Blockbuster Watchlist: Which New Drugs Have the Best Potential?By
What are the blockbusters of the future? A recent industry analysis points to the leading contenders from late-stage or newly approved drugs that are slated to launch in 2023 and that are poised for potential blockbuster status over the next five years. Which drugs are the leading contenders?
A recent report, Drugs to Watch, released by Clarivate Analytics earlier this year (January 2023) identifies 15 drugs expected to enter the market or launch key indications in 2023 that are projected to achieve blockbuster status (defined as sales of $1 billion or more) by 2027. See below for the drugs that are projected as potential blockbusters.
Bimzelx (bimekizumab) by UCB. Bimekizumab is the first dual IL-17 A/F inhibitor to treat moderate-to-severe plaque psoriasis. It was approved in the European Union (EU) in August 2021 for that indication. The Clarivate analysis points out that Phase III results showed superior skin clearance outcomes than existing treatments and that its less-frequent dosing schedule and good safety profile will likely be attractive to clinicians and patients.
Late last month (April 2023), the Committee for Medicinal Products for Human Use of the European Medicines Agency issued positive opinions recommending granting marketing authorization for bimekizumab in the EU for two additional indications: active axial spondyloarthritis and active psoriatic arthritis. A final review for those two indications is pending by the European Commission.
In the US, in November 2022, UCB announced resubmission to the US Food and Drug Administration (FDA) of its biologics license application for bimekizumab for the treatment of adults with moderate-to-severe plaque psoriasis, following receipt of a FDA Complete Response Letter in May 2022. In late December 2022, the company reported that the FDA had accepted the resubmission and that FDA review/action is expected in the second quarter of 2023.
Capivasertib by AstraZeneca. Capivasertib is a potential new treatment for breast cancer. It is an investigational oral treatment, currently in Phase III trials, for the treatment of multiple subtypes of breast cancer, prostate cancer, and a Phase II trial for hematologic malignancies. A potent, selective adenosine triphosphate (ATP)-competitive inhibitor of all three AKT isoforms (AKT1/2/3), capivasertib is being evaluated as a monotherapy and in combination with existing therapies in tumors harboring alterations in the AKT pathway (PI3K/AKT/PTEN), and in tumors reliant on signaling via this pathway for survival, according to information from AstraZeneca.
In its analysis, Clarivate notes that positive data have emerged from early-phase trials, with clinical benefit to patients irrespective of their PIK3CA/AKT1/PTEN mutational status.
Jesduvroq (daprodustat) by GSK. Daprodustat belongs to a novel class of oral treatments for chronic kidney disease (CKD)-related anemia and is a hypoxia-inducible factor–prolyl hydroxylase inhibitor, a promising class of drugs to treat CKD-related anemia, which has a high incidence rate and few effective, safe treatment options. Already available for CKD-related anemia in Japan, the Clarivate analysis points out that its uptake has been impressive. The drug was approved by the FDA in February (February 2023).
Sotyktu (deucravacitinib) by Bristol-Myers Squibb. Deucravacitinib is a drug to treat moderate-to-severe plaque psoriasis. Specifically, it is a first-in-class, oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor for treating adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. It was approved by the FDA in September 2022 and by the European Commission in March 2023. It mediates cytokine-driven immune and inflammatory signals and has the potential to fill a gap in the treatment for plaque psoriasis, according to the Clarivate analysis.
Foscarbidopa/foslevodopa by AbbVie. Foscarbidopa/foslevodopa is a novel reformulation of the gold-standard Parkinson’s disease treatment, carbidopa/levodopa, but delivered via a subcutaneous pump for the treatment of motor fluctuations in advanced Parkinson’s disease. In addition to serving a niche group of patients with high unmet need, it offers better efficacy than orally administered carbidopa-levodopa, dosing flexibility, and a more convenient pump than existing and upcoming competitors, according to the Clarivate analysis.
In March (March 2023), AbbVie announced it had received a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) for its new drug application (NDA) for foscarbidopa/foslevodopa for the treatment of motor fluctuations in adults with advanced Parkinson’s disease. In the CRL, the FDA requested additional information about the device (pump) as part of the NDA review. AbbVie says the CRL does not request that AbbVie conduct additional efficacy and safety trials related to the drug. AbbVie said it plans to resubmit the NDA as soon as possible (as reported on March 22, 2023).
Leqembi (lecanemab) by Eisai and Biogen and donanemaband by Eli Lilly and Company. Eisai’s/Biogen’s Leqembi (lecanemab) and Lilly’s donanemab are two drugs for treating early-stage Alzheimer’s disease. Supported by clinical data from a Phase III trial, Eisai’s/Biogen’s Leqembi (lecanemab) received accelerated approval by the US Food and Drug Administration (FDA) for treating Alzheimer’s disease in January (January 2023). FDA provides accelerated approval for drugs for serious conditions where there is an unmet medical need and a drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. The accelerated approval of Leqembi was based on the observed reduction of amyloid beta plaque, a marker of Alzheimer’s disease.
