Top 15 Potential Blockbuster Drugs to Watch

In its annual Drugs to Watch report, released on January 10, market research firm Clarivate analysts identified 15 late-stage experimental treatments that are each forecast to deliver annual sales of more than $1 billion within five years. The promising advancements include a broad spectrum of therapeutic developments for rare diseases and tough-to-treat conditions, including HIV, Parkinson’s disease, Crohn’s disease, alopecia, multiple myeloma and breast cancer.

“Despite the many urgent challenges life sciences companies will face in 2023 — from patent cliffs to capital investment — the industry is on the cusp of unlocking revolutionary technologies that could greatly advance human health,” Clarivate said in a news release.

The top 15 potential blockbuster treatments include:

1. and 2. lecanemab (Leqembi, Eisai and Biogen), approved by the FDA on January 6, and Eli Lilly’s donanemab, both for early-stage Alzheimer’s disease. Donanemab and others in the class such as Roche’s gantenerumab may also soon achieve FDA approval, pending the results of ongoing trials, Clarivate said.

Meher Baba Kumar Nakka

However, while lecanemab’s approval “represents a major commercial and clinical milestone in AD market, we assume limited uptake until the FDA grants or refutes full/traditional (expected in the second half of 2023) approval based on its phase 3 data,” Meher Baba Kumar Nakka, senior healthcare research & data analyst at Clarivate, told Formulary Watch.

It also remains to be seen if CMS grants full Medicare coverage for the drug, “which we assume will be crucial for drug’s commercial success (i.e., not restricted coverage under CED with prospective registry-based comparative studies),” Baba Kumar Nakka said.

Related:FDA Approves Alzheimer’s Medication Lecanemab

3. Bimekizumab (Bimzelx, UCB) is the first dual IL-17 A/F inhibitor to treat moderate-to-severe plaque psoriasis. “Phase 3 trial results showed superior skin clearance outcomes than existing treatments. Its less-frequent dosing schedule and good safety profile will likely be attractive to clinicians and patients,” Clarivate said.

4. Capivasertib (AstraZeneca). For patients with breast cancer, capivasertib is a novel, highly potent, selective ATP-competitive pan-AKT kinase inhibitor. “Positive data have emerged from early-phase trials, with clinical benefit to patients irrespective of their PIK3CA/AKT1/PTEN mutational status, and several phase 3 trials are now under way,” according to Clarivate. It is expected to launch in 2023.

5. Daprodustat (Duvroq, GSK) belongs to a novel class of oral treatments for chronic kidney disease (CKD)-related anemia and is a HIF-PHI developed to treat anemia associated with CKD, which has a high incidence rate and few effective, safe treatment options, according to Clarivate. "Already available for CKD-related anemia in Japan, its uptake has been impressive,” the research firm said. The PFUDA date is Feb. 1, 2023.

6. Deucravacitinib (Sotyktu, Bristol Myers Squibb) is a first-in-class oral, targeted agent that selectively inhibits tyrosine kinase 2 (TYK2) and a Janus kinase (JAK) family member that mediates cytokine-driven immune and inflammatory signals. “It has the potential to fill a gap in the treatment armamentarium for plaque psoriasis,” Clarivate said. This received approval in September 2022.

7. Foscarbidopa/foslevodopa (AbbVie) is a novel reformulation of the gold-standard Parkinson’s disease treatment (carbidopa/levodopa) 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,” Clarivate said. This is expected to launch in 2023.

8. Lenacapavir (Sunlenca, Gilead Sciences) is a first-in-class, long-acting HIV-1 capsid inhibitor approved to treat multi-drug resistant (MDR) HIV in people who have been heavily treated — a patient population with unmet medical need. Already approved in Europe, the drug is under evaluation by the FDA. This is expected to launch in 2023.

9. Mirikizumab (Eli Lilly) is a monoclonal antibody that will likely be first-in-class for ulcerative colitis and the third in the class approved for Crohn’s disease. “Part of a set of emerging therapies with novel mechanisms of action, it will contribute to the growing market share held by these therapies,” Clarivate said. This is expected to be launched first for ulcerative colitis in 2023; the Crohn’s disease launch expected in 2025.

10. Pegcetacoplan (Empaveli, Apellis Pharmaceuticals) for paroxysmal nocturnal hemoglobinuria (PNH), a rare hematological disease, has already launched in the United States and Europe. It is expected to be the first drug to launch for geographic atrophy (GA) or dry late age-related macular degeneration (AMD), which has no approved pharmacotherapy, according to Clarivate. Its PDUFA date for geographic atrophy is February 2023.

11. Ritlecitinib (Pfizer), which stimulate hair growth in alopecia, will “likely benefit from its first-in-class status, rapid onset of action and expected label for both adults and adolescents,” Clarivate said. This is expected to launch in 2023.

12. Sparsentan (Travere Therapeutics) 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, associated with progression of kidney disease. “Its development for IgA nephropathy and focal segmental glomerulosclerosis (FSGS) promises to halt that progression for many patients and fills a gap in the treatment armamentarium,” Clarivate said. The PDUFA date is Feb. 17, 2023.

13. Teclistamab (Tecvayli, Janssen) is a first-in-class bispecific antibody targeted to B-cell maturation antigen (BCMA) to treat multiple myeloma. Ongoing phase 3 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, Clarivate said. It was granted accelerated approval in October 2022.

14. Teplizumab (Tzield, Provention Bio) is the first immunotherapy to launch for type 1 diabetes. It is a “landmark drug given its potential ability to preserve beta cell function and delay the need for insulin treatment in those with T1DM,” Clarivate said. It was approved in November 2022.

15. Valoctocogene roxaparvovec (Roctavian, BioMarin Pharmaceutical) is poised to be the first gene therapy to launch in the United States 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 (FVIII) and negate the use of otherwise burdensome prophylaxis treatment,” Clarivate said. The PDUFA date is March 31, 2023.