“You’re going to save a fortune and this is also so good for overall health care,” Trump said at the White House event announcing the site’s launch.

The TrumpRx direct-to-consumer drug site does not sell medicines directly. Instead, it acts as a central hub that redirects users to pharmaceutical companies offering discounted drugs through their own direct-to-consumer platforms or provides printable coupons redeemable at pharmacies.

At launch, TrumpRx lists medicines from five companies that recently struck pricing agreements with the administration: AstraZeneca, Eli Lilly, EMD Serono, Novo Nordisk and Pfizer. Additional manufacturers are expected to be added in the coming months, according to the White House.

The service is intended for use by cash-paying consumers. Patients who do not have insurance or who are underinsured will likely benefit the most. Patients with insurance will likely gain less benefit, as TrumpRx purchases will probably not apply to deductibles or out-of-pocket maximums.

“If they’re able to get a drug covered by their insurance at a relatively affordable copay, then there’s not a great upside to using the TrumpRx website,” said Juliette Cubanski, deputy director of the program on Medicare Policy at KFF.

Among the well-known drugs already listed on the site are Novo Nordisk GLP-1 medicines Ozempic and Wegovy and Eli Lilly’s weight-loss injection Zepbound. Prices advertised on TrumpRx can involve significant discounts off the list price, although analysts warn that figure may be far from what consumers actually save compared with negotiated insurer prices.

TrumpRx marks the official debut of Trump’s “most favored nation” drug pricing policy intended to link US prices to those in other wealthy nations. While some have praised the effort for potentially increasing access to some expensive drugs, others say it fails to meaningfully lower prices or help most Americans with health insurance.

The acquired facility is located in Rockville, Maryland, within one of the United States’ key biotechnology clusters. It includes two cGMP manufacturing plants with a combined 60,000 liters of drug substance capacity and supports both clinical and commercial production at small and large scales. Existing products will continue to be manufactured at the site, while Samsung Biologics plans further investments to expand capacity and upgrade technologies. These planned enhancements are intended to support growing manufacturing programs and reinforce the Samsung Biologics U.S. manufacturing expansion as part of a broader strategy to deliver flexible, multi-site manufacturing options to global clients.

Under the agreement, closing is anticipated toward the end of Q1 of 2026. Samsung Biologics will acquire the Rockville assets for USD 280 million and retain more than 500 employees at the site to maintain operational continuity and workforce stability. Once integrated into the company’s global network, the Maryland facility will give customers manufacturing options in both the U.S. and Korea, supporting the reliable supply of life-saving therapeutics for American patients as the Samsung Biologics U.S. manufacturing expansion continues through the Samsung Biologics HGS acquisition.

Samsung Biologics continues to scale its global operations following the on-time completion of Bio Campus I and II and the recent securing of land for Bio Campus III, which will house dedicated R&D and manufacturing programs for new modalities. Across five plants, the company operates 785,000 liters of capacity, positioning it as the industry leader in large-scale biologics manufacturing. Its portfolio spans monoclonal antibodies, antibody-drug conjugates (ADCs), mRNA, organoid-based services, and next-generation therapies.

“This landmark acquisition is a testament to our unwavering commitment to advancing global healthcare and bolstering our manufacturing capabilities in the U.S. The investment will enable us to deepen our collaboration with federal, state, and local stakeholders to best serve our customers and partners while ensuring a reliable and stable supply of life-saving therapeutics,” said John Rim, CEO and President of Samsung Biologics.

Regis Simard, President, Global Supply Chain at GSK, said, “Today’s agreement to divest the Rockville manufacturing site to our valued long-term partner, Samsung Biologics, will secure the manufacture of two important medicines on US soil for US patients and further build GSK’s supply chain resilience. Along with GSK’s recent commitment to invest $30bn in R&D and manufacturing in the US over the next 5 years, this deal enables us to further focus on building the agility, capacity and capability needed in our manufacturing network to deliver the next generation of specialty medicines and vaccines. I am confident in a positive partnership and future for the Rockville site.”

