administration (FDA) has granted accelerated approval Genentech’s Columvi for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified or large B-cell lymphoma (LBCL) arising from follicular lymphoma, after two or more lines of systemic therapy. Columvi was approved based on an overall response rate of 56% and 43% of patients achieving a complete response durability of response in the Phase 1/2 NP30179 study. Sixty-eight percent of patients who responded continued to respond for at least nine months. The median duration of response was 1.5 years.1 Columvi launch and price are pending.
  • Vyvgart® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc): The FDA has approved Halozyme Therapeutics’ Vyvgart Hytrulo injection for subcutaneous (SC) use for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. Vyvgart® Hytrulo is a subcutaneous product combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as Vyvgart®, and recombinant human hyaluronidase PH20 (rHuPH20). The product is to be administered subcutaneously by a healthcare professional as a single injection (1,008 mg fixed dose) over 30-90 seconds in cycles of once weekly injections for four weeks.  Vyvgart Hytrulo was approved based on the Phase 3 ADAPT-SC study that compared the efficacy and safety of SC efgartigimod to IV efgartigimod in adults with gMG. Results demonstrated that treatment with Vyvgart Hytrulo met the primary endpoint of noninferiority to IV efgartigimod at day 29. Patients treated with Vyvgart Hytrulo achieved a mean total IgG reduction of 66.4% compared with a 62.2% reduction for IV efgartigimod.2 Launch and price are pending.

New Indications

  • Bylvay® (odevibat): The FDA has expanded Albireo’s Bylvay to include a new indication for the non-systemic ileal bile acid transport inhibitor (IBATi) for treatment of cholestatic pruritis in patients aged 12 months and older with Alagille syndrome (ALGS).
  • Lynparza® (olaparib): The FDA approved AstraZeneca’s Lynparza (olaparib) to be used 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).
  • Rinvoq® (upadacitinib): AbbVie’s Rinvoq (upadacitinib) was approved by the FDA to include for treatment of adults with moderately to severely active Crohn’s disease.

June News

  • “The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6-0 in favor of fully approving Eisai and Biogen’s Alzheimer’s drug Leqembi (lecanemab), which first won an accelerated approval in January. The full approval, expected on or before July 6, would open the door to wider insurance and Medicare coverage for those with mild cognitive impairment or mild dementia and confirmed presence of amyloid beta pathology but who could not or do not want to pay the $26,500 per year list price.”3
  • “The FDA has granted priority and standard review designations to the biologics license applications for exagamglogene autotemcel (exa-cel; formerly CTX001) for the treatment of patients with sickle cell disease and transfusion-dependent beta thalassemia, respectively, marking the first CRISPR gene editing filings to be accepted for review by the FDA. The FDA has set action dates of December 8, 2023, for the sickle cell indication, and March 30, 2024, for the transfusion-dependent beta thalassemia indication.”4
  • “Merck is teasing data for efinopegdutide, a GLP-1/glucagon receptor co-agonist, in nonalcoholic steatohepatitis (NASH) and how it compares to Novo Nordisk’s semaglutide, with plans to share more detailed data from the study at the European Association for the Study of the Liver’s annual meeting later this month. Merck plans to use data from efinopegdutide’s Phase IIa trial in adult patients with nonalcoholic fatty liver disease to inform a Phase IIb study set to begin later this month in NASH, a more severe form of the disease, according to Merck. The FDA recently granted the drug, which is also being evaluated as a treatment for obesity and diabetes, a fast track designation in NASH.”4

References

  1. https://www.gene.com/media/news-features/fda-approves-genentechs-fixed-duration-bispecific-antibody-for-relapsed-refractory-diffuse-large-b-cell-lymphoma#:~:text=On%20June%2015%2C%202023%2C%20the,more%20lines%20of%20systemic%20therapy.
  2. https://www.prnewswire.com/news-releases/halozyme-announces-argenx-receives-fda-approval-for-vyvgart-hytrulo-with-enhanze-for-subcutaneous-use-in-generalized-myasthenia-gravis-301855994.html
  3. https://endpts.com/fda-adcomm-votes-unanimously-in-favor-of-full-approval-for-eisais-new-alzheimers-drug/
  4. https://www.onclive.com/view/fda-set-to-review-exagamglogene-autotemcel-for-sickle-cell-disease-transfusion-dependent-beta-thalassemia
  5. https://endpts.com/merck-steps-into-liver-disease-space-with-early-data-for-its-glp-1-glucagon-receptor-co-agonist/
Specialty New Product Approvals in the Past Twelve Months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved
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
sodium phenylbutyrate and taurursodiol Relyvrio™ Amylyx Pharmaceuticals Inc. Amyotrophic lateral sclerosis (ALS) Oral September 2022
eflapegrastim-xnst Rolvedon® Spectrum Pharmaceuticals/ Hanmi Pharmaceutical Chemotherapy-induced neutropenia SC September 2022
sodium thiosulfate Pedmark™ Fennec Pharmaceuticals Prevention of ototoxicity induced by cisplatin IV September 2022
terlipressin Terlivaz® Mallinckrodt Improve kidney function in adults with hepatorenal syndrome (HRS) SC September 2022
deucravacitinib Sotyktu® Bristol Myers Squibb Plaque psoriasis Oral September 2022
spesolimab-sbzo Spevigo® Boehringer Ingelheim Generalized pustular psoriasis (GPP) flares IV September 2022
olipudase alfa-rpcp Xenpozyme® Sanofi Acid sphingomyelinase deficiency IV August 2022
sodium oxybate Lumryz™

