Specialty Pipeline Quarterly Update: December 2022

Critical updates in an ever-changing environment

December 29, 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

  • Tzield® (teplizumab-mzwv): The U.S. Food and Drug Administration (FDA) approved Provention Bio’s Tzield injection to delay the onset of stage 3 type 1 diabetes in adults and pediatric patients 8 years and older who currently have stage 2 type 1 diabetes. Tzield is administered by intravenous (IV) infusion once daily for 14 consecutive days. The efficacy of Tzield was evaluated in a Phase 2, randomized, double-blind, event-driven, placebo-controlled trial in 76 patients with stage 2 type 1 diabetes. The primary measure of efficacy was the time from randomization to development of stage 3 type 1 diabetes diagnosis. The trial results showed that over a median follow-up of 51 months, 45% of the 44 patients who received Tzield were later diagnosed with stage 3 type 1 diabetes, compared to 72% of the 32 patients who received a placebo. The mid-range time from randomization to stage 3 type 1 diabetes diagnosis was 50 months for the patients who received Tzield and 25 months for those who received a placebo.1In 2021 the Endocrinologic and Metabolic Drugs Advisory Committee voted 10-7 that the benefits of teplizumab outweighed its risks, with many panelists “struggling” to come down on one side or the other. Panel members raised concerns about how small the study was—it didn’t meet its enrollment goal and wound up testing teplizumab in just 44 patients—and about the fact that the study did not follow patients after their diabetes diagnosis. Multiple panelists pointed to a higher rate of serious side effects in the 44 patients who received teplizumab, compared to the 32 who got placebo (16% versus 3%), as well as three patient deaths in the teplizumab arm compared to none in the placebo group.2 Tzield has launched with an average wholesale price (AWP) $16,620 per vial. The infused treatment will carry a list price of $13,850 per vial, which works out to $193,900 for a 14-vial (one per day for 14 days) regimen for the average-sized patient.
  • Rebyota® (fecal microbiota, live-jslm): The FDA approved Ferring’s (Rebiotix) Rebyota for the prevention of recurrence of Clostridioides difficileinfection (CDI) in individuals 18 years of age and older. It is for use after an individual has completed antibiotic treatment for recurrent CDI. In the United States, the infection is associated with 15,000-30,000 deaths annually. Rebyota is administered rectally as a single dose. Rebyota is prepared from stool donated by qualified individuals. The donors and the donated stool are tested for a panel of transmissible pathogens, however, as Rebyota is manufactured from human fecal matter, it may carry a risk of transmitting infectious agents. In clinical trials, the overall estimated rate of success in preventing recurrent CDI through eight weeks was significantly higher in the Rebyota group (70.6%) than in the placebo group (57.5%).3 Rebyota launch and price are pending.
  • Rezlidhea® (olutasidenib): Forma Therapeutics’ Rezlidhea was approved by the FDA for use in certain patients with relapsed or refractory (R/R) acute myeloid leukemia(AML). Specifically, the drug is approved for use in patients who have R/R AML with a susceptible isocitrate dehydrogenase 1 (IDH1) mutation as detected by an FDA-approved test. The FDA also approved the Abbott RealTime IDH1 Assay to select patients for treatment. Rezlidhea is an oral inhibitor of mutated IDH1 that has been designed to bind and inhibit mutated IDH1 to reduce hydroxyglutarate levels and restore cellular differentiation of myeloid cells. Rezlidhea met its primary endpoint of a composite of a complete remission (CR) plus a complete remission with partial hematological recovery (CRh) in its pivotal clinical trial. Results from the trial demonstrated a 35% CR+CRh rate in mIDH1 R/R AML patients, with a median duration of response of 25.9 months. The median time to CR or CRh was 1.9 months.4 Rezlidhia has launched with an AWP of $644 per capsule.
  • Krazati® (adagrasib): The FDA has granted accelerated approval of Mirati Therapeutics’ Krazati, the inhibitor of the RAS GTPase family as oral monotherapy for second-line treatment of advanced non-small cell lung cancer (NSCLC) patients harboring the KRAS G12C mutation following at least one prior systemic therapy. Krazati was approved based on the Phase 2 cohort KRYSTAL-1 study which demonstrated an objective response rate (ORR) of 43% with 80% of patients achieving disease control. The median duration of response (DOR) was 8.5 months.5 Krazati launch and price are pending.

