Specialty Drug Pipeline Quarterly Update: March 2023

March 27, 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

  • Altuviiio® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl]: The U.S. Food and Drug Administration (FDA) has approved Sanofi’s Altuviiio, a first-in-class, high-sustained factor VIII replacement therapy. Altuviiio is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. Altuviiio is the first hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis. The FDA’s approval was based on the Phase 3 clinical trial, XTEND-1 that demonstrated once-weekly Altuviiio prophylaxis met the primary endpoint of providing significant bleed protection for people with severe hemophilia A with a mean annualized bleeding rate (ABR) of 0.70 and a median ABR of 0.0.1 Altuviiio is scheduled to be commercially available in April and will be priced at parity to the annual cost of treating a prophylaxis patient on Eloctate® which is $12 per unit or a cost of $12,000 for a patient requiring 1000IU weekly or $625,000 annually.2
  • Skyclarys™ (omaveloxolone): The FDA has approved Reata Pharmaceuticals’ Skyclarys for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. Friedreich’s ataxia is a rare, genetic, neurodegenerative condition. Patients with Friedreich’s ataxia typically experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue. Approximately 5,000 patients are currently diagnosed with Friedreich’s ataxia in the U.S. Skyclarys was approved based on a clinical trial that demonstrated patients treated with Skyclarys had a statistically significant lower modified Friedreich Ataxia Rating Scale (mFARS) score compared to placebo at Week 48, accounting for a placebo-corrected difference between the groups of –2.41 points.3 Skyclarys is available as a 50 mg capsule. Skyclarys has launched with an average wholesale price (AWP) of $37,000 per bottle (90 capsules).
  • Daybue® (trofinetide): The FDA approved Acadia Pharmaceuticals’ Daybue for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Daybue is the first FDA approved medication for Rett syndrome. Rett syndrome is caused by a genetic mutation in which the patients develop normally in infancy but then begin to deteriorate, losing their speech, motor control of their hands and other functions. There are approximately 4,500 diagnosed Rett syndrome patients in the US. Daybue is a twice daily oral solution that works by reducing inflammation in the brain, stopping certain types of cells from becoming overactive, and increasing the amount of the naturally occurring protein called IGF-1. Daybue was approved based on a Phase 3 Lavender trial which demonstrated that treatment with Daybue improved patients’ symptoms on two questionnaire scoring systems compared to placebo.4 Patients reported consistent benefits. Daybue is expected to be available by the end of April with an average list price estimated to be about $575,000 to $595,000 a year or $21.10 per milliliter for weight-based dosing.

New Indications

  • Verzenio® (abemaciclib): The FDA approved an expanded indication for Lilly’s Verzenio, in combination with endocrine therapy (ET), for the adjuvant treatment of adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-), node-positive, early breast cancer (EBC) at a high risk of recurrence. High risk patients eligible for Verzenio can now be identified solely based on nodal status, tumor size, and tumor grade (4+ positive nodes, or 1-3 positive nodes and at least one of the following: tumors that are 5 cm or Grade 3). This expanded adjuvant indication removes the Ki-67 score requirement for patient selection.
  • Kevzara® (sarilumab): The FDA expanded the indication of Sanofi and Regeneron’s Kevzara to include the treatment of adults with polymyalgia rheumatica (PMR) who had an inadequate response to corticosteroids or cannot tolerate corticosteroid taper.
  • Jemperli® (dostarlimab-gxly): The FDA granted a conversion from accelerated approval to full approval of GlaxoSmithKline (Tesaro) and AnaptysBio’s Jemperli with revised indication for treatment of patients with 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.

