A 64-week, late-stage study demonstrated that participants who took 25 mg of oral semaglutide once a day went on to lose an average of 16.6% of their body weight, as compared with 2.7% for those on a placebo. The pill was given a nod for chronic weight management within adults with obesity or overweight and a minimum of one related health condition, therefore widening the potential patient pool at a time when the insurers, employers, and governments are battling it out with spiraling healthcare expenses related to obesity. It could also help open the door to tens of millions of untapped patients in an international market, forecast to be somewhere around $150 billion a year by the next decade. As per chief AI officer Anand Iyer at Welldoc, a telehealth firm, there is going to be a huge uptake in the patient base that is about to be seen as new indications open up and as the oral versions hit the market. Novo’s executive vice president of U.S. operations, David Moore, said that a daily pill could also boost the interest and uptake of the drug. Novo is apparently manufacturing the pill in the United States in North Carolina and has been building up supplies of the pill for quite some time now to ensure that it has enough supply.

Around 40% of American adults are obese, as per the U.S. government data, and almost 12% say that they are at present on GLP-1 drugs, as per a poll published in November 2025 by KFF, the health policy research organization. Novo apparently had a first-to-market advantage in terms of injectables; however, it initially struggled to meet that explosive demand. Eventually, Lilly went ahead with its Zepbound, which now goes on to lead when it comes to weekly U.S. prescriptions. Novo, along with analysts, says that a weight-loss pill would very well address the injection hesitancy and hence lead to expansion in access.

According to managing director and partner at BCG, Christopher Chrisman, the pills are not going to displace or replace the injections, adding that some patients may prefer to go ahead with their weekly injections. However, the pills do offer clear benefits to some people. There is indeed a level of travel convenience and no requirement to have a fridge,” he added. Novo remarked that the 1.5-milligram starting dose of the Wegovy pill is going to be available in early January 2026. Novo as well as Lilly had gone on to agree to provide starter doses in terms of their weight-loss pills at $149 for every month for the U.S. government Medicare and Medicaid health insurance programs and also to cash-paying customers through the direct-to-consumer TrumpRx site from the White House.

Novo recently slashed the cash price for Wegovy to $349 per month, from $499. Mike Doustdar, the Novo CEO, said that in November 2025, people making use of the weight-loss drugs showed more consumer-like behavior as compared to its traditional diabetes patients, therefore acknowledging that the company is required to adapt to this and also bring in the new expertise. Whether another semaglutide product can go ahead and solve the current ills of Novo remains to be seen. The weight loss pill from Novo Nordisk, oral semaglutide, has to be taken in the morning on an empty stomach, which is 30 minutes prior to eating, drinking, or using any kind of other oral medication.

According to the Chief Medical Officer and Head of Global Product Development at Roche, Levi Garraway, MD, PhD, since follicular lymphoma often needs lifelong management, decreasing the burden of care for such individuals is indeed of major importance. Due to this FDA approval, treatment can now be administered in about one minute, which prominently decreases the time patients spend within the clinic and helps to sync care along with their individual requirements as well as preferences.

It is well to be noted that VELO decreases the treatment administration time with an approx. one-minute injection as compared with a 2-4 hour intravenous (IV) infusion. Like Lunsumio, which is administered intravenously, Lunsumio VELO by Roche can be administered outpatient and is a fixed-duration treatment that is given for a defined period, and that could be as short as six months. By contrast, treat-to-progression treatment alternatives are designed to be given to patients indefinitely until the time of disease progression or till treatment can no longer be tolerated. Tennessee Oncology and One Oncology’s Dr. Ian Flinn, MD, PhD, says that this approval is a major step when it comes to broadening access to effective treatments for people who are living with follicular lymphoma. Due to its manageable cytokine release syndrome profile as well as decreased administration time, Lunsumio VELO helps oncologists to roll out advanced care across the community practice settings.

Interestingly, the FDA approval has received support from the primary analysis of the GO29781 study, which evaluated Lunsumio VELO across patients having third-line or later (3L+ FL). Inferences showed that the objective response rate as well as the complete response rate within patients treated with Lunsumio VELO were 75% (95% confidence interval [CI]: 64–83%) and 59% (95% CI: 48–69%), respectively. The median duration when it comes to response was 22.4 months – 95% CI: 16.8–22.8. The most common adverse reactions (≥20%) were the injection site reactions, such as fatigue, rash, cytokine release syndrome (CRS), COVID-19 infection, and musculoskeletal pain, as well as diarrhea. Notably, the CRS rate was 30%, and events were mostly low grade, i.e., Grade 1–2 (28%) and Grade 3 (2.1%) occurred in Cycle 1, and all resolved post a median duration of two days (range: 1–15). Apparently, CRS can be severe as well as life-threatening.

