5 recent FDA oncology approvals that could change the future of healthcare

In the field of oncology, medical advancements in treatment are not just groundbreaking but life-changing for millions living with cancer worldwide. Over the past year, the FDA has greenlit several groundbreaking oncology drugs and devices, each offering hope and potential for improved patient outcomes.

The global oncology market in 2024

The global oncology market has seen steady growth in recent years and is expected to reach US$19.19 billion by the end of 2024, with a compound annual growth rate (CAGR) of 2.3%, according to the Oncologists Annual Market Report. Growth for new cancer treatments is being powered by the increasing prevalence of cancer globally. This is forcing drug manufacturers to look for new innovative treatments, with the emergence of precision medicine, shifts in epidemiology and advancements in immunotherapy all driving new ways to treat the disease.

From targeting specific genetic mutations to developing one-of-a-kind oncology treatments, these medications offer new hope and improved outcomes for patients with various forms of cancer. In this blog, we list 5 FDA approved oncology drugs and devices that are shaping the future of healthcare and also look at what other exciting approvals are coming soon.

1.Toripalimab-tpzi (Loqtorzi)

Developed by Shanghai Junshi Bioscience Co., Ltd. and Coherus BioSciences, toripalimab-tpzi, marketed under the name Loqtorzi, was approved by the FDA in October 2023, after being on the market in China since 2018.

The FDA approved toripalimab-tpzi (Loqtorzi) to treat adult patients with nasopharyngeal carcinoma, which is a rare tumour of the head and neck and around 1 in 100,000 are diagnosed with this each year globally. In a press release by Coherus, announcing the drug approval, Jong Chul Park, M.D., Assistant Professor, Harvard Medical School and attending physician at the Center for Head and Neck Cancers at Massachusetts General Hospital Cancer Center, said “ the FDA approval of Loqtorzi is very encouraging for those living with NPC who currently have very limited treatment options and are in need of new therapies to treat this aggressive and life-threatening form of cancer,” he went on to say, “Loqtorzi is a new treatment option that has demonstrated the ability to significantly improve PFS and OS and should quickly emerge as the new standard of care when used in combination with chemotherapy.”

2. Momelotinib (Ojjaara)

GSK’s Ojjaara (momelotinib) was approved in the US in September 2023, as the first and only treatment for myelofibrosis patients with anaemia. Ojjaara is an oral activin A receptor type I (ACVR1) and Janus kinase 1 and 2 (JAK1/JAK2) inhibitor developed by Cytopia Ltd, a biomedical business that studies and creates small molecule treatments for ailments like immunological, cardiovascular, and oncology.

Momelotinib addresses the primary symptoms, such as anemia, constitutional symptoms, and splenomegaly (enlarged spleen), of the rare blood cancer, myelofibrosis. In the global, randomised, double-blind Momentum trial, 195 patients with anaemia and symptoms of myelofibrosis who had previously taken a JAK inhibitor were compared between momelotinib and danazol therapy. According to efficacy evaluations, myelofibrosis symptoms were reduced by at least 50% in 25% of individuals treated with momelotinib, compared to 9% of patients treated with danazol.

In a press release announcing the drug’s approval, Nina Mojas, Senior Vice President, Oncology Global Product Strategy, GSK, commented, “The vast majority of myelofibrosis patients eventually develop anaemia, causing them to discontinue treatments and require transfusions. Given this high unmet need, we are proud to add Ojjaara to our oncology portfolio and address a significant medical need in the community. We look forward to helping improve outcomes in this difficult-to-treat blood cancer”.

3. Omidubicel-onlv (Omisirge)

Approved in May 2023, Gamida Cell Ltd’s Omisirge has been developed for treating blood cancers in patients aged 12 years and above who are scheduled for umbilical cord blood transplantation after undergoing a myeloablative conditioning regimen. This treatment is designed to enhance the body's neutrophil recovery and decrease the risk of infections. Results from the Study P0501 phase 3 trial revealed that 87% of the 52 patients who received omidubicel experienced neutrophil recovery within an average of 12 days. Notably, this therapy is the first allogeneic stem cell transplant treatment to gain approval based on a global, randomized Phase 3 clinical trial.

