Specialty Pipeline Quarterly Update: SEPTEMBER 2022

Critical updates in an ever changing environment

September 30, 2022

This quarterly 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 cell/gene therapy. 

New Drug Information

  • Xenpozyme® (olipudase alfa-rpcp): The U.S. Food and Drug Administration (FDA) has approved Sanofi’s Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type A and type B) in adult and pediatric patients. ASMD is a rare, progressive, and potentially life-threatening disease that results from a deficient activity of the enzyme acid sphingomyelinase (ASM), which is found in special compartments within cells called lysosomes and is required to breakdown lipids called sphingomyelin. If ASM is absent or not functioning as it should, sphingomyelin cannot be metabolized properly and accumulates within cells, eventually causing cell death and the malfunction of major organ systems. The deficiency of the lysosomal enzyme ASM is due to disease-causing variants in the sphingomyelin phosphodiesterase 1 gene (SMPD1). The estimated prevalence of ASMD is approximately 2,000 patients in the United States, Europe, and Japan.1 Xenpozyme was approved based on the Phase 3 ASCEND trial that demonstrated significantly improved lung function and significantly reduced spleen volume for Xenpozyme-treated patients at 52 weeks. Additionally, Xenpozyme’s data remained consistent throughout the 6.5-year outcomes for five adult patients with ASMD and two-year outcomes in 20 pediatric patients, as well as the open-label extension from the Phase 3 ASCEND trial in adults. Results showed the percent predicted lung function (DLco) mean increase was 55.3%, spleen volume mean decrease was 59.5%, and liver volume mean decrease was 43.7% compared to baseline of original study.1 Xenpozyme launch and price are pending.
  • Stimufend® (pegfilgrastim-fpgk): Fresenius Kabi’s Stimufend was approved by the FDA as a pegfilgrastim biosimilar. Stimufend is indicated for use in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. Stimufend is the fifth approved pegfilgrastim biosimilar using Amgen’s Neulasta as a reference product. The company expects to launch the product in a prefilled syringe early next year and in an on-body injector following FDA approval with pricing to follow.2
  • Sotyktu® (deucravacitinib): The FDA approved BMS’ Sotyktu for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. Sotyktu is a first-in-class, oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor. Sotyktu’s approval was based on the Phase 3 POETYK PSO-1 and POETYK PSO-2 trials that evaluated the percentage of patients who achieved Psoriasis Area and Severity Index (PASI) 75 and the percentage of patients who achieved static Physician’s Global Assessment (sPGA) score of 0 to 1 with at least a two-grade improvement from baseline, compared with placebo, at week 16.
    • Findings from the POETYK PSO-1 and POETYK PSO-2 trials, respectively, included the following:
      • Patients achieving PASI 75 response at week 16: 58% and 53% for deucravacitinib vs 13% and 9% for placebo (both <.0001) and 35% (P<.0001) and 40% (P =.0004) for apremilast.3
      • Patients achieving PASI 90 response at week 16: 36% and 27% for deucravacitinib vs 4% and 3% for placebo (both <.0001) and 20% (P=.0002) and 18% (P =.0046) for apremilast.3 
      • Patients achieving sPGA 0/1 at week 16: 54% and 50% for deucravacitinib vs 7% and 9% for placebo (both <.0001) and 32% and 34% for apremilast (both <.0001).3

Sotyktu has launched with an average wholesale price of $7,397 per bottle of 30 tablets.

  • Rolvedon™ (eflapegrastim-xnst): The FDA approved Spectrum Pharmaceuticals’ Rolvedon injection to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. Rolvedon’s approval was based on two identical Phase 3 clinical trials, ADVANCE and RECOVER, which evaluated the safety and efficacy of Rolvedon in early-stage breast cancer patients for the management of neutropenia due to myelosuppressive chemotherapy. In both studies, Rolvedon demonstrated non-inferiority (NI) in mean duration of severe neutropenia (DSN) and a similar safety profile to pegfilgrastim.4 Rolvedon also demonstrated non-inferiority to pegfilgrastim in the mean DSN across all four cycles in both trials. Rolvedon is scheduled to launch fourth quarter 2022 with pricing to follow.
  • Terlivaz™ (terlipressin): The FDA approved Terlivaz for injection. Terlivaz is the first FDA-approved product indicated to improve kidney function in adults with hepatorenal syndrome (HRS) with rapid reduction in kidney function, an acute and life-threatening condition requiring hospitalization. Terlivaz was approved based on the Phase 3, CONFIRM trial which used a surrogate endpoint of renal function improvement, avoidance of dialysis and short-term survival to demonstrate that Terlivaz reversed HRS-1 in 29% of patients who were treated with Terlivaz compared to 16% treated with placebo.5 In July 2020, the FDA Advisory Committee voted 8 to 7 to recommend approval of Terlivaz. The panel suggested that the benefits of Terlivaz outweigh the safety risks, Terlivaz and placebo groups experienced serious side effects at roughly the same rate: 65% versus 61%. Terlivaz launch and price are pending.

