13 drugs to watch in 2024

This week, the European Commission approved Casgevy to treat Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia, a few months after it was approved in the U.K. and the U.S. These approvals marked milestones, as the medicine was the first made using CRISPR-Cas9 gene-editing to come to market. Both the technology and the drug were highlighted in Clarivate’s recently released Drugs to Watch 2024 report. They’re not the only transformative moves on the horizon, with the organization saying a dozen other drugs and a handful of new technologies worth watching. Here’s what it chose: Trends to watchIn addition to gene editing, the report also pointed to artificial intelligence and machine learning (AI/ML) as new technologies that would improve drug discovery. More than 500 clinical trials related to artificial intelligence and machine learning were conducted in 2022, and the first drugs created by them are already in phase 2 testing. “This year, we’ll begin to see more tangible real-world examples of how AI/ML are going to transform the discovery, development and delivery of innovative new medicines,” the report says. It also pointed to an increased use of real-world data to help developers, regulators and clinicians understand patients’ needs and optimize clinical trials. Real-world data has already been included in trials for non-insulin-dependent diabetes, COVID-19 and hypertension, among other things. Read: All about drugs: Project cuts levels of pharmaceuticals found in water supplyDrugs that could transform careExagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) are one-time drugs that are the first disease-modifying therapies for sickle cell disease and beta-thalassemia. With exagamglogene autotemcel, patients’ own blood stem cells are modified using genome editing technology, and then transplanted back into their bodies. With Lyfgenia, blood stem cells are also modified to produce a gene-therapy-derived hemoglobin. This year also saw the first vaccines against RSV—a public health milestone for vulnerable groups. The new shots are the culmination of a search for an RSV vaccine that began in the 1960s and was given a boost in the 2010s when the focus shifted to the RSV F protein. RSVpreF (Abrysvo) and RSVpreF3 (Arexvy) are approved in Canada for some groups. The report points out they will face competition shortly, as nearly 20 more RSV vaccines are in the pipeline.Talquetamab is another important treatment. It’s made for patients with multiple myeloma. This drug, which the report describes as a “groundbreaking bispecific antibody,” targets CD3 and GPRC5D. It’s approved in the U.S. to treat patients who have exhausted most or all treatment options, and is being reviewed by Health Canada. Datopotamab deruxtecan is a new option to treat both HR-positive/HER2-negative and triple-negative breast cancer. The report says it can potentially become the best-in-class TROP2-targeted antibody-drug conjugate.Similarly, Zolbetuximab offers promise for a difficult to treat cancer. It’s a targeted treatment option for metastatic HER2-negative gastric of gastroesophageal junction adenocarcinoma, which has limited treatment options. The drug is also being investigated around pancreatic cancer. And Akeega (Niraparib and abiraterone acetate) was described in the report as a “leap forward” in treatment for aggressive prostate cancer. The tablet, which combines a PARP inhibitor and a next-generation hormonal therapy, was approved by Health Canada for deleterious or suspected deleterious BRCA-mutated, metastatic castration-resistant prostate cancer. Read: As cancer treatment advances, patients and doctors push back against drugs’ harsh side effectsDrugs that improve treatmentCOPD hasn’t had a new maintenance treatment in over a decade. Enter Ensifentrine. This inhaled dual PDE3 and PDE4 combines bronchodilator and nonsteroidal anti-inflammatory activities and promotes ciliary function, with fewer systemic side effects. It’s most likely to be used as an add-on to a long-acting bronchodilator therapy, the report says.Mirikizumab is a new first-in-class therapy for ulcerative colitis. The report says it will also likely be approved for Crohn’s disease as well. It’s the first interleukin-23p19 antagonist approved in Canada for moderately to severely active ulcerative colitis. High-dose aflibercept is a treatment for age-related macular degeneration, diabetic macular edema or diabetic retinopathy. Its 12- and 16-week dosing schedule is less frequent and offers similar efficacy and safety as the current standard for care. It’s already been approved by the FDA in the U.S. Another drug that wins on frequency is efanesoctocog alfa. It’s a once-weekly factor VIII replacement intravenous infusion therapy, while current regimens require every other day or twice a week. It’s been approved by the FDA. Finally, budesonide, which treats IgA nephropathy/Berger’s disease, both has better safety and efficacy than first-generation corticosteroids. It reduces protein and slows kidney function decline, and may have fewer side effects than other corticosteroids, even with long-term use. Read: Government launches Implementation Advisory Group as part of rare diseases plan

13 drugs to watch in 2024

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