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The FDA’s
Center for Drug Evaluation and Research (CDER) plays a vital role in the advancement of healthcare, approving an average of
47 novel therapeutics each year. This growing list of novel approvals has expanded treatment options for patients across a wide range of conditions, offering innovation and diversity to those who need it. A significant portion of these approvals introduce groundbreaking therapies, including first-in-class drugs and significant improvements over previous treatment options.
Throughout March 2025, the Center for Drug Evaluation and Research approved 2 novel therapeutics for clinical use in the United States:
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Blujepa (gepotidacin)
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Qfitlia (fitusiran)
This article will further explore each of these new medications and discuss their anticipated roles in mainstream healthcare.
As of March 25, 2025,
Blujepa (gepotidacin) has been FDA approved for the treatment of uncomplicated urinary tract infections (uUTIs) in female adults and pediatric patients 12 years of age and older. This first-in-class oral antibiotic from GlaxoSmithKline (GSK) is part of their infectious disease portfolio and offers a new treatment option for the
16 million women who experience urinary tract infections each year.
Upon its recent approval, Tony Wood, Chief Scientific Officer, GSK, shared that “The approval of Blujepa is a crucial milestone with uUTIs among the most common infections in women. We are proud to have developed Blujepa, the first in a new class of oral antibiotics for uUTIs in nearly three decades, and to bring another option to patients given recurrent infections and rising rates of resistance to existing treatments.”
According to the U.S. Department of Health & Human Services (HHS),
more than 50% of all women experience at least one urinary tract infection in their lifetime, and roughly 30% experience recurrent episodes. While most urinary tract infections can be easily treated with antibiotics, they can be painful, uncomfortable, and may lead to more serious infections.
While urinary tract infections do involve bacteria growth along the urinary tract, they are primarily characterized by the presence of accompanying symptoms. Common symptoms of a urinary tract infection may include:
Once a urinary tract infection is diagnosed, it can be further classified as “complicated” or “uncomplicated”. For a urinary tract infection to be “uncomplicated”, a patient must meet ALL of the following criteria:
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Female sex
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Not pregnant
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No known urologic abnormalities
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No known co-morbidities (e.g., diabetes, kidney disease)
It is important for clinicians to make this delineation between categories because it can help identify potential causative organisms and guide proper therapeutic approaches.
Approximately 80% of uncomplicated infections are caused by
Escherichia coli bacteria and the duration of treatment can last up to one week, depending on the antibiotic.
While over-the-counter pain relievers and nonpharmacologic interventions (e.g., hydration, warm compresses, hygiene) can help alleviate the symptoms associated with urinary tract infections, the mainstay of treatment is a full course of oral antibiotics. Since the majority of uncomplicated urinary tract infections are caused by
E. coli, any antibiotic choice should provide gram-negative coverage at a minimum.
Historically, common therapeutic agents for uncomplicated urinary tract infection include:
While the Infectious Diseases Society of America supports the first-line use of these drugs in their guidelines for uncomplicated urinary tract infection, patients with contraindications or those in areas with a high resistance rate may be able to use one of the following antibiotics instead:
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Amoxicillin/clavulanate
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Ciprofloxacin
With its novel mechanism of action, Blujepa (gepotidacin) is the first drug in a new class of antibiotics called triazaacenaphthylenes. This medication works by inhibiting two of the bacterial type II topoisomerase enzymes, DNA gyrase and topoisomerase IV. By inhibiting these enzymes, gepotidacin produces a
bactericidal response in susceptible pathogens by disrupting the process of DNA replication.
With antimicrobial resistance on the rise, gepotidacin’s unique binding sites reduce the likelihood of cross-resistance with other antibacterial agents, and its novel mechanism of action has shown no significant impact with common resistance mechanisms. In other words, gepotidacin can be a viable option for organisms that display resistance to other antibiotics, and it displays a low resistance profile with bacteria in general.
Blujepa’s FDA approval came after the pivotal
phase III EAGLE-2 and EAGLE-3 clinical trials that compared twice daily gepotidacin to nitrofurantoin for a five-day course of treatment. In EAGLE-2, Blujepa proved noninferiority to nitrofurantoin with therapeutic success in 50.6% of patients compared to nitrofurantoin’s 47%. Along with this, blujepa found statistically significant superiority compared to nitrofurantoin in the EAGLE-3 trial, with therapeutic success rates of 58.5% versus 43.6%, respectively.
Throughout its clinical trials, Blujepa has shown a favorable safety profile with common adverse reactions like gastrointestinal disturbances and potential QTc prolongation. While clinical trials are ongoing for the use of Blujepa in other infectious disorders like gonorrhea, it currently provides a safe and effective option to treat uncomplicated urinary tract infections. With the rising threat of antimicrobial resistance for current antibiotics, Blujepa offers a renewed hope for mitigating this threat.