It is well to be noted that Lunsumio IV was the first bispecific antibody that was approved for 3L+ FL. Long-term data from the SC as well as IV arms of the GO29781 study were presented at the 67th American Society of Hematology Annual Meeting and Exposition. These data have been submitted to other healthcare authorities throughout the world. Recently, the European Commission went on to grant a conditional marketing authorization of Lunsumio SC when it comes to the treatment of adult patients having R/R FL post two or more lines of systemic therapy.

Roche goes on to advance its bispecific antibody programme within the gamut of lymphoma, with ongoing phase III studies assessing Lunsumio and Lunsumio VELO within the earlier lines of treatment. This goes on to include the SUNMO study, which investigates the Lunsumio VELO in combination with Polivy® (polatuzumab vedotin) in the second-line or later large B-cell lymphoma, as well as the MorningLyte study, which investigates the Lunsumio VELO in combination with lenalidomide in past untreated FL.

The Cytokinetics Myqorzo approval follows a period of volatility for the biotech, which was founded in 1997 and has only now brought its first product to market. The company faced setbacks when a late-stage ALS program failed and another cardiovascular therapy was rejected by regulators, but Myqorzo’s progress reshaped its development outlook.  Like Camzyos, Myqorzo is a cardiac myosin inhibitor that works by relaxing heart muscle contraction to improve cardiac function, and both drugs are approved for the obstructive form of hypertrophic cardiomyopathy. But differences in how the therapies are prescribed could influence how the market develops. Myqorzo carries a heart failure warning similar to Camzyos, while its risk mitigation requirements are less restrictive. RBC Capital Markets analyst Leonid Timashev wrote that Myqorzo’s protocol allows more flexible dose adjustments, less frequent echocardiograms, and minimal drug-interaction monitoring, factors he said “should materially lower barriers to prescribing” compared with Camzyos.

Despite the significance of the Cytokinetics Myqorzo approval, investor reaction was muted, with analysts noting that labeling outcomes largely matched expectations. Stifel analyst James Condulis said there remains “debate” around the “extent” of Myqorzo’s differentiation from Camzyos, even as attention turns to upcoming clinical data. Cytokinetics is expected to report results from a study evaluating Myqorzo in patients with the non-obstructive form of hypertrophic cardiomyopathy, a setting in which Camzyos failed in a Phase 3 trial. Some analysts believe Myqorzo’s broader therapeutic dosing range could offer an advantage, though outcomes remain to be seen. “We’re bullish and expect [Myqorzo] to become the best [and] only drug for the entire HCM spectrum,” Condulis wrote.

Pricing for Myqorzo is expected to be “in line with” Camzyos, according to a company spokesperson, with details to be disclosed ahead of launch. Camzyos entered the market with an annual wholesale acquisition cost of $89,500. Myqorzo has also received regulatory clearance in China, extending its geographic footprint beyond the U.S. market. As Cytokinetics transitions from a development-stage biotech to a commercial drug company, the Cytokinetics Myqorzo FDA approval represents a pivotal step in establishing a long-term presence in cardiovascular therapeutics.

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.

“These approvals mark a significant milestone for treatment options for patients with uncomplicated urogenital gonorrhea,” said Adam Sherwat, M.D., director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research (CDER). Gonorrhea, caused by the bacterium Neisseria gonorrhoeae (N. gonorrhoeae), is a localized infection of the urethra or cervix that can lead to painful urination, discharge, and swelling, and may progress to infertility if left untreated. As the FDA approves two oral therapies, the agency highlighted the importance of expanding treatment choices as clinical practice has shifted from combination therapy with ceftriaxone and azithromycin to reliance on a single ceftriaxone injection.

