Novel FDA Approvals – January 2025

From oncology to pain management to transplant prep, January’s approvals touch multiple therapeutic pillars. With two first-in-class drugs and strong clinical performance across all three products, this month’s roundup showcases both scientific innovation and strategic positioning—setting the tone for a competitive year in novel therapies.

Empty space, drag to resize
The FDA’s Center for Drug Evaluation and Research (CDER) is responsible for regulating drug products in the United States and approving novel therapeutics. Over the past decade, from 2015 to 2024, the Center for Drug Evaluation and Research approved an average of 47 novel drugs per year. 
Write your awesome label here.
*Sourced from the FDA as of February 2025 at https://www.fda.gov/media/184967/download?attachment#:~:text=The%2010%2Dyear%20graph%20below,novel%20drug%20approvals%20per%20year.&text=CDER%20identified%2024%20of%20the,as%20first%2Din%2Dclass. 
Empty space, drag to resize
Of the 50 novel therapeutics approved in 2024, 24 (48%) of them were identified as first-in-class treatments that provide unprecedented pharmacologic effects in their targeted disease state.

During January 2025, The Center for Drug Evaluation and Research approved 3 novel drugs to enter the pharmaceutical market: 
  • Datroway (datopotamab deruxtecan-dlnk)
  • Grafapex (treosulfan)
  • Journavx (suzetrigine)
This article will take a deeper look into each of these newly approved medications, exploring the pharmacologic properties and approval pathways associated with them.  

Datroway 

Datroway (datopotamab deruxtecan-dlnk) was the first novel drug approved in the new year, receiving standard approval from the Center for Drug Evaluation and Research on January 17, 2025. Daiichi Sankyo is credited with the discovery of this drug, who actually joined forces with AstraZeneca to develop and commercialize it.

Datroway is a Trop-2-directed antibody drug conjugate (ADC) approved for the treatment of unresectable or metastatic, HR-positive, HER2-negative breast cancer. Datroway is intended for patients that have already received endocrine therapy and chemotherapy for unresectable or metastatic breast cancer. 

About Breast Cancer

Breast cancer has the highest incidence rate for cancer among women in the United States, accounting for roughly 30% of new female cancer diagnoses annually. The prognosis and treatment plans for each diagnosis, however, may differ depending on the subtype of breast cancer. In order from highest prevalence to lowest, those subtypes are: 
  • HR+/HER2-
  • HR-/HER2-
  • HR+/HER2+ 
  • HR-/HER2+
These breast cancer subtypes are important for identifying tumor cell characteristics in order to optimize treatment regimens. The presence of hormone receptors on tumor cells (HR+) allows for the binding of estrogen and/or progesterone to promote tumor growth. With this said, patients with HR+ breast cancer should always receive endocrine therapy as part of their treatment regimen. According to the Susan G. Komen Foundation, hormone receptor status can also determine the risk of breast cancer recurrence. The presence of human epidermal growth factor receptor 2 (HER2+) on tumor cells indicates a high level of HER2 protein production, promoting tumor cell growth and survival. All patients with a HER2+ subtype, regardless of hormone receptor status, should receive HER2 directed therapy as part of their regimen.  

How Does Datroway Work? 

As previously stated, Datroway is a TROP2-directed antibody drug conjugate. This drug is comprised of three sections that are designed to induce selective tumor cell death and reduce systemic exposure to the topoisomerase I inhibitor payload. In other words, Datroway has the ability to destroy tumor cells while minimizing drug exposure to healthy tissues.  

Mechanism of Action 

Understanding Datroway’s mechanism of action means understanding its three components:

Deruxtecan (DXd) Payload

Deruxtecan is the cytotoxic agent in Datroway that promotes cell death by inhibiting the replicative effects of topoisomerase I. The payload of deruxtecan is very potent but has a short half-life and is stable in plasma.

Trophoblast Cell Surface Antigen 2 (Trop-2) Monoclonal Antibody

 This monoclonal antibody binds to Trop-2 proteins on the tumor cell surface to selectively deliver the cytotoxic agent deruxtecan (DXd). 

Tumor-Selective Cleavable Linker

The linker in Datroway secures the deruxtecan to the monoclonal antibody. The linker is cleaved by tumor cell enzymes which releases the deruxtecan payload and causes tumor cell death.
Write your awesome label here.
*Sourced from Daiichi Sankyo as of February 2025 at https://datrowayhcp.com/mechanism-of-action 

Path to Approval 

The FDA approval process for Datroway involved all of the standard steps for ensuring its safety and efficacy, including preclinical trials, an investigational new drug application (IND), human clinical trials, and a new drug application (NDA).  