Earlier this month (May 2023), Lilly reported positive results for donanemab in slowing cognitive and functional decline in people with early symptomatic Alzheimer’s disease. Based on these results, Lilly said in a May 3, 2023, press release that it will proceed with global regulatory submissions as quickly as possible and anticipates making a submission to FDA this quarter (second quarter 2023) for consideration under the traditional approval pathway.
Sunlenca (lenacapavir) by Gilead Sciences. Lenacapavir is a first-in-class, long-acting HIV capsid inhibitor approved in the US, the UK, Canada, and the EU for the treatment of HIV infection, in combination with other antiretroviral(s), in people with multi-drug-resistant HIV who are heavily treatment-experienced. It is also currently being investigated to treat HIV and for pre-exposure prophylaxis. Its infrequent dosing and self-administration will likely make it a favored choice in a population with treatment adherence challenges, according to the Clarivate analysis.
Mirikizumab by Eli Lilly and Company. Mirikizumab, a monoclonal antibody targeting the p19 subunit of IL-23, will likely be a first-in-class treatment for ulcerative colitis (UC) and a third entry in this class of drugs approved for Crohn’s disease, according to the Clarivate analysis. “Part of a set of emerging therapies with novel mechanisms of action, it will contribute to the growing market share held by these therapies,” notes the analysis.
Lilly recently received approval for mirikizumab as a first-in-class treatment for adults with moderately to severely active UC in Japan. In addition, last month (April 2023), the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a positive opinion for mirikizumab as a first-in-class treatment for adults with moderately to severely active UC who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic treatment. Regulatory decisions are anticipated in additional markets around the world in 2023, according to Lilly.
Empaveli/Aspaveli /APL-2 (pegcetacoplan) by Apellis Pharmaceuticals. Pegcetacoplan has already launched in the US and Europe for paroxysmal nocturnal hemoglobinuria, a rare hematological disease. As one of the few drugs to have completed Phase II trials for geographic atrophy, a chronic progressive degeneration of the macula as part of late-stage age-related macular degeneration, it is expected to be the first drug to launch for geographic atrophy or “dry late age-related macular degeneration,” which has no approved pharmacotherapy, according to the Clarivate analysis.
Ritlecitinib by Pfizer. Ritlecitinib will likely benefit from its first-in-class status, rapid onset of action, and expected label for both adults and adolescents, potentially providing an effective option to stimulate hair growth in a stigmatizing disease, alopecia areata, according to the Clarivate analysis.
Pfizer reported in September 2022 that both the FDA and the EMA accepted its application for the drug. The FDA is expected to make a decision in the second-quarter 2023. The European Medicines Agency’s decision is anticipated in the fourth-quarter 2023.
Sparsentan developed by Travere Therapeutics. Sparsentan is a first-in-class, orally active, single molecule that functions as a high-affinity, dual-acting antagonist of both endothelin type A (ETA) and angiotensin II subtype 1 (AT1) receptors, which are associated with progression of kidney disease. Its development for IgA nephropathy, a kidney disease that occurs when an antibody called immunoglobulin A (IgA) builds up in the kidneys, and focal segmental glomerulosclerosis, scar tissue in the filtering unit of the kidneys, makes it a promising in filling a gap in current treatments, according to the Clarivate analyis. The drug received accelerated approval by the FDA in February (February 2023).
Tecvayli (teclistamab) by Johnson & Johnson. After receiving conditional approval from the European Commission, teclistamab is the first-in-class bispecific antibody targeted to B-cell maturation antigen (BCMA) to treat multiple myeloma. Ongoing Phase III trials are expected to provide confirmation of clinical benefit in teclistamab’s approved setting and lead to label expansions in other multiple myeloma patient populations, including in combination with other approved agents, according to the Clarivate analysis. The drug was approved by the FDA in October 2022.
Tzield (teplizumab) by Provention Bio/Sanofi. Teplizumab is the first immunotherapy to launch for Type I diabetes mellitus and is a landmark drug given its potential ability to preserve beta cell function and delay the need for insulin treatment in those with Type 1 diabetes mellitus, according to the Clarivate analysis. Sanofi gained the drug through its $2.9-billion acquisition of Provention Bio last month (April 2023).
The drug was approved by the FDA in 2022 for delaying the onset of Stage 3 Type 1 diabetes (T1D) in adults and pediatric patients aged eight years and older with Stage 2 T1D. Tzield is also in late-stage clinical development for the treatment of pediatric and adolescent patients that are newly diagnosed with clinical T1D (Stage 3). A Phase III trial is currently underway and top-line results are expected in the second half of 2023. Additional opportunities for the drug include re-dosing and formulation as well as new therapeutic indications, according to information from Sanofi.
Roctavian ( valoctocogene roxaparvovec) by BioMarin Pharmaceutical. Approved by the European Commission in August 2022, valoctocogene roxaparvovec is also poised to be the first gene therapy to launch in the US for severe hemophilia A. Treatment benefit is expected to last for years, reduce the number of bleeding events, minimize the need for replacement factor VIII, and negate the use of otherwise burdensome prophylaxis treatment, according to the Clarivate analysis.