Following the initial dosing phase, patients receive inebilizumab-cdon twice yearly, a schedule designed to simplify long-term management for individuals who may find frequent or complex regimens difficult to maintain. In addition to its newly approved indication for generalized myasthenia gravis, the therapy is also approved for adult patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder and immunoglobulin G4-related disease. Commenting on the decision, Jay Bradner, MD, executive vice president of research and development at Amgen, said in a news release, “This approval marks a significant advancement for people living with gMG. By selectively targeting CD19-positive B cells, [inebilizumab] offers a new approach to treatment that addresses a biological root cause of disease. [Inebilizumab] is conveniently dosed twice a year and delivers durable efficacy, helping people manage debilitating symptoms that can compromise daily function—including trouble breathing, speaking, and seeing.”

The FDA decision on inebilizumab-cdon for generalized myasthenia gravis was supported by findings from MINT (NCT04524273), a randomized, double-blind, placebo-controlled, parallel-group phase 3 trial evaluating efficacy and safety in adults with gMG. The study enrolled 238 patients, including 190 who were AChR-positive and 48 who were MuSK-positive. Participants were randomized to receive intravenous inebilizumab at a dose of 300 mg on days 1 and 15, with an additional dose on day 183 for AChR-positive patients, or a matching placebo. Treatment continued for 52 weeks in AChR-positive participants and 26 weeks in MuSK-positive participants. The primary endpoint assessed change from baseline in the Myasthenia Gravis Activities of Daily Living score at week 26, while a key secondary endpoint measured change in the Quantitative Myasthenia Gravis score over the same period.

Results from the trial showed that patients treated with inebilizumab achieved greater reductions in disease activity than those receiving placebo, supporting the decision as the FDA approves inebilizumab-cdon for generalized myasthenia gravis. Least-squares mean changes in MG-ADL scores were –4.2 with inebilizumab compared with –2.2 for placebo, while QMG scores declined by –4.8 versus –2.3, respectively. Richard J. Nowak, MD, MS, global principal investigator and director of the Myasthenia Gravis Clinic at Yale University, said in the news release, “[Inebilizumab] showed strong efficacy at 26 weeks in both AChR-positive and MuSK+ patients, with AChR+ patients continuing to improve through 52 weeks in MINT.” The most commonly reported adverse events included headache, cough, nasopharyngitis, infusion-related reactions, and urinary tract infections, with no higher incidence of serious adverse events observed. Manufacturers noted the potential risk of infections and possible fetal harm. Responding to the approval, Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America, said the therapy offers durable efficacy and extended treatment-free intervals for people living with gMG.

Congenital heart defects remain one of the most common abnormalities detected at birth, and about 1 in 500 newborns is classified as having a severe condition that requires urgent intervention, according to the National Institutes of Health. Carnegie Imaging for Women, an OB/GYN imaging facility affiliated with Mount Sinai, is the first center in New York City to adopt the FDA-cleared software developed by BrightHeart. The technology is now in use across the group’s three Manhattan locations, where clinicians are applying AI to improve the accuracy and efficiency of ultrasound evaluations at scale.

The study examined 200 deidentified fetal ultrasound examinations conducted between 18 and 24 weeks of gestation across 11 medical centers in two countries. Of these, 100 scans contained at least one suspicious finding. Seven obstetrician-gynecologists and seven maternal-fetal medicine specialists independently reviewed each examination, both with and without the AI tool, to assess whether the technology improved the detection of findings suspicious for severe congenital heart defects. The researchers found that AI assistance was associated with stronger detection of lesions, higher confidence scores, an 18 percent reduction in reading time, and a 19 percent improvement in confidence score, reinforcing the potential of AI-assisted fetal screening in second-trimester ultrasonography.