New Indications

  • Pemazyre® (pemigatinib): The FDA expanded Incyte’s Pemazyre indication to include treatment of relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.

September News

  • “Bluebird bio has reported its financial results and business highlights for the second quarter of 2022, in which the company revealed the anticipated launch of both betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel) gene therapies in the fourth quarter of the year. The news comes after both therapies were endorsed by the FDA Cell Tissue & Gene Therapy Advisory Committee (CTGTAC) in June 2022. Beti-cel is under priority review for the treatment of people with transfusion-dependent beta-thalassaemia (beta-thal), a rare genetic blood disease caused by a gene defect that impairs the ability of red blood cells to produce hemoglobin. Patients with the most severe form of beta-thal develop life-threatening anemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks. Eli-cel is under priority review for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients under the age of 18 who do not have an available and willing matched sibling donor. CALD is a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death. Beti-cel and eli-cel have Prescription Drug User Act Fee (PDUFA) goal dates of 19 August 2022 and 16 September 2022, respectively. If approved, the company anticipates that both therapies will be available in the fourth quarter of 2022.”6
  • Amylyx Pharmaceuticals Inc. shares climbed after its controversial treatment for amyotrophic lateral sclerosis won the support of a panel of U.S. regulatory advisers, putting the product on track for likely clearance. During a rare second panel meeting on Wednesday, the FDA peripheral and central nervous system drugs advisory committee voted 7-2 to back approval of the treatment. During a meeting in March, FDA advisers found that data from Amylyx’s relatively small initial trial was insufficient to prove that AMX0035 slowed ALS progression. The negative decision put the FDA in a bind, as the agency, under pressure from disease activists, has promised to exercise flexibility in approving drugs for dire illnesses such as ALS, given the urgent need for better treatments. New data from Amylyx “is not unequivocally persuasive, but nonetheless is quite reassuring,” said Liana Apostolova of Indiana University School of Medicine, who voted in favor of the drug this time after opposing it in March. She said she took into account that ALS “is a horrific disorder, it is a death sentence.””7
  • “Apellis Pharmaceuticals, Inc., a global biopharmaceutical company and leader in complement, today announced top-line data at 24 months showing increased effects over time with intravitreal pegcetacoplan, an investigational, targeted C3 therapy, in the Phase 3 DERBY and OAKS studies in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). In a pre-specified analysis of GA lesion growth over 24 months, both monthly and every-other-month (EOM) pegcetacoplan showed a clinically meaningful reduction in GA lesion growth from baseline compared to sham (all p-values are nominal): DERBY: 19% monthly, p=0.0004; 16% EOM, p=0.0030. OAKS: 22% monthly, p<0.0001; 18% EOM, p=0.0002. Between months 18-24, the pegcetacoplan treatment effect accelerated compared to previous six-month periods, with robust reductions of GA lesion growth versus sham (all p-values are nominal). The increased effects were driven by a greater slowing of lesion growth by pegcetacoplan and not by an increase in the lesion growth rate in the sham group, which was highly consistent over each of the four six-month intervals (1.0+/-0.05 mm2).”8
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved
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  
FT218 (sodium oxybate, controlled release) Lumryz™

Tentative approval

Avadel Pharmaceuticals Narcolepsy Oral July 2022  
vutrisiran Amvuttra® Alnylam Pharmaceuticals Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis SC June 2022  
edaravone, oral 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  
ganaxolone Ztalmy™ Marinus Pharmaceuticals, Inc. Seizures associated with CDKL5 deficiency disorder Oral March 2022  
mitapivat Pyrukynd® Agios Pharmaceuticals Pyruvate kinase (PK) deficiency Oral February 2022  
sutimlimab-jome Enjaymo® Sanofi US Cold agglutinin disease (CAD) IV February 2022  
faricimab-svoa Vabysmo® Genentech Diabetic macular edema Intravitreal January 2022  
abrocitinib Cibinqo® Pfizer And Lilly Atopic dermatitis Oral January 2022  
inclisiran Leqvio® Novartis Hyperlipidemia SC January 2022  
tezepelumab Tezspire® AstraZeneca and Amgen Asthma across phenotypes SC January 2022  
tralokinumab-ldrm Adbry® Leo Pharma Inc Atopic dermatitis SC December 2021  
levoketoconazole Recorlev® Strongbridge Biopharma Cushing’s syndrome Oral December 2021  
efgartigimod alfa-fcab Vyvgart® Argenx Generalized myasthenia gravis IV December 2021  
budesonide Tarpeyo® Calliditas Therapeutics Primary IgA nephropathy (IgAN) Oral December 2021  
vosoritide Voxzogo® BioMarin Achondroplasia SC November 2021  
ropeginterferon alfa-2b-njft Besremi® PharmaEssentia Polycythemia vera (PV) SC November 2021  
treprostinil inhalation powder Yutrepia®