March News

  • “A sudden change of heart at the FDA has sunk Sarepta Therapeutics’ stock. Two weeks after saying the FDA would assess its gene therapy without an advisory committee meeting, Sarepta has revealedthe agency will now convene experts to dig into the data on its Duchenne muscular dystrophy (DMD) submission. At the end of last month, Sarepta told investors it had completed the midcycle review for the SRP-9001 submission, and, with no significant safety or clinical issues coming up, the FDA had decided to proceed without an advisory committee meeting. The decision triggered a jump in Sarepta’s share price and put it on course to potentially win approval by May 29 without facing the scrutiny of independent experts.”5
  • “BioMarin Pharmaceutical Inc. announced that it received notice from the FDA that the agency has extended review of the company’s Biologics License Application (BLA) for Roctavian™ (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A. The FDA determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA Target Action Date of June 30, 2023. The company had previously communicated that this data submission could be qualified as a Major Amendment.”6
  • “Ahead of its advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show. Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease. “Despite the notable limitations of a failed study and the many post hoc exploratory analyses that were conducted after [the Phase III trial], the Division considers that the data may suggest a treatment effect of tofersen in SOD1-ALS,” the FDA wrote. “This is a very rare and devastating disease; therefore, it is of utmost importance that we give full consideration to all of the available data.” Tofersen did not achieve the primary goal of its Phase 3 VALOR trial, failing to prove it could slow SOD1-ALS patients’ functional decline better than a placebo. Reported in October 2021, the results showed there were higher odds that any observable drug effect on function was due to random chance than tofersen producing its desired outcome. Biogen said the p-value for the primary endpoint was p=0.97.”7
  • “The FDA is moving to optimize the development of gene therapies, including encouraging the use of biomarkers to help get accelerated approval of therapies for serious conditions, an agency official said. Biomarkers, such as pulse and blood pressure, are characteristics of the body that you can measure. The FDA will support using biomarkers as substitutes for other biological indicators in gene-therapy clinical trials to help clinch “accelerated approval”, said the agency’s Peter Marks.”8

References

  1. https://www.sanofi.com/en/media-room/press-releases/2023/2023-02-23-21-00-00-2614759
  2. https://www.sanofi.com/en/media-room/press-releases/2023/2023-02-23-21-00-00-2614759
  3. https://www.neurologylive.com/view/fda-approves-omaveloxolone-first-treatment-friedreich-ataxia
  4. https://www.rettsyndrome.org/trofinetideapproved/
  5. https://www.fiercebiotech.com/biotech/fda-shocks-sarepta-ordering-adcomm-dmd-gene-therapy-sudden-change-heart
  6. https://investors.biomarin.com/2023-03-06-BioMarin-Provides-Update-on-FDA-Review-of-ROCTAVIAN-TM-Valoctocogene-Roxaparvovec-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A
  7. https://endpts.com/fda-indicates-willingness-to-approve-biogen-als-drug-despite-failed-phiii-study/
  8. https://www.reuters.com/world/us/us-fda-official-says-agency-needs-start-using-accelerated-approval-gene-2023-03-20/
Specialty New Product Approvals in the Past Twelve Months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved
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
vutrisiran Amvuttra® Alnylam Pharmaceuticals Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis SC June 2022
edaravone Radicava ORS® Mitsubishi Tanabe Pharma Amyotrophic lateral sclerosis (ALS) Oral May 2022
trientine tetrahydrochloride Cuvrior™ Orphalan Wilson’s disease Oral May 2022
treprostinil dry powder for inhalation Tyvaso DPI® United Therapeutics Corp New dry powder formulation of for treatment of pulmonary arterial hypertension Inhaled May 2022
mavacamten Camzyos® Bristol-Myers Squibb Symptomatic obstructive hypertrophic cardiomyopathy (oHCM) Oral April 2022

 

New Indications for Approved Specialty Products
Generic Name Brand Name Manufacturer New Indication(s) Date Approved
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 years of age and older July 2022
pegloticase + methotrexate Krystexxa® + methotrexate Horizon Co-administration with methotrexate for the treatment of chronic gout July 2022
setmelanotide Imcivree® Rhythm Pharmaceuticals Obesity and control of hunger in adult and pediatric patients 6 years of age and older with Bardet-Biedl syndrome (BBS) June 2022
dupilumab Dupixent® Sanofi/ Regeneron For add-on maintenance treatment of pediatric patients aged 6 months to 5 years old with moderate to severe atopic dermatitis June 2022
baricitinib Olumiant® Lilly/ Incyte Alopecia areata June 2022
mycophenolate mofetil Cellcept® Roche Prophylaxis of organ rejection to pediatric recipients of allogenic heart and liver transplants June 2022
brolucizumab-dbll Beovu® Novartis Diabetic macular edema June 2022
risdiplam Evrysdi® Genentech Pre-symptomatic babies under 2 months of age with Spinal Muscular Atrophy (SMA) May 2022
dupilumab Dupixent® Sanofi Pharmaceuticals To treat eosinophilic esophagitis (EoE) in adults and pediatric patients 12 years and older weighing at least 40 kilograms (which is about 88 pounds) May 2022
baricitinib Olumiant® Lilly/ Incyte For the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO) May 2022
upadacitinib Rinvoq® Abbvie Adults with active ankylosing spondylitis April 2022
ravulizumab-cwvz Ultomiris® AstraZeneca (Alexion) Generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody-positive April 2022
remdesivir Veklury® Gilead Certain pediatric patients 28 days of age and older weighing at least 3 kilograms with positive results of direct SARS-CoV-2 viral testing April 2022