New Indications

  • Rylaze® (asparaginase erwinia chrysanthemi (recombinant)- rywn): The FDA approved Jazz Pharmaceuticals’ Rylaze new dosing regimen for the asparagine specific enzyme for administration on Monday, Wednesday and Friday as component of a multi-agent chemotherapy regimen for treatment of 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.
  • Imjudo® (tremelimumab-actl): AstraZeneca’s Imjudo has been approved by the FDA to expand its indication for use in combination with AZ’s PD-L1 inhibitor durvalumab (Imfinzi) and platinum-based chemotherapy for treatment of adults with metastatic non-small cell lung cancer (NSCLC) with no sensitizing EGFR mutation or anaplastic lymphoma kinase (ALK) genomic tumor aberrations.
  • Imfinzi® (durvalumab): AstraZeneca’s Imfinzi has been approved by the FDA to include the treatment of adults with metastatic non-small cell lung cancer (NSCLC) with no sensitizing EGFR mutation or anaplastic lymphoma kinase (ALK) genomic tumor aberrations.
  • Tecentriq® (atezolizumab): Genentech’s Tecentriq received FDA approval for adult and pediatric patients 2 years of age and older with unresectable or metastatic alveolar soft part sarcoma.

December News

  • “Johnson & Johnson unit Janssen sued Amgen over the company’s proposed biosimilar to its megablockbuster Stelara (ustekinumab), after Amgen said it was ready to launch next May or as soon as the FDA signs off on it. If Amgen carries through with that plan, Janssen told the Delaware district court that the Thousand Oaks, CA-based company will infringe on at least two Janssen patents. The suit follows Amgen’s announcement in April that preliminary results from a Phase III study evaluating the efficacy and safety of its ustekinumab biosimilar, known as ABP 654, met its primary efficacy endpoint in adult patients with moderate-to-severe plaque psoriasis, demonstrating no clinically meaningful differences between ABP 654 and Stelara.”6
  • “After more than a decade and a half in development, the FDA’s Cardiovascular and Renal Drugs Advisory Committee today voted 8-3 against approval of Cytokinetics’ heart failure drug. Adcomm members said the drug’s potential benefits did not seem to outweigh its risks, with panel members raising concerns about the drug’s safety and its limited efficacy. The drug, known as omecamtiv mecarbil (OM), has seen mixed clinical results, with a first Phase 3 readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.”7
  • “BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration (FDA) review of the Biologics License Application (BLA) of ROCTAVIAN™ (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review.  Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date.”8
  • “Looking back, 2022 brought two key successful Phase 3 trials. In September, Eisai and Biogen announced lecanemab met its primary endpoint of slowing the rate of cognitive decline in a 1,906-patient Phase 3 study. Just last week, Anavex announced blarcamesine met its primary endpoint of slowing decline in a 509-patient Phase 2b/3 trial. However, 2022 was also full of bumps and controversies. In November, Roche announced its antibody, gantenerumab, missed the mark in the Phase 3 GRADUATE-1 and GRADUATE-2 studies. Meanwhile, Medicare announced in April it would severely restrict coverage of Aduhelm, shattering the sales prospects of what analysts once believed to be a multi-billion dollar drug. And over the summer, a report suggested data manipulation in studies backing the amyloid theory behind most current AD therapies. As sponsors work to make sense of 2022, here’s a look at three major questions that could shape AD drug development in 2023. Will Lilly’s donanemab break through? Will AD trials become more inclusive? Will there be new approaches to AD drug development?”9