Tentative approval

Liquidia Pulmonary arterial hypertension Inhaled dry powder November 2021  
ranibizumab via ocular implant Susvimo® Genentech Age-related macular degeneration Intravitreal injection October 2021  
triamcinolone acetonide Xipere® Clearside Biomedical and Bausch + Lomb (Bausch Health) Macular edema associated with uveitis Suprachoroidal injection October 2021  
avacopan Tavneos® Chemocentryx ANCA-associated vasculitis Oral October 2021  
allogeneic processed thymus tissue-agdc Rethymic® Enzyvant (Sumitomo Dainippon, Sumitovant) Certain forms of congenital athymia Surgical October 2021  




Generic Name Brand Name Manu-facturer New Indication(s) Date Approved  
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 and Regeneron For add-on maintenance treatment of pediatric patients aged six months to fve years old with moderate to severe atopic dermatitis June 2022
baricitinib Olumiant® Lilly and 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 two 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 and 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 three kilograms with positive results of direct SARS-CoV-2 viral testing April 2022
upadacitinib Rinvoq® Abbvie Moderately to severely active ulcerative colitis March 2022
tocilizumab Actemra® IV Genentech Adults with giant cell arteritis February 2022
von Willebrand factor, recombinant Vonvendi® Takeda Bleeding episodes in adults with von Willebrand disease (VWD) January 2022
risankizumab-rzaa Skyrizi® Abbvie Treatment of adults with active psoriatic arthritis January 2022
upadacitinib Rinvoq® Abbvie Adults and adolescents with moderate to severe atopic dermatitis (eczema) January 2022
remdesivir Veklury® Gilead Mild to moderate COVID-19 in adults and adolescents who are at high risk for progression to severe COVID-19, including hospitalization or death January 2022
secukinumab Cosentyx® Novartis Active enthesitis-related arthritis in four years and older, and active juvenile psoriatic arthritis (JPsA) in patients two years and older December 2021
apremilast Otezla® Amgen Mild to moderate plaque psoriasis who are candidates for phototherapy or systemic therapy December 2021
voxelotor Oxbryta® Global Blood Therapeutics, Inc Pediatric indication and new dispersible tablet for oral suspension dosage form of the sickle hemoglobin polymerization inhibitor for treatment of sickle cell disease (SCD) in patients aged four to 11 years December 2021
abatacept Orencia® BMS Prophylaxis of acute graft versus host disease (aGVHD) in combination December 2021
tofacitinib Xeljanz® Pfizer Ankylosing spondylitis (AS) December 2021
upadacitinib Rinvoq® Abbvie Psoriatic arthritis December 2021
dupilumab Dupixent® Sanofi and Regeneron Add-on treatment of pediatric patients aged 6 to 11 years of age with uncontrolled moderate-to-severe asthma October 2021
bictegravir, emtricitabine and tenofovir alafenamide Biktarvy® Gilead Expanded pediatric indication for the integrase strand transfer inhibitor (INSTI)-based single-tablet regimen and new low-dose tablet formulation for treatment of HIV-1 infected pediatric patients weighing at least 14 kg October 2021



Generic Name Brand Name Manufacturer Indication(s) Route of Administration Date Approved  
eflapegrastim-xnst Rolvedon® Spectrum Pharmaceuticals and Hanmi Pharmaceutical Chemotherapy-induced neutropenia SC 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
lutetium Lu 177 vipivotide tetraxetan Pluvicto® Novartis Prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) IV March 2022
sirolimus topical gel Hyftor® Nobelpharma Facial angiofibroma Gel March 2022
relatlimab and nivolumab Opdualag® Bristol Myers Squibb Certain forms of metastatic melanoma IV March 2022
pacritinib Vonjo® CTI BioPharma Myelofibrosis patients with severe thrombocytopenia Oral February 2022
tebentafusp-tebn Kimmtrak® Immunocore Metastatic uveal melanoma (mUM) IV January 2022
cabazitaxel N/A Accord Metastatic castration-resistant prostate cancer IV December 2021
sirolimus albumin-bound nanoparticles for injectable suspension Fyarro® Aadi Bioscience Metastatic or locally advanced malignant perivascular epithelioid cell tumor (PEComa) IV November 2021
asciminib Scemblix® Novartis Certain forms of chronic myeloid leukemia (CML) Oral October 2021