TROPION-Breast01 

The phase III TROPION-Breast01 clinical trial was pivotal for the FDA approval of Datroway. The purpose of this study was to evaluate the safety and efficacy of Datroway compared to the standard-of-care chemotherapy agents in patients with inoperable or metastatic HR+/HER2- breast cancer. The patients enrolled in this study had already undergone one or two lines of systemic chemotherapy in the setting of inoperable/metastatic breast cancer and were randomized to receive either Datroway or another standard chemotherapy chosen by the investigator.

The TROPION-Breast01 trial showed a 37% reduction in the risk of progression or death with 6.9 months median progression-free survival compared to 4.9 months for patients on standard chemotherapy. Datroway also displayed an objective response rate of 36% compared to 23% in the investigator chosen chemotherapy group. In short, Datroway achieved a statistically significant and clinically meaningful improvement in progression-free survival for patients with HR+/HER2- breast cancer in the TROPION-Breast01 trial, leading to its approval shortly thereafter.  

Fitting Into the Current Landscape 

Regarding the current trajectory for HR+/HER2- breast cancer progression, Caitlin Lewis, Senior Vice President of Strategy & Mission, Living Beyond Breast Cancer, stated that “Only one in three patients with metastatic HR-positive, HER2-negative breast cancer live more than five years following diagnosis, highlighting the urgent need for additional effective therapies. The approval of Datroway is a significant advance, offering patients with metastatic HR-positive breast cancer a new and much-needed treatment option.”

Another notable comment regarding Datroway’s recent approval comes from Aditya Bardia, MD, MPH, Program Director of Breast Oncology and Director of Translational Research Integration at the UCLA Health Jonsson Comprehensive Cancer Center and Global Principal Investigator for TROPION-Breast01. Dr. Bardia shares that “Despite considerable progress in the HR-positive, HER2-negative metastatic breast cancer treatment landscape, new therapies are still needed to tackle the frequent and complex challenge of disease progression after endocrine and initial chemotherapy. The approval of datopotamab deruxtecan, a novel TROP2-directed antibody drug conjugate, marks a major therapeutic milestone and provides patients with metastatic breast cancer a new treatment alternative to conventional chemotherapy.”

In adding Datroway to the growing arsenal of therapeutic options for HR+/HER2- breast cancer, patients and providers have access to a new treatment that can improve the chance of progression-free survival in patients who have already endured previous treatment.  

Grafapex

On January 21, 2025, Medexus Pharma’s new drug Grafapex (treosulfan) obtained standard FDA approval for marketing in the United States. Grafapex is an alkylating drug indicated in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients >1 year old with acute myeloid leukemia and/or myelodysplastic syndrome. Before explaining what that really means, the following primer on each of these disease states can be beneficial in understanding what this new drug is and how it works. 

Acute Myeloid Leukemia and Myelodysplastic Syndrome 

Acute myeloid leukemia (AML) is a subtype of blood cancer that originates in the bone marrow. In this disease state, acquired mutations in myeloid stem cells transform them into cancerous blast cells that do not function normally. These leukemic blast cells block the production of normal stem cells and surpass their functions with superior growth and survival, causing rapid progression.

Myelodysplastic syndrome is another form of blood cancer that originates in bone marrow but differs from acute myeloid leukemia in that there are no genetic mutations or an oversupply of blast cells. Myelodysplastic syndrome is instead characterized by an abundance of abnormal (dysplastic) stem cells that cannot effectively produce new blood cells, and for 1 in 3 patients the disease can progress to acute myeloid leukemia.

Despite the specific pathology of these two disease states, the resulting stem cell abnormalities lead to an inadequate supply of healthy blood cells, which can result in the following conditions: 

Anemia

a low number of red blood cells that presents with fatigue and shortness of breath in patients

Neutropenia

a low number of neutrophils (a type of white blood cell) that prevents the immune system from effectively fighting infections

Thrombocytopenia

a low number of platelets that can cause excess bleeding and easy bruising

Pancytopenia

a low number of red blood cells, neutrophils, and platelets
Typical treatments for these conditions include chemotherapy and supportive therapies like blood transfusions and growth factors to improve blood cell counts. 

How Does Grafapex Work? 

As previously stated, grafapex is an alkylating agent. Grafapex attaches to and alkylates the DNA in rapidly dividing cancer cells to stop them from multiplying and ultimately kill them. Grafapex is used to deplete cancerous stem cells from the body prior to initiation of stem cell transplantation. The purpose of jointly administering fludarabine with grafapex is to produce a synergistic effect that impairs DNA damage repair.