Tentative approval

Avadel Pharmaceuticals Narcolepsy Oral July 2022

 

New Indications for Approved Specialty Products
Generic Name Brand Name Manufacturer New Indication(s) Date Approved
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
dupilumab Dupixent® Sanofi/ Regeneron Adult patients with prurigo nodularis September 2022
lumacaftor and ivacaftor Orkambi® Vertex Cystic fibrosis in patients aged 12 months to less than 24 months who are homozygous for the F508del mutation in the CFTR gene September 2022
risankizumab-rzaa Skyrizi® SC Boehringer Ingelheim/ AbbVie Crohn’s disease September 2022
ustekinumab Stelara® Johnson & Johnson (Janssen) Pediatric patients aged 6 years and older with psoriatic arthritis (PsA) August 2022
belimumab Benlysta® GlaxoSmithKline Patients aged 5 to 17 years old with active lupus nephritis July 2022
ruxolitinib topical cream Opzelura® Incyte Nonsegmental vitiligo in adults and adolescents 12 year of age and older July 2022
pegloticase + methotrexate Krystexxa® + methotrexate Horizon Co-administration with methotrexate for the treatment of chronic gout July 2022

 

Oncology Product Approvals in the Past Twelve Months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Date Approved
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
futibatinib Lytgobi® Taiho Oncology Metastatic cholangiocarcinoma Oral September 2022

 

New Indications for Approved Oncology Drugs
Generic Name Brand Name Manufacturer New Indication Date Approved
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
selpercatinib Retevmo® Lilly (Loxo) Certain forms of locally advanced or metastatic solid tumors with a rearranged during transfection (RET) September 2022
durvalumab Imfinzi® AstraZeneca Use in combination with standard of care chemotherapy for treatment of locally advanced or metastatic biliary tract cancer (BTC) September 2022
pemigatinib Pemazyre® Incyte R/R myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement August 2022
capmatinib Tabrecta® Novartis Certain forms of metastatic NSCLC August 2022
darolutamide Nubeqa® Bayer/ Orion In combination with docetaxel for treatment of metastatic hormone-sensitive prostate cancer (mHSPC) August 2022
fam-trastuzumab deruxtecan-nxki Enhertu® Daiichi Sankyo/ AstraZeneca Unresectable or metastatic HER2-low breast cancer August 2022
crizotinib Xalkori® Pfizer Inflammatory myofibroblastic tumor (IMT) July 2022

 

Specialty Pipeline
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Approval date*
ritlecitinib N/A Pfizer Alopecia areata Oral June 2023
obeticholic acid N/A Intercept Pharmaceuticals Nonalcoholic steatohepatitis Oral June 2023
quizartinib N/A Daiichi Sankyo Acute myeloid leukemia Oral July 2023
nirsevimab Beyfortus™ AstraZeneca Prevention of respiratory syncytial virus (RSV) IM July 2023
rozanolixizumab N/A UCB Generalized myasthenia gravis (gMG) SC July 2023
avacincaptad pegol Zimura™ Iveric Bio Geographic atrophy (GA) secondary to age-related macular degeneration (AMD) Intravitreal August 2023
palovarotene Sohonos™ Ipsen (Clementia) Fibrodysplasia ossificans progressiva (FOP) Oral August 2023
elranatamab N/A Pfizer Relapsed or refractory multiple myeloma (RRMM) SC August 2023
nirogacestat N/A SpringWorks Therapeutics Desmoid tumors Oral August 2023
avasopasem manganese N/A Galera Therapeutics Oral mucositis (SOM) in patients with head and neck cancer (HNC) undergoing standard-of-care treatment IV August 2023
talquetamab N/A Johnson & Johnson (Janssen) RRMM IV August 2023
melphalan chemosaturation system Hepzato Kit™ Delcath Systems Drug/device combination for treatment of unresectable hepatic-dominant metastatic ocular melanoma (mOM) Injectable August 2023
pozelimab N/A Regeneron CHAPLE disease IV or SC August 2023
remestemcel-L N/A Novartis/Mesoblast Graft versus host disease IV August 2023
momelotinib N/A Sierra Oncology, Inc. Myelofibrosis Oral September 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
motixafortide + G-CSF Aphexda™ + G-CSF BioLineRx Ltd. Multiple myeloma SC September 2023
atezolizumab Tecentriq™ SC Roche/Halozyme Certain forms of cancer SC September 2023
etrasimod N/A Pfizer Ulcerative colitis Oral October 2023
CTP13 SC (remsima SC biobetter) N/A Celltrion Crohn’s disease SC October 2023
vamorolone N/A Santhera Pharmaceuticals /ReveraGen BioPharma Duchenne muscular dystrophy (DMD) Oral October 2023
TAK-755 (apadamtase alfa) N/A Takeda An ultra-rare inherited ADAMTS13 deficiency disorder IV October 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
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
camrelizumab N/A Hengrui Pharma (Jiangsu Hengrui) Unresectable hepatocellular carcinoma (uHCC) IV May 2024

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

 

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