First RSV shots, groundbreaking gene therapy among 2024 drugs to watch

Some of the pioneering medical breakthroughs in recent history, including the first RSV vaccines and a first-of-its-kind gene therapy, have made it into the 2024 edition of the Drugs to Watch report compiled by the data analytics company Clarivate (CLVT).

The report highlights 13 recently launched or upcoming drugs with game-changing potential, including those expected to achieve blockbuster status (the ability to generate $1B or more in annual sales) within five years.

For its analysis, the U.K.-based firm only considered drugs launched early last year or investigational drugs undergoing Phase 2 and Phase 3 trials in the pre-registrational stage and excluded treatments launched before 2023.

“New technology platforms that are likely to achieve significant proof of concept in 2024 include CRISPR-Cas9 gene editing as well as artificial intelligence /machine learning tool applications in drug discovery, clinical development, and commercial launch,” said Henry Levy, who leads Clarivate’s Life Sciences & Healthcare segment.

Regeneron’s (REGN) recently launched eye disease therapy, high-dose Eylea leads the list.

The VEGF inhibitor indicated at 8 mg for retinal diseases including diabetic macular edema (DME) “offers less-frequent administration while achieving similar efficacy and safety as the current standard of care, Eylea 2mg or Roche’s (OTCQX:RHHBY) (OTCQX:RHHBF) Lucentis,” Clarivate (CLVT) wrote.

The firm projects $1.77B in 2027 sales for Eylea HD in G7 countries from patients with wet age-related macular degeneration (AMD) alone.

With its German partner, Bayer (OTCPK:BAYZF) (OTCPK:BAYRY), the U.S. drugmaker expects to recognize ~$123M in U.S. net product sales for the drug in Q4 2023.

Calliditas Therapeutics’ (CALT) (OTCPK:CLTEF) delayed-release corticosteroid formulation, Budesonide, fully approved last year for adults with immunoglobulin A nephropathy, is ranked second.

Datopotamab deruxtecan, a cancer therapy from AstraZeneca (AZN) and Daiichi Sankyo (OTCPK:DSKYF), is placed third. After Trodelvy from Gilead (GILD), the antibody-drug conjugate is set to be the second to launch for both HR-positive/HER2-negative and triple-negative breast cancer, Clarivate (CLVT) said.

This week, Japan-listed shares of Daiichi (OTCPK:DSNKY) traded higher after the company said it has filed for its U.S. approval. The treatment is also under investigation as a late-line option for non-small cell lung cancer.

Roche (OTCQX:RHHBY) and Sobi’s (OTCPK:BIOVF) Factor VIII replacement therapy, efanesoctocog alfa for hemophilia A, is fourth in the list on its appeal for cases where novel treatments such as gene therapies are not available.

Verona Pharma’s investigational therapy for chronic obstructive pulmonary disease (COPD), ensifentrine, is also among Clarivate’s (CLVT) drugs to watch given its novelty.

The non-steroidal therapy is currently under FDA review, with a target action date of June 26, 2024. The firm highlighted its potential against neutrophilic inflammation, a key mechanism in COPD that often responds poorly to steroids.

Abrysvo and Arexvy, for which developers Pfizer (NYSE:PFE) and GSK (NYSE:GSK)/Agenus (AGEN), respectively, received U.S. approval to launch in 2023 as the first FDA-cleared vaccines for respiratory syncytial virus (RSV), also made their debut in the list.

As did one-time gene therapies, Casgevy and Lyfgenia from Crispr Therapeutics (NASDAQ:CRSP)/ Vertex Pharma (NASDAQ:VRTX), and Bluebird Bio (BLUE). Both drugs are the first disease-modifying therapies for inherited blood disorders, sickle cell disease (SCD) and beta-thalassemia, Clarivate (CLVT) said.

According to the firm, Casgevy, the first gene editing therapy to receive regulatory approval in November, is expected to generate $1.32B in sales in 2029.

While the U.S. approved both treatments for SCD in December, Casgevy is currently under FDA review for transfusion-dependent beta-thalassemia, with a target action date of March 30.

With estimated sales of $2.7B in 2029, Johnson & Johnson’s (JNJ) investigational prostate cancer therapy Akeega has also made it onto the list, along with its bispecific antibody, Talvey, cleared in the U.S. last year under the FDA’s accelerated pathway for multiple myeloma.

Omvoh and zolbetuximab, two monoclonal antibodies developed by Eli Lilly (LLY) and Astellas Pharma’s (OTCPK:ALPMF), respectively, round out the list.

“A delayed U.S. launch due to manufacturing concerns by the U.S. FDA means that it remains a drug to watch for 2024, Clarivate (CLVT) wrote about Omvoh, approved in October for ulcerative colitis.

This week, Astellas (OTCPK:ALPMY) failed to win an FDA nod for zolbetuximab in gastric or gastroesophageal junction cancer due to issues at a third-party manufacturing facility. The company expects to refile for approval.

Clarivate (CLVT) highlighted 14 treatments as drugs to watch in its 2023 list. Twelve of those were granted regulatory approval. Donanemab, Eli Lilly’s (LLY) Alzheimer’s therapy, and Roctavian, BioMarin’s (BMRN) gene therapy for hemophilia A were part of that list.

Donanemab did not clear regulatory hurdles last year, while Roctavian had a lackluster launch in its first few months after approval. “These examples show just how challenging drug launches can be,” Clarivate’s (CLVT) Levy argued.