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The FDA’s
Center for Drug Evaluation and Research (CDER) is responsible for the continued evaluation and approval of potential new drug products. After ensuring that a product is both safe and effective, dozens of novel approvals are approved to enter the commercial market each year. Novel drug approvals are important as they often signify major medical advancements, address unmet healthcare needs, and offer improved treatment options to distinct patient populations.
While these approvals work to enhance patient care and drive innovation within the pharmaceutical industry, many novel approvals may go unnoticed by the public as they typically impact a small portion of patients with specialized illnesses. By highlighting novel approvals each month, Ceuticon aims to equip growing professionals with the tools they may need to make a stronger impact on patient care delivery.
The following content will cover 3 new drug products that have been approved by the FDA during April 2025:
This article will take a deeper look into each of these newly approved medications, exploring the pharmacologic properties and approval pathways associated with them.
Following its
accelerated approval from the FDA on April 2, 2025, atrasentan (
Vanrafia; Novartis) is the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary
IgA nephropathy (IgAN). Primary IgA nephropathy is characterized by the buildup of immunoglobulin A protein in the kidneys, which can lead to inflammation and damage. As a result of this, patients with IgA nephropathy are at a high risk of rapidly progressing kidney disease, and there has been an insistent need for novel treatment options that better manage disease progression and reduce therapeutic toxicity.
“[Vanrafia’s] approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,” explained Richard Lafayette, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member.
As a highly selective endothelin A receptor antagonist,
atrasentan displays a heightened ability to selectively prevent the binding of endothelin-1 (ET-1) to endothelin A receptors (ETAR) on patient renal cells. In IgA nephropathy, patients experience an upregulation of endothelin-1, which can lead to mesangial cell proliferation, podocyte damage, inflammation, and fibrosis – key drivers of proteinuria and progressive
glomerulosclerosis. By blocking the activation of endothelin A receptors, atrasentan reduces the harmful downstream effects that many patients with IgA nephropathy experience.
Vanrafia is only FDA approved for use in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, defined as a
urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g. Vanrafia is available for use in this patient population as an 0.75mg tablet taken once daily.
Vanrafia may interact with OATP1B1/1B3 inhibitors, increasing exposure to atrasentan in the body and potentially increasing the risk of adverse effects. OATP1B1/1B3 inhibitors may include, but are not limited to, antivirals like ritonavir, antibiotics like rifampin, and immunosuppressants like cyclosporine. It should also be noted that Vanrafia is a CYP3A substrate, and concomitant use with moderate to strong
CYP3A inducers should be avoided, as it may decrease the therapeutic effects of atrasentan.
Vanrafia is absolutely contraindicated in pregnancy, and it is recommended that contraceptives be used before, during, and 2 weeks after the duration of Vanrafia treatment. For biological males, it is also noteworthy that Vanrafia may reduce sperm count during therapy via adverse effects on spermatogenesis. Since some endothelin receptor antagonists have been linked to liver failure, patients taking atrasentan should receive baseline liver enzyme testing with repetition throughout treatment as clinically indicated.
Vanrafia’s accelerated FDA approval can be attributed to preliminary findings from the phase 3
ALIGN trial. This 1:1 placebo-controlled trial evaluated efficacy, as measured by urine protein to creatinine ratio, and safety measured through adverse effects after 36 weeks of therapy.
From an efficacy standpoint, data from the ALIGN study showed a 36.1% reduction in proteinuria (P<0.0001) versus placebo with improvements first seen at week 6 and sustained through week 36. The safety profile throughout this study was ultimately favorable, with notable adverse effects including peripheral edema (10%) and anemia (6%).
While the safety and efficacy results after 36 weeks did warrant accelerated approval, continued approval may be contingent upon verifiable clinical benefits at week 136 of this ongoing study. The FDA will be looking for a reduced
estimated glomerular filtration rate (eGFR) at this endpoint to better determine Vanrafia’s ability to slow disease progression.
Best clinical practice for the management of IgA nephropathy typically includes the use of a renin-angiotensin system inhibitor (i.e., ACE inhibitors or ARBs) for blood pressure control and an SGLT2 inhibitor (e.g., empagliflozin; Jardiance) for supportive care. Novartis, as supported through the ALIGN trial, suggests the use of Vanrafia alongside current SGLT2 inhibitor and renin-angiotensin system inhibitor therapy. Systemic corticosteroids may be used in high-risk patients, though utilization is generally limited due to concerns of toxicity.
Filspari (sparsentan) is another endothelin receptor antagonist that was initially approved for use in patients with IgA nephropathy in 2023. While head-to-head data is lacking between atrasentan and sparsentan, the introduction of Vanrafia introduces competition among these products, which can be extremely useful in providing patients with more treatment options and negotiating lower price points.
On April 23, 2025, the biopharmaceutical company
Akeso, Inc. announced the approval of its differentiated programmed cell death protein 1 (PD-1) monoclonal antibody, named penpulimab-kcqx. Akeso has not disclosed a potential brand name for the drug at this time but plans to further its development and commercialization through a joint venture with Chia Tai-Tianqing Pharmaceutical Group. Penpulimab-kcqx received its approval to treat adults with recurrent or metastatic non-keratinizing
nasopharyngeal carcinoma (NPC).