“AI assistance in prenatal diagnosis offers not only improved detection, but has the potential to offer significant improvement in workflow and efficiency benefits,” said corresponding author Jennifer Lam-Rachlin, MD, Assistant Clinical Professor of Obstetrics, Gynecology and Reproductive Science at the Icahn School of Medicine at Mount Sinai. “We, as clinicians, should embrace innovation and technology that is available, in order to maximize quality patient care. This technology allows for ‘leveling’ of the field of prenatal diagnosis to offer close to expert-level review of fetal ultrasounds, particularly in centers or geographical locations without fetal heart experts.”

Co-author Andrei Rebarber, MD, Director of the Division of Maternal-Fetal Medicine at Mount Sinai West, added that the findings “should prompt and encourage future research into AI-assisted software’s ability to improve detection rates, once integrated into clinical workflows, to reduce the variability and inequity of detection of congenital heart defects globally.”

BrightHeart funded the study, which brought together researchers from multiple U.S. and international institutions, including the Division of Pediatric Cardiology at the Icahn School of Medicine at Mount Sinai; New York University School of Medicine; Maternal Fetal Medicine Associates in New York City; Pediatrics – Cardiology at Stanford University School of Medicine; Palo Alto Medical Foundation, Sutter Health; the Fetal Diagnostic Center of Pasadena; Université Grenoble Alpes and CHU Grenoble Alpes in France; Medical Training Center in Rouen; Centre d’Echographie de l’Odéon and UE3C-Unité d’Explorations Cardiologiques-Cardiopathies Congénitales in Paris; Hôpital Necker-Enfants Maladies in Paris; Michigan Perinatal Associates, Corewell Health East; Wayne State University School of Medicine; Fetal Echocardiography and Perinatal Research–Valley Health System; the Division of Maternal Fetal Medicine at Pennsylvania Hospital, University of Pennsylvania; and Maternal Fetal Medicine, Perinatal Specialists of the Palm Beaches in Florida. Their collective work underscores the growing role of AI-assisted fetal screening as clinicians look to improve prenatal detection and care.

The clearance for this single-dose gene replacement therapy gives healthcare teams another way to manage motor-neuron mutation in older children, teens, and adults with confirmed SMA.

The FDA authorisation covers a one-time fixed-dose therapy that replaces the SMN1 gene without any need to adjust for age or body weight. Novartis says the treatment is built to address the underlying genetic cause of SMA by supplying a working copy of the SMN1 gene to help maintain motor-function stability.

The regulatory green light follows results from the open-label Phase IIIb STRENGTH study and the Phase III STEER trial, both of which reported meaningful gains in stabilisation and motor outcomes over 52 weeks. The clinical data also indicated a consistent safety profile. SMA itself stems from a missing or defective SMN1 gene, which disrupts production of SMN protein required for neuromuscular function.

Further programme details show that Itvisma is administered as a single intrathecal injection to sustain SMN protein expression, aligning with durable gene replacement therapy modalities that reduce treatment frequency. Novartis plans to introduce the product in the US market in December 2025.

“After redefining SMA care with the first gene replacement therapy for this challenging disease, we can now help address unmet needs across an even broader SMA population with the approval of Itvisma.” said Novartis US president Victor Bultó. “We are proud to support the SMA community by empowering patients of all ages through our innovative, one-time therapies, offering the potential to reduce the burden that comes with chronic treatment.”

• Merck’s FDA clearance introduces the first PD-1 inhibitor and ADC combination for cisplatin-ineligible MIBC patients, widening its position in the bladder cancer treatment space.
  
• Strong Keynote-905 results may shift perioperative management by elevating the Keytruda–Padcev regimen as a viable alternative to surgery alone.
  
• Intravenous and subcutaneous delivery options give providers more flexibility, which could support broader clinical uptake.

Merck said the US Food and Drug Administration (FDA) has approved Keytruda (pembrolizumab) and Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) for use alongside Padcev (enfortumab vedotin-ejfv) as a perioperative regimen for adults with muscle-invasive bladder cancer (MIBC) who cannot receive cisplatin-based chemotherapy.

