The FDA delivered two notable approvals for RSV immunization, UroGen overcame a negative advisory committee vote to secure an approval in bladder cancer, and more key regulatory nods from the past month.
Moderna Expands mRNA RSV Shot—But CDC Recommendation Still Uncertain
The FDA on June 13 signed off on the broader use of Moderna’s mRNA vaccine mResvia for respiratory syncytial virus, opening up inoculation for at-risk adults aged 18 through 59 years.
For analysts at William Blair, however, approval is just the first regulatory hoop the vaccine will have to go through. “In our view, investor focus will likely shift to the CDC’s Advisory Committee on Immunization Practices (ACIP)’s next meeting,” they wrote in a note at the time, noting that the shot—and the broader vaccine market—could be battered by more headwinds still if the ACIP recommends “restrictive” guidelines.
Currently, the CDC recommends a narrower use of RSV shots, where blanket vaccination is only recommended in adults 75 and older. In those 60 through 74 years of age, the CDC endorses immunization for those who are at risk of severe RSV.
Earlier this month, Health and Human Services Secretary Robert F. Kennedy Jr. purged the ACIP of all 17 members in a move he claimed was “necessary to reestablish public confidence in vaccine science.” The next day, he named eight new panelists, many of whom have a documented history of vaccine skepticism and two who have previously spoken out specifically against the mRNA technology that underpins Moderna’s mResvia.
The new committee members met on June 25 and 26 but did not vote on mResvia.
The RSV vaccine was first approved in May 2024 for all adults 60 and above.
Merck’s RSV Antibody Clears FDA Bar to Challenge Sanofi, AstraZeneca
The FDA approved the use of Merck’s anti-RSV antibody clesrovimab in infants on June 10. Merck will market the therapy under the brand name Enflonsia.
The approval covers the use of Enflonsia to prevent RSV-related lower respiratory tract disease in newborns and infants who are entering their first season of circulating respiratory diseases. According to Merck, Enflonsia provides “rapid and durable” RSV protection persisting for five months, the length of a typical RSV season. Enflonsia is dosed at 105 mg regardless of patient weight.
Enflonsia’s approval was backed by data from the Phase IIb/III CLEVER study, which found that in preterm and full-term infants up to 1 year, the antibody reduced RSV-associated medically attended lower respiratory infections by 60.5% versus placebo. Enflonsia also cut RSV hospitalizations by 84.3% as compared with placebo.
Additional supporting data came from the Phase III SMART trial, which compared Enflonsia against Sobi’s Synagis, which is also a monoclonal antibody designed to prevent RSV. Interim results announced in October 2024 showed that Enflonsia could match Synagis’ safety and efficacy profiles through five months of observation.
With Enflonsia, Merck will now go up against Sanofi and AstraZeneca, which own the immunizing antibody Beyfortus. Approved in July 2023, Beyfortus has since become a top-performing asset for the companies, hitting blockbuster status in its first full commercial year with €1.7 billion (approximately $1.9 billion) in sales.
On June 26, the CDC’s Advisory Committee for Immunization Practices gave Merck another win, voting to recommend Enflonsia to cover babies less than eight months who are not protected by maternal antibodies.
Bayer Broadens Use of Nubeqa in Prostate Cancer
On June 4, the FDA allowed the use of Bayer’s oral androgen receptor blocker Nubeqa for patients with metastatic castration-sensitive prostate cancer (mCSPC), regardless of concurrent chemotherapy.
Before this approval, Nubeqa could only be used in the castration-sensitive patient population when it was combined with docetaxel, according to its label. The drug, taken twice daily, was originally approved in July 2019 for men whose cancers had yet to metastasize and were resistant to castration before winning an expansion in August 2022 for metastatic hormone-sensitive prostate cancer.
Nubeqa’s latest approval was backed by data from the Phase III ARANOTE trial, a randomized, double-blinded and placebo-controlled trial with nearly 670 participants. Patients were given 600-mg Nubeqa on top of androgen deprivation therapy (ADT).
Results, published in September 2024, showed that the Nubeqa regimen significantly improved radiographic progression-free survival, resulting in a 46% drop in the risk of disease progression or death as compared with placebo plus ADT. This efficacy was maintained in patients with high-volume mCSPC, who saw a 40% risk reduction, and was improved in those with low-volume disease, in which the risk was reduced by 70%.
Nubeqa brought in €1.523 billion, or around $1.75 billion for Bayer in 2024.
UroGen Surmounts AdComm Apprehension to Win Bladder Cancer Approval
Despite failing to secure the backing of an external panel of advisors, UroGen won the FDA’s approval on June 13 for its intravesical drug mitomycin for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer. UroGen will market the drug under the brand name Zusduri.
Zusduri is the first FDA-approved therapy for this indication and is meant for use in adults with recurrent disease, according to UroGen’s announcement. The drug, which is administered via a catheter directly into the bladder in an out-patient procedure, will be available “on or around” July 1.
The Phase III ENVISION study, which is still ongoing, supported the regulatory decision with a 79.6% complete response rate at three months. The single-arm pivotal trial documented an 80.6% duration of response at 18 months, according to the latest data, released in April.
Zusduri’s approval comes as a bit of a surprise, given that the FDA’s Oncologic Drugs Advisory Committee last month recommended against it—though only narrowly so. With a 5–4 split, the independent panel of experts pointed to several issues with UroGen’s application package, including the lack of a completely randomized study and the short follow-up in ENVISION.