Penpulimab-kcqx is a humanized
monoclonal antibody that binds to the programmed cell death protein 1 (PD-1) receptor on T cells. The PD-1 receptors are ultimately responsible for the inhibition of T cell activation, so by blocking this receptor, penpulimab-kcqx allows patient T cells to recognize and attack tumor cells more effectively.
As previously stated, penpulimab-kcqx is approved for use in adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma, which is a type of cancer that originates in the upper region of the throat. For patients with this disease state, penpulimab-kcqx may be used in one of two ways:
Penpulimab-kcqx is given as a
200mg intravenous injection with frequency of therapy ranging from 2-3 weeks, depending on a patient’s clinical status.
According to Akeso, the phase 3 AK105-304 trial played a vital role in supporting penpulimab’s FDA approval. Compared to placebo, the use of penpulimab in conjunction with standard chemotherapy throughout this trial resulted in an increased objective response rate (ORR) as well as a 4-month longer duration of response. AK105-304 also displayed a favorable safety profile, with findings similar to those of other PD-1 inhibitors and minimal discontinuation due to adverse events.
While the incidence of nasopharyngeal cancer in the United States is considered rare, it often goes unnoticed until it has reached an advanced stage. According to the
American Cancer Society, typical regimens for treating nasopharyngeal cancer include chemotherapy and/or immunotherapy. PD-1 inhibitors like pembrolizumab (
Keytruda) and toripalimab (
Loqtorzi) are already on the market for treating nasopharyngeal cancer, and adding penpulimab-kcqx to the landscape will allow for a wider variety of therapeutic choices and therefore be utilized to negotiate better coverage options to promote accessibility.
Johnson and Johnson reached a significant milestone on April 29, 2025, receiving FDA approval for its new drug product Imaavy (nipocalimab-aahu). Indicated for the treatment of myasthenia gravis, Imaavy offers a new treatment option for patients that can potentially provide lasting disease control.
To understand Imaavy’s mechanism of action, it is first important to understand the pathophysiologic background of myasthenia gravis. In this disease state, IgG autoantibodies mistakenly attack acetylcholine receptors at the neuromuscular junction. As acetylcholine receptors are responsible for initiating muscle contractions, the reduced number of functioning receptors in patients with myasthenia gravis leads to muscle fatigue and the hallmark facial droops associated with this condition.
Imaavy belongs to a class of medications called neonatal Fc receptor (FcRn) blockers. As a humanized IgG1 monoclonal antibody, this product binds to the neonatal Fc receptor to accelerate the degradation of excess IgG. What sets Imaavy apart from other medications in this class is its ability to provide long-lasting, targeted binding for prolonged therapeutic effects.
Nipocalimab-aahu is approved for use in adults and pediatrics ≥ 12 years of age who are anti-muscle-specific kinase (MuSK) or anti-acetylcholine receptor (AChR) antibody positive. In other words, each of these receptors bind to IgG, and roughly 90% of patients with generalized myasthenia gravis are classified under one of these subtypes.
“We consistently hear from individuals living with myasthenia gravis who are hopeful for new treatment options that may help bring greater stability, independence and predictability to their lives,” said Samantha Masterson, President and CEO, Myasthenia Gravis Foundation of America. “Today’s announcement provides another option which could help address the constant uncertainty and heavy physical and mental toll that MG symptom relapse presents to patients and their families.”
The ongoing phase 3 Vivacity-MG3 and Vibrance-MG studies were pivotal in achieving approval for Imaavy. In comparison to placebo with standard of care, patients who received Imaavy along with standard of care experienced superior disease control, reduced autoantibody levels, and maintained improvements up to 20-months of follow-up. All patients assigned to take Imaavy received an intravenous infusion loading dose followed by one maintenance dose every 2 weeks.
Throughout this trial, according to Dr. Nicholas J. Silvestri, M.D., Professor of Neurology at University of Buffalo, “Patients experienced substantial symptom relief and lasting disease control that translated into better daily function and did not fade over 24 weeks in the pivotal Vivacity-MG3 study.
Johnson and Johnson anticipates widespread adoption of Imaavy for the treatment of generalized myasthenia gravis and has already launched a
patient support program for patients in the United States. In using this program, Johnson and Johnson states that qualifying patients with commercial insurance can receive their first treatment dose in as little as one week with a cost of $0 for infusion services.
April’s novel approvals underscore the growing impact of targeted therapies in managing autoimmune disease states. While many existing autoimmune therapies exert broad immunosuppression or cytotoxic effects, each of the products mentioned above offer a new hope to patients in managing their condition and reducing adverse effects.
At Ceuticon, we believe that keeping up with drug approvals isn’t just about staying informed—it’s about staying prepared. With each new therapy, professionals have the opportunity to deepen their clinical fluency, expand their strategic insight, and better support patient outcomes. Our monthly FDA approval roundups are built to help you do just that. Use them to grow your knowledge, sharpen your recommendations, and lead with credibility in a fast-moving field.
