First injections of RSV and gene editing drug among the drugs of 2024 to take into account

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

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

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

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

Regeneron's (REGN) recently launched a therapy for eye diseases, Eylea in high doses leads the list.

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

The company projects $1.77 billion in 2027 sales of Eylea HD in G7 countries from wet age-related macular degeneration (AMD) patients alone.

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

Calliditas Therapeutics (CALT) Delayed-Release Corticosteroid Formulation (OTCPK:CLTEF), budesonide, fully approved last year for adults with immunoglobulin A nephropathy, ranks second.

Datopotamab deruxtecan, a cancer therapy from AstraZeneca (AZN) and Daiichi Sankyo (OTCPK:DSKYF), comes in third. After Gilead's (GILD) Trodelvy, the antibody-drug conjugate will be the second to launch for 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 had applied for approval in the United States. The treatment is also being investigated as a late option for non-small cell lung cancer.

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

Verona Pharma's investigational therapy for chronic obstructive pulmonary disease (COPD), ensientrina, It is also among the Clarivate (CLVT) medications to consider given its newness.

The nonsteroidal therapy is currently under review by the FDA, with an expected 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 Arexvyfor which the developers Pfizer (New York Stock Exchange:PFE) and GSK (New York Stock Exchange:GSK)/Agenus (AGEN), respectively, received US approval for launch in 2023, while the first FDA-approved vaccines against respiratory syncytial virus (RSV) also made their debut on the list.

Like 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 the inherited blood disorders sickle cell anemia (SCD) and beta-thalassemia, Clarivate (CLVT) said.

Casgevy, the first gene-editing therapy that received regulatory approval in November, is expected to generate $1.32 billion in sales in 2029, according to the company.

While the United States approved both treatments for SCD in December, Casgevy is currently under FDA review for transfusion-dependent beta-thalassemia, with an expected action date of March 30.

With estimated sales of $2.7 billion in 2029, Johnson & Johnson's (JNJ) investigational prostate cancer therapy with akee has also made the list, along with its bispecific antibody, Talvey, approved in the US last year under the FDA's fast track for multiple myeloma.

Omvoh and zolbetuximabtwo monoclonal antibodies developed by Eli Lilly (LLY) and Astellas Pharma (OTCPK:ALPMF), respectively, complete the list.

“A delayed launch in the US due to manufacturing concerns by the US FDA means 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 gain FDA approval for zolbetuximab in gastric or gastroesophageal junction cancer due to problems at a third-party manufacturing facility. The company hopes to resubmit the application for approval.

Clarivate (CLVT) highlighted 14 treatments as drugs to watch for its 2023 list. Twelve of them gained 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 failed to clear regulatory hurdles last year, while Roctavian had a lackluster launch in the first months after its approval. “These examples show how challenging drug launches can be,” Clarivate's (CLVT) Levy argued.