References

  1. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-can-delay-onset-type-1-diabetes
  2. https://www.ajmc.com/view/fda-panel-supports-approving-teplizumab-to-delay-type-1-diabetes
  3. https://www.statnews.com/2022/11/30/fda-approves-first-microbiome-drug/
  4. https://www.rigel.com/investors/news-events/press-releases/detail/349/rigel-announces-u-s-fda-approval-of-rezlidhia
  5. https://www.prnewswire.com/news-releases/mirati-therapeutics-announces-us-fda-accelerated-approval-of-krazati-adagrasib-as-a-targeted-treatment-option-for-patients-with-locally-advanced-or-metastatic-non-small-cell-lung-cancer-nsclc-with-a-krasg12c-mutation-301700902.html
  6. https://endpts.com/protecting-its-megablockbuster-janssen-challenges-amgens-stelara-biosimilar-ahead-of-planned-2023-launch/
  7. https://endpts.com/fda-adcomm-votes-8-3-against-cytokinetics-potential-heart-failure-drug/
  8. https://investors.biomarin.com/2022-11-23-BioMarin-Announces-Advancements-in-FDA-Review-of-ROCTAVIAN-TM-Valoctocogene-Roxaparvovec-for-Adults-with-Severe-Hemophilia-A
  9. https://www.clinicaltrialsarena.com/features/alzheimers-2023/
SPECIALTY NEW PRODUCT APPROVALS IN THE PAST TWELVE MONTHS
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved
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 and 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
ganaxolone Ztalmy™ Marinus Pharmaceuticals, Inc. Seizures associated with CDKL5 deficiency disorder Oral March 2022
sirolimus topical gel Hyftor® Nobelpharma Facial angiofibroma Gel 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-ekko Tezspire® AstraZeneca and Amgen Asthma across phenotypes SC January 2022

 

NEW INDICATIONS FOR APPROVED SPECIALTY PRODUCTS
Generic Name Brand Name Manufacturer New Indication(s) Date Approved
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 and 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
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
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 6 months to 5 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 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 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 3 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

 

ONCOLOGY PRODUCT APPROVALS IN THE PAST TWELVE MONTHS
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Date Approved
adagrasib Krazati® Mirati Therapeutics Second-line treatment of advanced non-small cell lung cancer (NSCLC) Oral December 2022
olutasidenib Rezlidhea® Forma and 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
lutetium Lu 177 vipivotide tetraxetan Pluvicto® Novartis Prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) IV 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
NEW INDICATIONS FOR APPROVED ONCOLOGY DRUGS
Generic Name Brand Name Manufacturer New Indication Date Approved
atezolizumab Tecentriq® Genentech Alveolar soft part sarcoma 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 Metastatic non-small cell lung cancer (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 non-small cell lung cancer (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 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 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 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 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
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
olaparib Lynparza® AstraZeneca and Merck BRCA-mutated, HER2-negative high-risk early breast cancer who have already been treated with chemotherapy either before or after surgery March 2022
nivolumab Opdivo® Bristol Myers Squibb Combination with chemotherapy for adult patients with non-small cell lung cancer (NSCLC) in the neoadjuvant setting March 2022
SPECIALTY PIPELINE
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Approval date*
tislelizumab N/A BeiGene and Novartis Certain forms of esophageal squamous cell carcinoma (ESCC) IV Delayed
miglustat (for in combination with cipaglucosidase alfa) N/A Amicus Therapeutics Pompe disease Oral Delayed
cipaglucosidase alfa (in combination with miglustat) N/A Amicus Therapeutics Pompe disease IV Delayed
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
palovarotene Sohonos™ Ipsen (Clementia) Fibrodysplasia ossificans progressiva (FOP) Oral December 2022
mosunetuzumab N/A Genentech (Roche) R/R follicular lymphoma (FL) IV December 2022
mirikizumab N/A Lilly Ulcerative colitis IV and SC 1Q2023
lebrikizumab N/A Lilly Atopic dermatitis SC 1H2023
NexoBrid N/A MediWound Burn tissue Topical gel January 2023
ACER-001 (sodium phenylbutyrate) N/A Acer Therapeutics and Relief Therapeutics Urea cycle disorders Oral January 2023
lecanemab N/A Eisai Alzheimer’s disease IV January 2023
sparsentan N/A Travere Therapeutics  IgA nephropathy (IgAN) Oral February 2023
pegcetacoplan N/A Apellis Pharmaceuticals Geographic atrophy secondary to AMD Intravitreal 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 Lamazede™ Chiesi Global Rare Diseases Alpha-mannosidosis IV March 2023
trofinetide N/A Acadia Pharmaceuticals Inc. Rett syndrome Oral March 2023
leniolisib N/A Pharming Activated phosphoinositide 3-kinase delta syndrome Oral March 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
SER-109 (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
[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
momelotinib N/A Sierra Oncology, Inc. Myelofibrosis Oral June 2023
ritlecitinib N/A Pfizer Alopecia areata Oral June 2023
zilucoplan N/A UCB Generalized myasthenia gravis SC September 2023

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

 

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