It is also important to note that Grafapex has a boxed warning for myelosuppression at the recommended dose of 10 g/m2/day IV. While hematopoietic stem cell transplantation typically follows administration of Grafapex, it is required to prevent the potentially fatal complications of prolonged myelosuppression.

Clinical Trial Evidence

The phase III clinical trial of Grafapex compared its safety and efficacy to that of busulfan as a preparative regimen for allogeneic stem cell transplantation in 570 patients. The major endpoints in this trial were event-free survival and overall survival between the two groups after 36 months. Event-free survival in the Grafapex group was 59.5% compared to 49.7% in the busulfan group, and overall survival was 66.8% compared to 56.3% in the control group.

This clinical trial helped pioneer FDA approval for Grafapex by displaying an increased achievement of overall survival in patients with acute myeloid leukemia and myelodysplastic syndrome. 

Entering the Current Landscape 

Grafapex holds an orphan drug designation from the FDA, meaning that Medexus will have regulatory exclusivity over this product for 7.5 years in its approved indications. In a press release from Medexus Pharmaceuticals, Dr. Filippo Milano, a stem cell transplant physician-scientist and principal investigator in clinical trials using Grafapex stated that "This FDA approval provides a useful option for adult and pediatric patients, with the potential to enhance overall survival while minimizing side effects."

Journavx

On January 30, 2025, Vertex Pharmaceutical received FDA approval for its first-in-class non-opioid analgesic, Journavx, for the treatment of moderate to severe acute pain in adults.

The new drug application for Journavx received Breakthrough Therapy, Fast Track, and Priority Review designations by the FDA prior to approval. In response to these designations, Jacqueline Corrigan-Curay, J.D., M.D., acting director of the FDA's Center for Drug Evaluation and Research, shared that “This action and the agency’s designations to expedite the drug’s development and review underscore FDA’s commitment to approving safe and effective alternatives to opioids for pain management.” 

How Does Journavx Work? 

Journavx is an oral sodium channel blocker that targets the Nav1.8 channel to inhibit the transmission of pain signals in patients experiencing moderate to severe acute pain. Journavx is a highly selective inhibitor of Nav1.8 and it is known that Nav1.8 is not expressed in the human brain. By targeting pain without acting on mu opioid receptors, Journavx is not a controlled substance and displays no evidence of addictive potential. A visual representation of this mechanism of action can be seen below: 
Write your awesome label here.
*Sourced from Vertex as of February 2025 at https://www.journavxhcp.com/mechanism-of-action 

Clinical Trial Evidence

Prior to FDA approval, Journavx was evaluated in more than 800 patients across two phase III trials for the management of moderate to severe acute pain. The primary endpoint in both trials was superiority against placebo on the time-weighted sum of the pain intensity difference (SPID) as recorded on the Numeric Pain Rating Scale (NPRS) from 0 to 48 hours. This means that in both clinical trials, clinical effect was determined by a measurement of pain severity over 48 hours, and investigators hypothesized a superiority in pain reduction with Journavx compared to placebo. The first study involved patients experiencing moderate to severe pain following abdominoplasty, and the second study analyzed patients with moderate to severe pain following bunionectomy.

In each of these clinical trials, all patients were randomized to receive Journavx, placebo, or hydrocodone/acetaminophen in a 2:1:2 ratio. While both studies met the primary endpoint of demonstrating a statistically significant superior reduction in pain versus placebo, no superiority in pain reduction was met between Journavx and hydrocodone/acetaminophen. 

Entering the Current Landscape

Pain is an extremely common medical problem, and pain relief is an important therapeutic goal for acute pain caused by trauma or injury. While various analgesic options exist for pain management, opioids are the most common choice for moderate to severe pain. The major issue with opioids, however, is the high risk for physical and psychological dependence along with potentials for abuse and overdose. With Journavx approved to enter the market, patients and providers now have access to a non-opioid analgesic that can effectively mitigate moderate to severe pain without having to necessarily rely on opioids.  

A Summary of January Approvals

It is no secret that the pharmaceutical landscape is constantly seeing innovation and growth, and staying up to date on approval trends enables pharmacists to better optimize patient care, ensure medication safety, and provide informed recommendations to healthcare teams. By delivering timely updates on novel therapeutics and emerging treatment trends, Ceuticon aims to support pharmacists in maintaining professional competence, adapting to industry advancements, and enhancing overall healthcare outcomes. 

Keeping up with approvals is just the beginning...

We break down the why it matters—
so you’re not just informed, you’re industry-ready.

🚀 Join our list and get early access to new tools, career content, and practical updates.

Empty space, drag to resize
Thank you! Keep an eye on your email to get early access to new tools, career content, and practical updates. 📬
Created with