Thirteen potential blockbuster drugs to watch in 2024


Thirteen new-to-market therapeutics and drugs poised to launch in 2024 will achieve ‘blockbuster’ status by 2029 or deliver game-changing benefits to patients.

They include therapeutics for conditions including breast cancer, haemophilia A, sickle cell disease, Crohn’s disease, ulcerative colitis, respiratory syncytial virus (RSV) and multiple myeloma, among others.

The predictions were made by Clarivate in their annual Drugs to Watch report, which provides in-depth predictive analysis of drugs with the potential for standout commercial and/or clinical success.

2024 is anticipated to be a transformational year for the pharma industry, with new modalities like antibody drug conjugates and AI/machine learning providing therapies for patients with limited treatment options.

However, government initiatives to contain healthcare costs, the sustained high cost of capital and global geopolitical disputes are negatively impacting investor appetites for the sector.

Mike Ward, Global Head of Thought Leadership, Life Sciences and Healthcare, Clarivate said: “The fundamentals underpinning the biopharma sector have never been stronger, with new technologies fuelling medical advancements and providing treatment options to patients with previously unmet needs. Leveraging deep industry expertise and comprehensive therapeutic area differentiated data, this year’s Drugs to Watch report identifies innovative medicines based on recent scientific breakthroughs poised to have extraordinary impacts on patient outcomes.”

The 2024 Drugs to Watch, are:

1. Aflibercept (high dose; Eylea HD), Bayer and Regeneron

A drug for individuals with wet age-related macular degeneration (AMD), diabetic macular oedema (DME) or diabetic retinopathy (DR) whose treatment choices include invasive, burdensome administration that limits treatment uptake, high-dose aflibercept offers less-frequent administration while achieving similar efficacy and safety as the current standard of care.

2. Budesonide (Tarpeyo/Kinpeygo/Nefecon), Calliditas Therapeutics, Everest Medicines and STADA Arzneimittel

Tarpeyo/Kinpeygo (developed under the project name Nefecon) is a second-generation, synthetic, non-halogenated form of the corticosteroid budesonide. The delayed release formulation of budesonide has shown greater efficacy for protein reduction and slowing the decline in kidney function in primary immunoglobulin A (IgA) as well as a much better safety profile than conventional corticosteroids.

3. Datopotamab deruxtecan (Dato-DXd), AstraZeneca and Daiichi Sankyo

With the potential to become the best-in-class TROP2-targeted antibody drug conjugate (ADC), datopotamab deruxtecan is set to be second to market (after Trodelvy; Gilead Sciences) for both HR-positive/HER2-negative and triple-negative breast cancer, and to enter the non-small cell lung cancer (NSCLC) market. The therapy has shown positive results in Phase III trials in both breast and lung cancer.

4. Efanesoctocog alfa (Altuviiio/BIVV001), Sanofi (Bioverativ Therapeutics) and Swedish Orphan Biovitrum

Efanesoctocog alfa is the first once-weekly factor VIII (FVIII) replacement intravenous infusion therapy, which will help reduce the burden associated with the injection frequency of other currently available FVIII therapies. For patients reluctant to receive novel therapies, such as mAbs or gene therapy, efanesoctocog alfa will likely be an appealing option.

5. Ensifentrine (RPL554), Verona Pharma

Ensifentrine is an inhaled dual phosphodiesterase (PDE)3 and PDE4 inhibitor that is expected to reduce exacerbations in moderate to severe chronic obstructive pulmonary disease (COPD) without the systemic side effects of current PDE inhibitors that are delivered orally. If approved, it would be the first in class as well as the first novel mechanism that has become available for maintenance COPD treatment in more than 10 years.

6/7. Exagamglogene autotemcel (Casgevy/exa-cel), CRISPR Therapeutics and Vertex Pharmaceuticals, and lovotibeglogene autotemcel (Lyfgenia/lovo-cel), Bluebird Bio

All eyes are on exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel, the first disease-modifying therapies for sickle cell disease and beta-thalassemia. The excitement around exagamglogene autotemcel also stems from the landmark first approval of a CRISPR/Cas9 gene-edited therapy globally, and the approval sets the stage for upcoming approvals in other regions.

8. Mirikizumab (Omvoh/LY-3074828), Eli Lilly

Mirikizumab, an mAb targeting the p19 subunit of IL-23, was approved as first-in-class therapy for ulcerative colitis by the European Medicines Agency (EMA), the US Food and Drug Administration (FDA) and the UK’s Medicines and Healthcare Regulatory Agency, and will likely be the third in the class approved for Crohn’s disease. Due to a delayed US launch it remains a drug to watch for 2024. 

9. Niraparib + abiraterone acetate (Akeega), Johnson & Johnson

This is the first and only dual action tablet combining a PARP inhibitor (niraparib) and a next-generation hormonal therapy (abiraterone acetate). Its ability to serve as a treatment for patients with deleterious or suspected deleterious BRCA-mutated, metastatic castration-resistant prostate cancer (mCRPC) should help to fulfil the need for more effective treatments.

10/11. RSVpreF (Abrysvo/PF-06928316), Pfizer, and RSVpreF3 (Arexvy/GSK-3844766A), GSK

Respiratory syncytial virus (RSV) infections continue to be a public health concern, particularly for infants and older adults (65 years and older). A common upper respiratory infection that can result in hospitalisations in severe cases, RSV infection tends to be seasonal and present with symptoms similar to those of influenza and Covid-19. The first approvals of RSV vaccines (RSVpreF and RSVpreF3) targeted at infants and older adults mark a significant public health milestone.

12. Talquetamab (Talvey), Johnson & Johnson

After receiving conditional and accelerated approval from the European Commission and FDA, respectively, talquetamab became the first-in-class bispecific antibody targeted to CD3 and GPRC5D to treat multiple myeloma. It was approved based on the pivotal Phase I/II MonumenTAL-1 trial for heavily pretreated patients with relapsed or refractory (R/R) multiple myeloma. Ongoing Phase III trials are expected to provide confirmation of clinical benefit in talquetamab’s approved setting and lead to label expansions in other multiple myeloma patient populations.

13. Zolbetuximab (IMAB362), Astellas Pharma

Metastatic HER2-negative gastric and gastroesophageal junction (GEJ) adenocarcinoma is notoriously difficult to treat and has a significant unmet need for new efficacious treatments. In contrast to HER2-positive disease (for which HER2-targeted agents such as trastuzumab [Genentech] and ENHERTU [Daiichi Sankyo] are available), targeted treatment options are more limited for HER2-negative patients. Zolbetuximab would address some of that unmet need as a first-in-class claudin 18.2 inhibitor in oncology as well as first-line metastatic HER2-negative gastric or GEJ adenocarcinoma.

Diana Spencer, Senior Digital Content Editor, DDW

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