Citeline gives insights on longer-term outlook at some key late-stage drugs projected to hit market in 2023

Citeline has recently published the “Key Potential Drug Launches in 2023” report giving insights on a longer-term outlook of some key late-stage drugs projected to hit the market in 2023. These drugs represent new drug classes, major changes to standards of care and large market opportunities across the globe.

According to the report, Chronic Heart Failure (CHF) remains a key area of attention for drug makers. In this scenario, Furoscix is a reformulation of the diuretic furosemide which has been developed for the treatment of decompensated heart failure and designed to be self-administered in the outpatient setting through a subcutaneous infusion via a wearable, on-body drug delivery system. It will be used to treat worsening heart failure, due to congestion, in patients with New York Heart Association Class-II and Class-III chronic heart failure who display reduced responsiveness to oral diuretics, and who do not require hospitalisation. The PDUFA date for Furoscix was set as 8th October, 2022 following a resubmission of an NDA in April 2022 which included data from the phase-III Freedom HF trial, where the overall and heart failure-related costs of treating congestion in patients with CHF were investigated.

Simultaneously, Omecamtiv Mecarbil is another soon-to-be-launched drug, which will provide additional means of improving outcomes on top of the standard of care for those patients with more advanced stages of CHF. The report mentioned that this particular drug has a novel mechanism of action as a myosin stimulant, which works by improving cardiac contractility without affecting calcium transients or myocardial oxygen consumption, and has the potential to become an add-on therapy to standard of care for HFrEF patients with worsening heart failure.

A modest magnitude of benefit was shown in phase-III results, which led to an NDA filing being submitted and accepted in February 2022, but the initial PDUFA date was extended by three months to 28th February, 2023 to provide time for a full review of the additional pharmacokinetic analyses which were submitted, as requested by the FDA.

In the oncology space, for bone marrow transplant and stem cell transplant, Gamida Cell’s omidubicel is a nicotinamide (NAM)-enabled stem cell therapy being studied for use in allogeneic hematopoietic (bone marrow) stem cell transplants for patients with hematologic malignancies like acute lymphocytic leukemia. Umbilical Cord Blood (UCB) is currently used as an alternative for these patients when there is no donor-matched cells. However, this approach brings its own set of problems, such as the length of engraftment, increased infection risk and prolonged hospital stays, added the report.

Given the difficulties in sourcing donors for traditional bone marrow transplants, omidubicel provides a more ready alternative to mismatched donors with comparable efficacy. The company expects to capture 18 per cent of the US patient market share or 2000 patients, with omidubicel expected to be launched at a similar price to Chimeric Antigen Receptor (CAR)- T cell therapies ($3,73,000 – $4,75,000), which could lead to the US sales of close to $1 billion within three years of launch. In June 2022, Gamida Cell completed its submission to the FDA for omidubicel’s biologics licence application, with a final decision expected in June 2023, if there are no delays, it mentioned.

It also said that Breyanzi has demonstrated encouraging results in the phase-I/II Transcend-CLL-004 trial, reporting higher observed overall response rates compared to other investigational CD 19-directed CAR-T therapies, such as Kymriah. While the indolent nature of CLL means this patient population is small, it remains a major unmet need, with limited safe treatment options in the third line and beyond setting, especially for patients at risk of Richter’s transformation. Breyanzi looks set to emerge as a revolutionary option for this last-line treatment setting, pending its supplemental approval in 2023.

For Haematology, Vertex had announced that global regulatory filings for exa-cel (CTX001) in transfusion-dependent beta-thalassemia (TDT) and sickle cell disease are expected by the end of 2022, which, if successful, could, in 2023, make it the first CRISPR/Cas 9-based product ever approved, an important boost for the gene editing technology. If approved, these therapies could provide important new options for severe patients with these conditions who do not have well-matched donors for allogeneic bone marrow transplantation, noted the report.

These potential drug launches are expected to boost the drug discovery and development market and most forecasts indicate that 2023 will be another highly lucrative year for drug developers across therapeutic areas, the report concluded.