 

 

 

Oncology Product Approvals in the Past Twelve Months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Date Approved
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
bortezomib Bortezomib® Hospira Multiple myeloma

mantle cell lymphoma

IV or SC May 2022
alpelisib Vijoice® Novartis

 

Severe manifestations of PIK3CA-related overgrowth Spectrum (PROS) Oral April 2022
New Indications for Approved Oncology Drugs
Generic Name Brand Name Manufacturer New Indication Date Approved
abemaciclib Verzenio® Lilly Certain forms of 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 two 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 one 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
carfilzomib Kyprolis® Onyx (Amgen) R/R multiple myeloma (RRMM) who have received one to three lines of therapy. June 2022
lisocabtagene maraleucel Breyanzi® Bristol-Myers Squibb R/R large B-cell lymphoma (LBCL) after failure of first-line therapy June 2022
dabrafenib and trametinib Tafinlar® and Mekinist® Novartis Adult and pediatric patients aged 6 years and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment June 2022
fam-trastuzumab deruxtecan-nxki Enhertu® Daiichi Sankyo/ AstraZeneca Unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen May 2022
tisagenlecleucel Kymriah® Novartis R/R follicular lymphoma (FL) after two prior lines of treatment May 2022
nivolumab and ipilimumab Opdivo® and Yervoy® Bristol Myers Squibb Unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) May 2022
ivosidenib Tibsovo® Agios Pharmaceuticals In combination with azacitidine for newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy May 2022
azacitadine Vidaza® Bristol Myers Squibb (Celgene) New indication for the nucleoside metabolic inhibitor for treatment of pediatric patients aged one month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML) May 2022
axicabtagene ciloleucel Yescarta® Kite Second line treatment of adults with R/R large B-cell lymphoma April 2022

 

Specialty Pipeline
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Approval date*
toripalimab Tuoyi™ Junshi Biosciences/ Coherus In combination with gemcitabine and cisplatin for first-line treatment for patients with advanced recurrent or metastatic nasopharyngeal carcinoma/ Monotherapy for second-line or later treatment of recurrent or metastatic NPC patients with disease progression on or after platinum-containing chemotherapy IV Delayed
leniolisib N/A Pharming Activated phosphoinositide 3-kinase delta syndrome Oral March 2023
mirikizumab N/A Lilly Ulcerative colitis IV and SC April 2023
tofersen N/A Biogen Superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (SOD1-ALS Intrathecal April 2023
quizartinib N/A Daiichi Sankyo Acute myeloid leukemia Oral April 2023
SER109 (consortium of highly purified Firmicutes spores) N/A Seres Therapeutics Recurrent C difficile infection Oral April 2023
palopegteriparatide TransCon PTH™ Ascendis Hypoparathyroidism SC April 2023
pegunigalsidase alfa N/A Chiesi Global Rare Diseases/ Protalix Fabry disease IV May 2023
[Vic-] trastuzumab duocarmazine N/A Byondis HER2-positive unresectable locally advanced or metastatic breast cancer IV May 2023
nogapendekin alfa + BCG Anktiva™ ImmunityBio Bladder cancer IV May 2023
bimekizumab Bimzelx™ UCB Pharma Psoriasis SC May 2023
somatrogon N/A Pfizer/ Opko Pharmaceuticals Growth hormone SC May 2023
epcoritamab N/A Genmab A/S R/R large B-cell lymphoma (LBCL) after two or more lines of systemic therapy IV May 2023
momelotinib N/A Sierra Oncology, Inc. Myelofibrosis Oral June 2023
efgartigimod N/A Argenx Generalized myasthenia gravis (gMG) SC- New formulation June 2023
ritlecitinib N/A Pfizer Alopecia areata Oral June 2023
obeticholic acid N/A Intercept Pharmaceuticals Nonalcoholic steatohepatitis Oral June 2023
glofitamab N/A Roche Relapsed or refractory large B-cell lymphoma IV 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
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
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
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
eplontersen N/A Ionis Pharmaceuticals Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) SC December 2023

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

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