Under the newly approved approach, patients are treated with Keytruda or Keytruda Qlex combined with Padcev prior to surgery, with therapy resuming after cystectomy. The decision marks the first time a PD-1 inhibitor and antibody-drug conjugate combination has been authorised for this specific patient group.

Regulators issued the approval after reviewing findings from the phase 3 Keynote-905 study, also known as EV-303, which Merck conducted in collaboration with Pfizer and Astellas. In the trial, researchers reported that after a median follow-up of 25.6 months, the combined therapy reduced the risk of event-free survival events by 60% when compared with surgery alone. Investigators also observed a 50% improvement in overall survival, while the pathologic complete response rate reached 57.1% versus 8.6% in the control arm.

The company noted that Keytruda Qlex carries a contraindication for patients with hypersensitivity to berahyaluronidase alfa, hyaluronidase or any of the formulation’s excipients. Merck also highlighted that immune-mediated reactions, potentially severe or fatal, may arise across organ systems. Infusion-related events remain a known risk for both treatments, and the therapies can cause fetal harm if administered during pregnancy.

Commenting on the clinical relevance, Dr Matthew Galsky, Lillian and Howard Stratton Professor of Medicine at Mount Sinai Tisch Cancer Center and Keynote-905 investigator, said: “Pembrolizumab plus enfortumab vedotin is poised to address a critical unmet need. Half of patients with MIBC may experience cancer recurrence even after having their bladder removed, and many of these patients are ineligible to receive cisplatin.”

Dr Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories, added: “We are honoured to provide these patients who previously had only one option — surgery — with a choice to receive their immunotherapy either intravenously or subcutaneously.”

Baby KJ, a newborn diagnosed with carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a rare and severe metabolic disorder, was treated under a single-patient, expanded-access investigational new drug (IND) application that the FDA processed within a week. The patient’s medical team developed a customised CRISPR-based therapy to repair the underlying gene defect. In May 2025, KJ became the first individual worldwide to receive a bespoke CRISPR treatment, marking a milestone that would later shape the new regulatory pathway.

Under the new framework, the FDA will consider therapies targeting well-defined molecular or cellular abnormalities rather than broadly characterised diseases. To qualify, the treatment must act on the biological source of the disorder and address a condition with a clearly documented natural history. Evidence of successful gene or molecular targeting through animal or non-animal models, or biopsy where relevant, will be required. Moreover, the product must show measurable improvement in patient outcomes, with the agency applying a lower threshold for diseases that cause progressive decline. Notably, the plausible mechanism pathway eliminates the need for traditional clinical trial data, relying instead on expanded-access programmes to gather real-world safety and efficacy information.

The FDA will grant marketing authorisation once developers demonstrate consistent success across several patients, continuing to monitor long-term results through real-world evidence (RWE). While the scheme prioritises rare disorders, it could extend to more common conditions lacking proven treatments.

The move aligns with the Trump administration’s efforts to expand access to personalised therapies and reduce long-term treatment costs.

Jon Martin, US Commercial Lead, Biosimilars and Established Brands at Organon said that POHERDY’s approval not only introduces the first PERJETA biosimilar in the US but also aligns with Organon’s broader effort to strengthen its women’s health and oncology biosimilars portfolio. “Our collaboration with Henlius is critical to our goal of making health care more sustainable for US patients.”

The approval of the PERJETA biosimilar covers POHERDY’s use as a HER2/neu receptor antagonist in combination with trastuzumab and docetaxel for adults diagnosed with HER2-positive metastatic breast cancer who have not previously received anti-HER2 therapy or chemotherapy for metastatic disease. Additional indications include its combined use with trastuzumab and chemotherapy for adults undergoing neoadjuvant treatment for HER2-positive, locally advanced, inflammatory, or early stage breast cancer greater than 2 cm or node positive, as well as adjuvant treatment for high-risk HER2-positive early breast cancer.

Safety information highlights the potential for pertuzumab products to cause subclinical and clinical cardiac failure, including decreased left ventricular ejection fraction (LVEF) and congestive heart failure (CHF). Clinicians are advised to monitor cardiac function and discontinue treatment if a clinically significant decline is confirmed. The product also carries warnings regarding embryo-fetal death and birth defects and recommends effective contraception.

