Top 5 Breakthrough Drugs of the Year

Pharmaceutical researchers made astounding strides this year towards developing drugs to target specific genes and pathways involved in tumor progression, potentially improving treatment outcomes and perhaps curing cancer and other incurable diseases altogether.

Daprodustat (Leqembi) is an oral medication designed specifically to address anemia among dialysis patients. It works by inhibiting an enzyme involved in heme production pathway.

1. Daprodustat

Daprodustat (known as Duvroq in Japan) is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) designed to increase hemoglobin and noninferior to epoetin as a treatment option in patients on dialysis with chronic kidney disease suffering anemia. As one of the few new offerings available within a treatment class that has largely stagnated over the decades.

Hemodialysis-dependent patients with chronic kidney disease (CKD) frequently experience anemia due to both ongoing blood losses and impaired iron absorption due to elevated serum hepcidin levels, making the condition one of the leading causes of morbidity and mortality among these individuals. Anemia remains one of the primary contributors to morbidity and mortality associated with CKD.

HIF-PHIs such as daprodustat, roxadustat, and vadadustat work by directly inhibiting PHD dioxygenases to stimulate erythropoiesis. They have an excellent safety record, are inexpensive to administer without needing special hematology equipment, and do not cause drug interactions such as those seen when injectable erythropoietins interact with inhibitors/inducers of CYP2C8 enzymes (CYP2C8 inhibitors/inducers).

2. Tremelimumab

Imjudo, an immunotherapy drug commonly referred to as Imjuvo, is a monoclonal antibody designed to target and inhibit cytotoxic T lymphocyte-associated protein 4 (CTLA-4). By blocking CTLA-4, Imjudo enhances your body’s natural defenses against cancer cells and strengthens their natural response against them.

Tremelimumab shows promising results when used alongside durvalumab for treating liver cancer, particularly among patients with unresectable hepatocellular carcinoma. It was proven effective through the phase II trial STRIDE.

Tremelimumab should not be used by individuals with preexisting autoimmune conditions as its use amps up their immune systems and may lead to serious side effects including hepatitis. Females of reproductive potential are advised against breastfeed while taking this medicine since its effect passes through the placenta and can harm an unborn baby; pregnancy should therefore be avoided during use and for three months afterwards.

3. Teclistamab-cgyv

Teclistamab-cqyv is the first bispecific antibody approved by the FDA for treating multiple myeloma, working by targeting two molecules found on cancerous cells: BCMA-Directed T cell Engagr (BiTE) molecule and CD3 receptor present on immune cells – this enables their own immune systems to attack myeloma cells with double binding action and kill them effectively.

MajesTEC-1 trial data presented at ASCO 2023 supported its approval as treatment for patients with relapsed/refractory multiple myeloma who had failed at least three or four previous lines of therapy, such as lenalidomide, proteasome inhibitors and an anti-CD38 monoclonal antibody.

Due to potential CRS and neurologic toxicities associated with immune effector cell-associated neurotoxicity syndrome (ICANS), this medication must be made available under a restricted program known as Tecvayl REMS and requires careful management during step-up dose regimen and treatment for CRS.

4. T-DXd

Trials showed that T-DXd quickly reduced tumors in people with advanced HER2-positive cancer and, in many cases, even stopped them from growing for months or even years.

This drug is an antibody-drug conjugate, composed of a monoclonal antibody called trastuzumab chemically linked with cell-killing chemotherapy drug deruxtecan. Trastuzumab acts as an anchor that finds cancer cells that express HER2 protein on their surface, before the conjugate delivers deruxtecan into each one to kill it off.

T-DXd was recently approved by the FDA as an adjuvant treatment for metastatic HER2-positive breast cancer that has failed to respond to other targeted therapies, such as trastuzumab or pertuzumab. Additional trials are taking place to see whether T-DXd can also treat low HER2 cancers unresectable or metastatic breast cancers; as well as serve as adjuvant therapy or neoadjuvant therapy in early-stage disease.

5. Brentuximab

Brentuximab is a monoclonal antibody designed to target CD30, a protein found on lymphocytes and certain cancer cells. As part of an antibody-drug conjugate class of medications, Brentuximab may be used to treat classical Hodgkin lymphoma (sALCL), systemic anaplastic large cell lymphoma, peripheral T-cell lymphomas and primary cutaneous anaplastic large cell lymphomas.

Healthcare professionals typically administer this injection intravenously over 30 minutes via intravenous infusion to patients at least every 2 or 3 weeks, depending on your weight. Doses vary according to body mass index (BMI). While the treatment can help lower your blood cell counts, you will require regular medical exams due to possible blood cell changes caused by this drug. Serious allergic reactions have also been reported with its use; be sure to discuss with your physician ways you can mitigate or manage these potential side effects.