Generic Name Brand Name Manufacturer New Indication Date Approved
pemigatinib Pemazyre® Incyte R/R myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement August 2022
capmatinib Tabrecta® Novartis Certain forms of metastatic non-small cell lung cancer (NSCLC) August 2022
darolutamide Nubeqa® Bayer and Orion In combination with docetaxel for treatment of metastatic hormone-sensitive prostate cancer (mHSPC) August 2022
fam-trastuzumab deruxtecan-nxki Enhertu® Daiichi Sankyo and 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 six 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 and 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 Uresectable 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
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 2022



abemaciclib Verzenio® Lilly In combination with endocrine therapy (tamoxifen or aromatase inhibitor) for adjuvant treatment of adults with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) October 2021
brexucabtagene autoleucel Tecartus® Gilead Sciences Adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) October 2021


Generic Name Brand Name Reference


Manufacturer Indication(s) Route of Administration Month Approved
pegfilgrastim-fpgk Stimufend® Neulasta® biosimilar Fresenius Kabi Neutropenia IV or SC September 2022
adalimumab-bwwd 100mg/mL Hadlima® HC Humira® biosimilar Samsung Bioepis and Organon Autoimmune SC August 2022
ranibizumab-eqrn First interchangeable biosimilar for Lucentis Cimerli® Lucentis® biosimilar Coheres biosciences/bioeq Age-related macular degeneration Injection into the eye August 2022
pegfilgrastim-pbbk Fylnetra Neulasta® Amneal and Kashiv (Adello) Neutropenia IV or SC May 2022
bevacizumab-maly Alymsys Avastin® mAbxience, Insud Pharma; Amneal Oncology IV April 2022
filgrastim-ayow Releuko Amneal Neutropenia Blood modifying IV February 2022
insulin glargine-aglr Rezvoglar Lantus® Eli Lilly Diabetes SC December 2021
adalimumab-aqvh Yusimry Humira® Coherus Autoimmune SC December 2021
ranibizumab-nuna Byooviz Lucentis® Samsung Bioepis Age-related macular degeneration Intravitreal September 2021
insulin glargine-yfgn

