Perspectives
Specialty Drug Pipeline Quarterly Update: March 2023
March 27, 2023This 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
- https://www.sanofi.com/en/media-room/press-releases/2023/2023-02-23-21-00-00-2614759
- https://www.sanofi.com/en/media-room/press-releases/2023/2023-02-23-21-00-00-2614759
- https://www.neurologylive.com/view/fda-approves-omaveloxolone-first-treatment-friedreich-ataxia
- https://www.rettsyndrome.org/trofinetideapproved/
- https://www.fiercebiotech.com/biotech/fda-shocks-sarepta-ordering-adcomm-dmd-gene-therapy-sudden-change-heart
- 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
- https://endpts.com/fda-indicates-willingness-to-approve-biogen-als-drug-despite-failed-phiii-study/
- 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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