[Photo by Karolina Grabowska via Pexels]
Here, we survey 25 potential blockbusters that have either recently won regulatory approval are poised to do so in the near future:
| Drug name | Brand name | Developer(s) | Description | |
| 1. | Adagrasib | Krazati | Mirati Therapeutics |
The KRASG12C inhibitor adagrasib won Breakthrough Therapy Designation for patients with advanced non-small cell lung cancer (NSCLC) with the KRASG12C mutation in 2021. In early January 2023, a Phase 1/2 study of the drug was published in NEJM. The drug candidate appeared to be effective against colorectal cancer with mutated KRAS G12C as oral monotherapy and in combination with cetuximab.
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| 2. | Aprocitentan | Johnson & Johnson |
The investigational antihypertensive drug aprocitentan from Johnson & Johnson significantly lowered blood pressure (BP) when used in conjunction with background antihypertensive therapy in the Phase 3 PRECISION study.
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| 3. | Bimekizumab | Bimzelx | UCB |
Bimekizumab is a dual IL-17 A/F inhibitor for moderate to severe plaque psoriasis. In the BE VIVID and BE READY Phase 3 studies, patients who received bimekizumab had superior skin clearance compared to those who received placebo or Janssen’s Stelara (ustekinumab).
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Capivasertib (AZD5363)
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AstraZeneca/Astex Therapeutics |
In high-level results from a Phase 3 CAPItello-291 study, capivasertib with Faslodex (fulvestrant) showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) in breast cancer versus placebo plus Faslodex. The CAPItello-291 study focused on patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-low or negative locally advanced or metastatic breast cancer. Patients in the study had prior recurrence or progression on or after endocrine therapy.
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| 5. | Etranacogene | Hemgenix | CSL Behring |
Etranacogene dezaparvovec is an adenovirus-associated virus–based gene therapy for adults with hemophilia B. FDA approved the drug in late 2022. The price tag for the gene therapy, which is administered as a single infusion, is $3.5 million.
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| 6. | Daprodustat | Duvroq in Japan | GSK |
Daprodustat, also known as GSK1278863, is an oral therapy for chronic kidney disease-related anemia. In an open-label Phase 3 trial summarized in NEJM, daprodustat was noninferior to darbepoetin alfa in terms of change in hemoglobin level from baseline and cardiovascular outcomes in patients with chronic kidney disease-related anemia. NEJM summarized results from Phase 3 studies focused on treating patients with anemia who are undergoing and not undergoing dialysis.
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| 7. | Deucravacitinib | Sotyku | Bristol Myers Squibb |
Deucravacitinib, also known as BMS-986165, would be a novel daily, oral, targeted drug that selectively inhibits tyrosine kinase 2 (TYK2) to treat plaque psoriasis. In April 2021, BMS revealed positive data from two pivotal Phase 3 studies focused on deucravacitinib for moderate to severe plaque psoriasis. A significant number of plaque psoriasis patients are not candidates for injections, which could drive demand for this oral option.
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| 8. | Donanemab | Eli Lilly |
Also known as LY3002813, donanemab could be another promising monoclonal antibody for treating early-stage Alzheimer’s. While a pivotal Phase 3 trial is still ongoing, donanemab appears to have higher efficacy at reducing amyloid plaques than aducanumab in the TRAILBLAZER-ALZ 4 study.
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| 9. |
Foscarbidopa/foslevodopa
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AbbVie |
Foscarbidopa/foslevodopa (ABBV-951) from AbbVie is a solution of prodrugs for continuous subcutaneous delivery for patients with advanced Parkinson’s who have motor fluctuations. The drug candidate would be administered with a subcutaneous pump. The novel formulation could offer improved efficacy than orally administered carbidopa-levodopa.
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| 10. | Lebrikizumab | Eli Lilly |
Topical lebrikizumab is a promising interleukin 13 inhibitor that supported durable skin clearance and reduced itching in a subset of patients with atopic dermatitis in Phase 3 studies. In early 2023, Lilly announced that adults and adolescents with moderate to severe atopic dermatitis who received lebrikizumab and topical steroids had significant improvements in symptoms in the 16-week Phase 3 ADhere trial. The original developer of the drug, Roche, initially tested the drug candidate as an asthma therapy.
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| 11. | Lecanemab | Leqembi | Eisai/Biogen |
In early January 2023, FDA granted accelerated approval to Leqembi (lecanemab), the amyloid beta-protein inhibitor from Eisai and Biogen. Eisai set the an annual launch price of $26,500 for the drug. The drug is poised to outshine Aduhelm (aducanumab), which was also developed by Biogen and Eisai, given its lower price and superior Phase 3 efficacy data.
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| 12. | Lenacapavir | Sunlenca | Gilead Sciences |
Gilead Sciences’ capsid protein inhibitor lenacapavir (Sunlenca/GS-6207) won Breakthrough Therapy Designation from FDA in 2019 for HIV-1-infected individuals who are heavily treatment-experienced and resistant to multiple drugs. The drug is now approved in Europe. In mid-2022, Gilead resubmitted the application to FDA for the twice-yearly HIV drug. The company is also investigating its use to treat HIV and for pre-exposure prophylaxis (PrEP).
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| 13. | Mavacamten | Camzyos | Bristol Myers Squibb/MyoKardia |
In August 2022, mavacamten won FDA approval for adults with symptomatic New York Heart Association Class II-III Obstructive Hypertrophic Cardiomyopathy (HCM). Mavacamten is the only cardiac myosin inhibitor specifically targeting the source of obstructive HCM to win FDA approval. The drug could generate peak annual sales topping $4 billion by 2029.