UroGen has promised to see the ENVISION trial through to completion “to further characterize the clinical benefit of Zusduri” and will provide the FDA with yearly updates regarding the drug’s duration of response for all treated patients with ongoing complete responses.
Nuvation Wins First Commercial Approval With Lung Cancer Drug Ibtrozi
On June 12, the FDA approved Nuvation Bio’s oral ROS1 blocker taletrectinib, which will be sold as Ibtrozi. The approval covers the drug’s use in non-small cell lung cancer. Ibtrozi is Nuvation’s first commercial product.
Ibtrozi’s approval was backed by data from the Phase II TRUST-I and TRUST-II trials, which together account for “one of the largest global clinical trial programs in ROS1+ NSCLC [non-small cell lung cancer] to date,” according to Nuvation’s Wednesday release. Over 300 patients were enrolled across both trials.
Results from TRUST-I showed a confirmed overall response rate (cORR) of 90% in patients with no prior exposure to tyrosine kinase inhibitor (TKI). TRUST-II confirmed these findings with an 85% cORR. Because of the single-arm nature of the TRUST program, progression-free survival isn’t included in Ibtrozi’s label.
The TRUST studies also specifically looked at the benefits of Ibtrozi in patients with brain metastases—which Nuvation on Wednesday called “among the most common and devastating complications” in ROS1-positive NSCLC—and found high rates of treatment response. In patients with measurable brain metastasis at baseline, Ibtrozi demonstrated an intracranial response of 73% in the TKI-naïve subgroup and 63% in those who had previously been exposed to TKI treatments.
Analysts at Jefferies at the time said that while Ibtrozi’s approval was positive for Nuvation, “patience may be needed by investors with several quarters to execute and demonstrate strong launch momentum.”
BeOne Secures Approval for Twice-Daily Brukinsa Schedule
BeOne—formerly known as BeiGene—received FDA approval on June 12 for a new tablet formulation of the tyrosine kinase inhibitor Brukinsa to be taken twice-daily.
This label expansion covers all approved indications of Brukinsa, including mantle cell lymphoma, marginal zone lymphoma, chronic lymphocytic leukemia, small lymphocytic lymphoma and Waldenström’s macroglobulinemia.
“The recommended dose of Brukinsa remains at 320 mg daily,” BeOne noted in a news release announcing the approval. The new formulation delivers a 160-mg dose of the drug in each tablet, giving patients the option to “take two tablets daily rather than four of the current 80 mg capsules.” With the tablet nod, Brukinsa has become “the only BTK inhibitor to offer the flexibility of once or twice daily dosing,” with some leeway to adjust the schedule based on patient needs, the company said.
Supporting the FDA’s decision are bioequivalence data generated from two Phase I crossover trials of healthy adults, which showed that the tablets are as safe and as effective as the capsules.
Matt Shaulis, North America general manager of BeOne, said the tablet approval makes “treatment simpler and more convenient” for patients. BeOne expects to completely replace the 80-mg capsules with the 160-mg tablets by October 2025.
AbbVie’s Mavyret Becomes First Antiviral for Acute Hepatitis C
Also notching a notable regulatory nod this month is AbbVie, which on June 11 won the FDA’s first approval for an acute hepatitis C virus (HCV) drug.
The approval of Mavyret, an oral antiviral, was supported by data from a Phase III single-arm prospective trial that enrolled nearly 300 treatment-naïve adults who had acute infection. Mavyret was given once-daily for eight weeks. Results, which are reflected in the drug’s label, show that Mayret elicited a sustained virological response of 96% 12 weeks post-treatment. None of the study participants experienced virologic failure.
According to AbbVie, the label expansion allows doctors to treat patients “immediately at the time of diagnosis.” While hepatitis C infection is a curable disease, it often goes undiagnosed, as per the pharma’s news release announcing the approval. “If left untreated, people with acute HCV could progress to chronic disease, including liver-related complications” like cancer and cirrhosis.
Mavyret is a direct-acting antiviral tablet that delivers a combination of drugs: glecaprevir, a protease inhibitor that disrupts the replication of the virus’ genetic material, and pibrentasvir, which blocks viral RNA replication while also preventing the formation of the viral particle itself. First approved in August 2017, Mavyret is the first-ever product to be indicated for all types of HCV.
Mavyret brought in roughly $1.3 billion in 2024, a nearly 7% decline year-on-year.
AstraZeneca, Daiichi Sankyo Earn Accelerated Approval for Datroway in NSCLC
Datroway, an antibody-drug conjugate jointly developed and commercialized by AstraZeneca and Daiichi Sankyo, can now be used for patients with advanced or metastatic non-small cell lung cancer (NSCLC) carrying EGFR mutations. The FDA granted the biologic accelerated approval in this indication on June 24.
The regulatory decision was backed by data from a subgroup analysis of the Phase II TROPION-Lung05 study, also supported by findings from the Phase III TROPION-Lung01 trial. Findings from these studies showed that Datroway elicits a confirmed objective response rate of 45% in patients who had undergone prior lines of treatment. This includes 4.4% complete responses and 40% partial responses. Median duration of response was 6.4 months.
As per the terms of the FDA’s accelerated pathway, AstraZeneca and Daiichi Sankyo will need to validate Datroway’s clinical benefit in future confirmatory trials to maintain this approval.
Datroway won its first U.S. approval in January 2025, allowing its use in patients with unresectable or HR-positive, HER2-negative breast cancer who had been previously treated. The therapy works by binding to TROP2, a surface protein found on many tumor cells, and delivering its topoisomerase I inhibitor payload, according to its label. This mechanism damages DNA in cancer cells, ultimately leading to their death.