Regulators based their decision on a complete data package incorporating analytical comparison, clinical pharmacokinetic studies, and head-to-head clinical trials designed to show POHERDY’s high similarity and interchangeability with PERJETA in safety, purity, and potency (safety and effectiveness).

Henlius and Organon first established their partnership in 2022 through a license and supply agreement that granted Organon exclusive commercialization rights to several biosimilars, including POHERDY, in all markets except China. According to both companies, POHERDY’s FDA clearance expands their shared oncology portfolio and strengthens their ability to bring quality biologics to a broader patient population.

HRT has long been used to ease menopausal symptoms, but its usage fell sharply in the early 2000s after the FDA issued boxed warnings based on a Women’s Health Initiative study. That study reported a statistically non-significant rise in breast cancer diagnoses, involved participants with an average age of 63 years, well beyond the typical onset of menopause, and used a hormone formulation that is no longer standard.

Following a full scientific review, an expert panel meeting in July, and a public comment process, the FDA is now moving to remove those broad black box warnings. The agency is coordinating with manufacturers to revise product labels and eliminate references to risks involving cardiovascular disease, breast cancer, and probable dementia. One exception remains: the boxed warning for endometrial cancer will stay in place for systemic estrogen-alone medications.

As estrogen and progesterone decline during menopause, FDA-approved HRT, whether estrogen-progesterone combinations or estrogen alone for women without a uterus, can be used to address symptoms such as night sweats, hot flashes, bone loss, and sleep disruption.

Randomized studies indicate that starting HRT within 10 years of menopause onset, typically before age 60, can lower all-cause mortality and fracture risk. Women may also see reductions of up to 50% in cardiovascular diseases, a 35% decrease in Alzheimer’s disease, and a 50 to 60% drop in bone fractures. The FDA’s guidance continues to recommend initiating systemic HRT before age 60 or within a decade of menopause onset, with final decisions resting between patients and their clinicians.

Alongside the warning changes, the FDA has approved two new treatments for menopause-related symptoms. One is a generic version of Premarin (conjugated estrogens), marking the first such approval in more than 30 years and expected to improve access while matching the brand’s quality and effectiveness. The second is a non-hormonal therapy designed for moderate to severe vasomotor symptoms, offering an alternative for women who cannot or prefer not to take hormone therapy.

“We continue to execute our science-led business development strategy, augmenting our pipeline with CD388, a potentially first-in-class, long-acting antiviral designed to prevent influenza in individuals at higher risk of complications,” said Robert M. Davis, chairman and chief executive officer, Merck. “We intend to build on the Cidara team’s remarkable progress and are confident that CD388 has the potential to be another important driver of growth through the next decade, creating real value for shareholders.”

At the center of the acquisition of Cidara Therapeutics is Cidara’s lead candidate, CD388, which pairs a small molecule neuraminidase inhibitor with a proprietary Fc fragment of a human antibody engineered to prevent influenza A and B. The program is being tested in the Phase 3 ANCHOR study (NCT07159763) involving adult and adolescent participants at elevated risk of influenza-related complications. The U.S. Food and Drug Administration granted Breakthrough Therapy Designation following data from the Phase 2b NAVIGATE study (NCT06609460), which met all primary and secondary endpoints tied to preventing symptomatic laboratory-confirmed influenza in healthy adults ages 18 to 64. CD388 also previously received Fast Track Designation from the FDA.

“This acquisition expands and complements our respiratory portfolio and pipeline. Influenza continues to pose a significant global health threat, causing widespread illness, morbidity and death each year especially in older adults and immunocompromised individuals, such as those with cancer and chronic diseases,” added Dr. Dean Y. Li, president, Merck Research Laboratories. “CD388 is a novel late-phase candidate with important strain-agnostic properties being evaluated for the prevention of symptomatic influenza in high-risk individuals.”