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| 14. | Mirikizumab | Eli Lilly |
The interleukin-23 subunit p19 inhibitor mirikizumab could be a competitive offering for ulcerative colitis (UC) and Crohn’s disease. In 2021, mirikizumab met its primary endpoint of clinical remission in UC at 52 weeks in the Phase 3 LUCENT-2 study.
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| 15. | Nirsevimab | Beyfortus | AstraZeneca/Sanofi |
In a Phase 3 study, the recombinant human IgG1 kappa monoclonal antibody nirsevimab had 75% efficacy against lower respiratory tract infections resulting from lower respiratory tract infections stemming from RSV. The treatment landscape for lower respiratory tract infections associated with RSV is currently limited. Data from a Phase 3 trial of nirsevimab was published in 2022 in NEJM.
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| 16. | Pegcetacoplan | Empaveli | Apellis Pharmaceuticals |
Pegcetacoplan (Empaveli/Aspaveli/APL-2) is a drug for paroxysmal nocturnal hemoglobinuria (PNH). Its developer, Apellis Pharmaceuticals, also aims to win approval to use intravitreal pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
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| 17. | Ritlecitinib | Pfizer |
Also known as PF-06651600, ritlecitinib could help patients with alopecia areata regrow hair. Ritlecitinib would be a daily oral highly selective kinase inhibitor that targets both the TEC family of tyrosine kinases and Janus kinase 3 (JAK3). In September 2022, the drug’s developer, Pfizer, announced that FDA and EMA had both accepted regulatory submission for patients 12 and older with alopecia areata.
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| 18. | Roflumilast | Daxas, Daliresp, Zoryve, | Arcutis Biotherapeutics |
The topical cream version of the phosphodiesterase-4 (PDE4) inhibitor roflumilast for plaque psoriasis won FDA approval for plaque psoriasis. The drug is the second PDE4 inhibitor to hit the market, following oral Otezla (apremilast) from Amgen. An oral form of the drug won FDA approval in 2011 to reduce the frequency of exacerbations or worsening symptoms associated with severe chronic obstructive pulmonary disease.
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| 19. | RSVpreF | Pfizer |
The bivalent respiratory syncytial virus (RSV) prefusion vaccine candidate RSVpreF won Breakthrough Therapy Designation from the FDA to prevent RSV-associated lower respiratory tract illness in infants from birth up to six months of age via immunization of pregnant women. In November 2022, Pizer announced positive topline data from a Phase 3 clinical trial investigating the vaccine candidate.
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| 20. | Sparsentan | Travere Therapeutics |
Sparsentan would be a novel, oral small-molecule therapy that holds promise for IgA nephropathy and focal segmental glomerulosclerosis — two types of kidney disease. The drug candidate is a dual endothelin angiotensin receptor antagonist that targets the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor. The PDUFA target action date for sparsentan is February 17, 2023. In 2022, FDA asked the developer to update its proposed Risk Evaluation Mitigation Strategy (REMS) to include liver monitoring for the drug candidate in line with other approved endothelin receptor antagonists.
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| 21. | Teclistamab | Tecvayli | Johnson & Johnson |
Also known as JNJ-64007957, teclistamab is a novel bispecific antibody that targets the B-cell maturation antigen (BCMA) to treat multiple myeloma. Teclistamab was profiled in 2022 in NEJM. In a Phase 1/2 study, teclistamab resulted in a “high rate of deep and durable response” in patients with triple-class–exposed relapsed or refractory multiple myeloma. The drug candidate has received conditional approval from the European Commission.
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| 22. | Teplizumab | Tzield | Provention Bio |
In November 2022, the CD3 antigen inhibitor Tzield (teplizumab-mzwv) won FDA approval to delay stage 3 type 1 diabetes in individuals at least 8 years old. A vial of the drug will have a wholesale cost of $13,850. A course of treatment will cost $193,900 for average patients.
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| 23. | Tirzepatide | Mounjaro | Eli Lilly |
It’s long been clear that Lilly’s tirzepatide had blockbuster potential. Clinical trials showed that the drug supported the reduction of hemoglobin A1C (HbA1c) in people with type 2 diabetes. The drug is likely to win an FDA indication to support weight loss in individuals who are overweight or obese. In the Phase 3 SURMOUNT-1 study, tirzepatide supported up to 22.5% weight loss in adults who were overweight or obese.
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| 24. | Ublituximab | Briumvi | TG Therapeutics |
In late 2022, anti-CD20 therapy ublituximab won FDA approval for relapsing multiple sclerosis. In particular, adult patients with clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease, will be eligible for the therapy. TG Therapeutics anticipates that ublituximab will hit the market in the first quarter of 2023. After patients receive a starting dose of ublituximab, follow-up doses can be administered in a one-hour infusion two times per year.
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| 25. | Valoctocogene roxaparvovec | Roctavian | BioMarin Pharmaceutical |
If approved, valoctocogene roxaparvovec would be the first gene therapy for severe hemophilia A in the U.S. After hitting multiple roadblocks, the gene therapy won conditional marketing authorization from the European Commission. The gene therapy could be inching toward approval in the U.S. as well. In late November, BioMarin Pharmaceutical (Nasdaq:BMRN) said FDA’s review of the Biologics License Application (BLA) of Roctavian (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A had progressed. FDA also notified BioMarin that it no longer planned to hold an advisory committee meeting to discuss the application for valoctocogene roxaparvovec.
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Filed Under: Drug Discovery and Development