Following FDA approval on March 28, 2025, Sanofi’s
Qfitlia (fitusiran) is now the first therapy in the United States approved to treat both hemophilia A and B with or without the use of factor VIII or IX inhibitors.
This approval follows Qfitlia’s Orphan Drug Designation for hemophilia A and B, and Fast Track Designation for hemophilia A and B with and without factor VIII or IX inhibitors. Let's briefly discuss what each of these designations mean:
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Orphan Drug Designation: this designation is granted to drugs and biologics that are intended to treat rare diseases and is designed to incentivize the development of therapeutic options in areas that may have limited commercial potential. This classification provides both financial and marketing benefits to pharmaceutical companies that pursue orphan drug development, including tax credits for clinical trial costs and up to seven years of market exclusivity upon final approval.
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Fast Track Designation: with the primary goal of getting new treatments onto the market faster, this designation expedites the development and review of therapies that treat serious conditions. The FDA grants this designation to drugs that cover unmet needs and display potential advantages over existing therapies.
Hemophilia is an X-linked recessive disorder that causes the inability of blood cells to clot properly. Hemophilia may manifest as
type A or type B, depending on the clotting factor protein that is genetically deficient. Both types of hemophilia can cause prolonged bleeding and increased bruising, but hemophilia A is marked by a factor VIII deficiency while hemophilia B is associated with a deficiency in factor IX. Hemophilia A is more common than hemophilia B, but both are considered rare diseases.
Historically, treatments for hemophilia have prioritized factor replacement therapy via routine infusions that may occur
2-3 times every week. Brain Foard, Executive Vice President, Head of Specialty Care, Sanofi, explains that “This approval highlights our commitment to advancing innovation and improving care for the rare blood disorders community. Qfitlia has the potential to meaningfully change the hemophilia landscape through effective bleed protection, infrequent dosing, and simplified administration. Our robust portfolio of hemophilia treatment options continues to grow as we focus on offering protection with reduced treatment burden that best fits an individual’s needs.”
Qfitlia (fitusiran) is the first hemophilia therapy that works by reducing the levels of antithrombin in patients. Qfitlia is a small interference RNA therapeutic that utilizes
Alnylam Pharmaceutical’s ESC-GalNAc conjugate technology to promote thrombin generation and restore hemostasis.
Antithrombin is a protein in the blood that naturally inhibits thrombin production and thus prevents blood clotting. While antithrombin levels are not necessarily elevated in patients with hemophilia, lowering its activity in the coagulation cascade has proven beneficial in managing hemophilia-related bleeding complications.
This therapy is given as a subcutaneous injection, and the frequency of treatment may be determined by antithrombin activity levels. The graphic below can be used as a guidance for clinicians in determining how often to dose Qfitlia in their patients.
The
ATLAS clinical development program includes a number of phase III clinical trials that evaluate the safety and efficacy of Qfitlia. The FDA published its approval for Qfitlia after reviewing results from the three completed ATLAS trials (i.e., ATLAS-INH, ATLAS-A/B, and ATLAS-PPX) that each demonstrated a clinically meaningful bleed protection in patients with hemophilia.
While it has been clinically proven that Qfitlia can help prevent or reduce bleeding complications in adult and pediatric patients 12 years and older with hemophilia A or B, further studies are still ongoing. While the ongoing trials are not technically completed yet, Sanofi has still used some of their early results to guide therapeutic recommendations. For example, the ATLAS-OLE trial is studying the safety and efficacy of Qfitlia on an antithrombin-based dosing regimen, which Sanofi already recommends to providers for dosing purposes.
Following the completion of these early studies and upon FDA approval, Guy Young, MD, Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles, shared the following statement: " Qfitlia delivers the fewest doses of any prophylactic therapy in hemophilia, and its unique mechanism allows it to be used to treat all types of hemophilia, including with inhibitors and hemophilia B, where unmet medical needs remain. By targeting antithrombin, which can be reliably measured with an FDA-cleared blood assay, Qfitlia is proven to help rebalance hemostasis and improve bleed rates and protection."
The recent approvals of Blujepa and Qfitlia emphasize the need for advanced therapies that address unmet needs in patient care. The pharmaceutical landscape is complex, and whether a condition is common, like urinary tract infections, or uncommon, like hemophilia, every therapeutic area has the need for innovative solutions. This constant room for improvement contributes to the ever-evolving nature of healthcare practices and pharmaceuticals.
Whether it’s an urgent public health challenge or a rare disease breakthrough, new approvals like Blujepa and Qfitlia signal important shifts in how we treat and talk about care. At Ceuticon, we translate these changes into tools and insights you can use—so you’re not just staying informed, you’re staying ahead. Explore our resources to sharpen your strategy, boost your visibility, and lead with confidence in a constantly evolving field.