In a company press release, Abbey Jenkins, President and Chief Executive Officer of Gamida Cell commented, “FDA approval of Omisirge is a major advancement in the treatment of patients with hematologic malignancies that we believe may increase access to stem cell transplant and help improve patient outcomes”. Steven M. Devine, M.D., Chief Medical Officer at the National Marrow Donor Program, added “Patients who are Black or African American have just a 29% chance of finding a match via the donor registry vs. a 79% chance for patients who are White. Adding Omisirge as a new donor source may help increase access to stem cell transplant for patients from racially or ethnically diverse backgrounds who struggle to find a fully matched donor in the registry”.

4. Hepzato Kit (melphalan for Injection/Hepatic Delivery System) 

Approved in August 2023 and developed by Delcath Systems, Inc., Hepzato Kit is a treatment for patients diagnosed with metastatic uveal melanoma that is unresectable and prevalent in the liver.

Melphalan has been approved and used in the United States since 1964, the Hepzato Kit takes the potency of high-dose melphalan and gets it directly into the liver. Melphalan causes fast and irreversible DNA damage, killing off cancer cells.

Eligible patients are those with unresectable hepatic metastases affecting less than 50% of the liver function, absence of extrahepatic disease, or limited extrahepatic disease in the bone, lymph nodes, subcutaneous tissues, or lung that can be treated with resection or radiation. Approval for this treatment was based on findings from the phase 3 focus study, which reported an overall response rate of 36.3% (95% CI, 26.4-47.0) and an average response duration of 14 months. The study also showed a disease control rate of 73.6%, with seven patients (7 out of 100) achieving a complete response and 26 achieving partial responses (28.6%).

5. Retifanlimab-dlwr (Zynyz)

Retifanlimab-dlwr (Zynyz), developed by Incyte Corporation, was approved for the treatment of patients with metastatic or recurrent, locally advanced merkel cell carcinoma (MCC). Drugs such as Merck & Co’s blockbuster Keytruda and Pfizer’s Bavencio are currently approved to treat this rare skin cancer, but with incidence rates are rising, there is increasing demand for new treatment option.

The approval for Zynyz was based on a mid-stage study, which showed the therapy helped decrease the size of tumors or remove all signs of the cancer in patients. Results from the phase 2 PODIUM-201 trial showed a 52% overall response rate. Of the patients that responded, twenty-six had a duration of response (DOR) of at least six months, and twenty-one had a DOR of at least twelve months.

Zynyz is also thought to have potential to be beneficial to patients with other variations of the disease and is currently being studied for the treatment of other types of cancers.

Other upcoming oncology approvals to look out for

This year will see the development of more oncology drugs, such as monoclonal antibodies, oral medications, and gene treatments. Here are approvals that are eagerly anticipated and looking to get the seal of FDA approval this year:

Datopotamab (deruxtecan)

Created in collaboration between AstraZeneca and Daiichi Sankyo, datopotamab deruxtecan, or Dato-DXd, is an antibody-drug combination that targets TROP2. Topoisomerase I inhibitor (a derivative of camptothecin) is coupled to a cleavable tetrapeptide linker using a humanised anti-TROP2 IgG1 monoclonal antibody to form Dato-DXd.

Datopotamab deruxtecan showed a statistically significant improvement for the dual primary endpoint of progression-free survival (PFS) in patients with locally advanced or metastatic NSCLC treated with at least one prior line of therapy compared to docetaxel, the current standard of care, in the TROPION-Lung01 Phase III trial.

Both companies are expecting to file for FDA approval in the near future, but this could push into 2025 as the path to approval hasn’t been straightforward, after patient deaths in a lung cancer trial raised questions over the drug’s safety. In October 2023, AstraZeneca and Daiichi told Fierce Biotech that despite these deaths, which occurred as a result of damaged lungs, the benefit-risk “still seems to tip towards Dato-DXd,” particularly in patients with non-squamous non-small cell lung cancer.

AnktivaTM

The FDA has chosen AnktivaTM, the main cytokine infusion protein from ImmunityBio, as a breakthrough therapy and fast track designation for the treatment of BCG-unresponsive CIS non-muscle invasive bladder cancer. In a Phase 2/3 clinical trial treating NMIBC in situ (CIS) that was not responding to BCG, Anktiva with BCG accomplished the primary endpoint for patients with papillary tumour subtype, with 57% of patients achieving a 12-month disease-free survival (DFS).

Equipped with the necessary funds, ImmunityBio will seek FDA clearance of Anktiva by April 23 and accelerate commercialisation efforts. It will also conduct clinical trials to extend the molecule's application to other solid tumours. Speaking at the November investor gathering, ImmunityBio CEO Rich Adcock stated that the business intends to “have product in the field right away if the FDA signs off.”

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