First interchangeable biosimilar for Lantus

Semglee Lantus® Mylan and Biocon Diabetes SC July 2021




Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Approval date*
eflornithine and sulindac Flynpovi® Cancer Prevention Pharmaceuticals Familial adenomatous polyposis (FAP) Oral Delayed  
donislecel purified allogeneic deceased donor pancreas derived islets of Langerhans Lantidra® CellTrans Brittle type 1 diabetes IV Delayed  
SH-111 N/A Shorla Pharma Pediatric patients with T-cell leukemia IV Delayed  
inolimomab Leukotac® Farmaceutici Spa Steroid-refractory acute graft-versus-host disease IV Delayed  
efbemalenograstim alfa Ryzneuta™ Evive Biotech Chemotherapy-induced neutropenia SC Delayed  
penpulimab Annik™ Akeso and Sino Biopharmaceutical Metastatic nasopharyngeal carcinoma IV Delayed  
tislelizumab N/A BeiGene and Novartis Certain forms of esophageal squamous cell carcinoma (ESCC) IV Delayed  
miglustat (for AT-GAA combination with cipaglucosidase alfa) N/A Amicus Therapeutics Pompe disease Oral Delayed  
teclistamab N/A Johnson & Johnson (Janssen) Relapsed or refractory multiple myeloma IV or SC Delayed  
sodium phenylbutyrate and taurursodiol Albrioza™ Amylyx Pharmaceuticals, Inc. Amyotrophic lateral sclerosis (ALS) Oral September 2022  
sodium thiosulfate, STS Pedmark™ Fennec Pharmaceuticals Prevention of ototoxicity induced by cisplatin IV September 2022  
futibatinib N/A Taiho Oncology Metastatic cholangiocarcinoma Oral September 2022  
subcutaneous furosemide, sc2Wear furosemide infusor-drug/device combination Furoscix™ ScPharmaceuticals Diuresis in patients with worsening heart failure SC minipump October 2022  
tremelimumab N/A AstraZeneca Certain forms of unresectable hepatocellular carcinoma IV October 2022  
cipaglucosidase alfa (in combination with miglustat) N/A Amicus Therapeutics Pompe disease IV October 2022  
SPN-830 (apomorphine infusion pump) N/A Supernus Pharmaceuticals Parkinson’s disease SC Minipump October 2022  
teplizumab Tzield™ Provention Bio Diabetes IV November 2022  
sparsentan N/A Travere Therapeutics  IgA nephropathy (IgAN) Oral November 2022  
poziotinib N/A Spectrum Pharmaceuticals, Inc. Certain forms of metastatic non-small cell lung cancer (NSCLC) Oral November 2022  
mirvetuximab soravtansine N/A ImmunoGen Certain forms of folate receptor alpha-high platinum-resistant ovarian cancer IV November 2022  
131I-omburtamab Omblastys™ Y-mAbs Pediatric patients with central nervous system and leptomeningeal metastasis from neuroblastoma IV November 2022  
RBX2660 fecal microbiota transplant Rebyota™ Ferring (Rebiotix) Clostridioides difficile Fecal microbiome transplant November 2022  
pegcetacoplan N/A Apellis Pharmaceuticals Geographic atrophy secondary to AMD Intravitreal November 2022  
toripalimab Tuoyi™ Junshi Biosciences and 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 December 2022  
adagrasib N/A Mirati Therapeutics Second-line treatment of advanced non-small cell lung cancer (NSCLC) Oral December 2022  
palovarotene N/A Ipsen (Clementia) Fibrodysplasia ossificans progressiva (FOP) Oral December 2022  
mosunetuzumab N/A Genentech (Roche) R/R follicular lymphoma (FL) IV December 2022  
NexoBrid N/A MediWound Burn tissue Gel January 2023  
ACER-001 (sodium phenylbutyrate) N/A Acer Therapeutics and Relief Therapeutics Urea cycle disorders Oral January 2023  
tofersen N/A Biogen Superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (SOD1-ALS Intrathecal injection January 2023  
lecanemab N/A Eisai Alzheimer’s disease IV January 2023  
olutasidenib N/A Forma and Rigel Relapsed or refractory acute myeloid leukemia Oral February 2023  
elacestrant N/A Radius Health and Menarini ER+/ HER2- advanced or metastatic breast cancer Oral February 2023  
omidubicel N/A Gamida Cell Blood cancer Cell therapy February 2023  
omaveloxolone Omav™ Reata Pharmaceuticals, Inc. Friedreich’s ataxia Oral February 2023  
efanesoctocog alfa N/A Sanofi and Sobi Hemophilia A IV February 2023  
velmanase alfa N/A Chiesi Global Rare Diseases Alpha-mannosidosis IV March 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  
momelotinib N/A Sierra Oncology, Inc. Myelofibrosis Oral June 2023  
ritlecitinib N/A Pfizer Alopecia areata Oral June 2023  

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




Generic name Brand name Manufacturer Indication(s) Route of administration Anticipated approval Date*
MYL-1402O N/A (Avastin® biosimilar) Mylan NV and Biocon Ltd. Oncology IV 2022  
SB8 N/A (Avastin® biosimilar) Samsung Bioepis and Merck Oncology IV 2022  
FKB238 N/A (Avastin® biosimilar) Centus Biotherapeutics Oncology IV 2022  
BAT-1706 N/A (Avastin® biosimilar) Bio-thera solutions Oncology IV 2022  
CT-P16 N/A (Avastin® biosimilar) Celltrion Oncology IV 2022  
EG12014 N/A (Herceptin® biosimilar) Sandoz Oncology IV 4Q2022  
TX05 N/A (Herceptin® biosimilar) Tanvex Oncology IV 2022  
Blood Modifiers            
lupifil-p N/A (Neulasta® biosimilar) Lupin Neutropenia SC 2022  
lapelga N/A (Neulasta® biosimilar) Apotex/Accord Neutropenia SC 2022  
TX01 N/A (Neupogen® biosimilar) Tanvex BioPharma Neutropenia SC 2022  
grastofil N/A (Neupogen® biosimilar) Apotex/Accord Neutropenia SC 2022  
xlucane N/A (Lucentis® biosimilar) Xbrane & Bausch Age-related macular degeneration Injection into the eye 2023  
MYL-1701P N/A (Eylea® biosimilar) Mylan Age-related macular degeneration Injection into the eye 2022  
CT-P17 Yuflyma® (Humira® biosimilar) Celltrion Autoimmune SC 2022  
MSB11022 Idacio® (Humira® biosimilar) Fresenius Autoimmune SC 2022  
MSB11456 N/A (Actemra® IV/SC biosimilar) Fresenius Autoimmune IV/SC 2Q2023  


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