Approval of Nuzolvence was supported by a study involving 930 patients with uncomplicated urogenital gonorrhea. Two-thirds of participants received a single 3-gram dose of Nuzolvence dissolved in water, while the remaining patients received standard treatment consisting of a ceftriaxone shot and an azithromycin pill. Bacterial clearance assessed 4 to 8 days after treatment showed cure rates of 91% for Nuzolvence and 96% for standard treatment, demonstrating comparable effectiveness. Blujepa was evaluated in a separate study of 628 patients, where participants received either two 3,000 mg doses taken 10 to 12 hours apart or standard therapy. Clearance rates assessed 4 to 10 days after treatment showed cure rates of 93% for Blujepa and 91% for standard treatment.

The safety results were in line with what is already known about both drugs. The most common adverse events reported with Nuzolvence included low white blood cell counts, headache, dizziness, nausea, and diarrhea, while Blujepa’s most frequently reported side effects included gastrointestinal symptoms, headache, fatigue, excessive sweating, and dizziness, with warnings related to heart rhythm effects, acetylcholinesterase inhibition, and allergic reactions. Both therapies received Fast Track, Qualified Infectious Disease Product, and Priority Review designations. As part of the FDA approval for gonorrhea medications, approval of Nuzolvence was granted to Entasis Therapeutics, while approval for Blujepa was granted to GSK.

A Change in Regulatory Pattern

Historically, if we talk of the FDA, it has mostly depended upon two adequate as well as well-controlled trials as the yardsticks so as to demonstrate the safety and efficacy of a product. This approach went on to offer confirmatory evidence along with a decreased set of uncertainty within the regulatory decision-making. Over the past decade, the agency has, however, gone on to demonstrate an increasing flexibility, especially by way of orphan drug programs and accelerated approval pathways, along with situations involving high unmet medical requirements.

There are many developers who have already submitted a single clinical study that is supplemented by real-world evidence, supportive data, or secondary analyses. The new policy looks forward to formalizing what has gradually gone on to become quite a common practice.

Details of the New Default Benchmark 

FDA Commissioner Makary stressed the fact that while the default is now going to be one pivotal trial, the FDA is not going to eliminate the option or, for that matter, the need for a second study wherever it is warranted. Products having complex mechanisms, early-stage data that’s inconsistent, narrow therapeutic windows, or also higher safety risks may very well still require more extensive evidence packages.

This sort of approach looks forward to introducing efficiency without compromising on the scientific rigor. The FDA, apparently, still remains responsible in order to ensure that the evidence supporting approval is strong and reproducible as well as dependable.

What are the implications for drugmakers and patients?

When we talk of the industry stakeholders, the policy shift could as well lead to decreased development timelines, lower research expenses, and also earlier submission opportunities. It may also go ahead and encourage certain smaller companies and innovators who are working in niche therapeutic spaces by way of lowering the evidentiary threshold for entry within the regulatory process.

As far as the patients are concerned, especially those having rare or serious diseases, this transition may help with faster access to new therapies. Simultaneously, the FDA also maintains its oversight mechanisms in order to track the post-marketing safety and go ahead and confirm the long-term outcomes.

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.”

The back-to-back approvals position Lynkuet as one of Bayer’s most strategically important pipeline assets, reflecting the commercial potential of a product aimed at a rapidly expanding patient base and a therapeutic category undergoing accelerated innovation. With decisions now finalised on both sides of the Atlantic, Bayer is preparing to scale the rollout across major pharmaceutical markets.

The European Commission has authorised Lynkuet menopause treatment (elinzanetant) for moderate to severe VMS linked to menopause or endocrine treatment for breast cancer. This follows the FDA’s approval, granted roughly one month earlier, for the menopause-related VMS indication in the U.S. The drug, an oral neurokinin (NK) 1 and 3 antagonist, enters a competitive field currently led by Astellas’ NK 3 antagonist Veozah/Veoza (fezolinetant), which received its first approval in 2023 for menopause-related VMS.

Market interest in non-hormonal VMS therapies has grown steadily, with Astellas reporting that Veozah/Veoza sales rose nearly 55% in the first six months of the current fiscal year to approximately $146 million. However, updated FDA labeling that includes a warning on rare but serious liver injury has reshaped expectations for the product’s long-term growth. Lynkuet menopause treatment faces related considerations, as its use has been linked to elevated liver enzyme levels requiring regular blood-test monitoring.

Bayer has positioned Lynkuet as its next major pharmaceutical launch, planning commercial availability in the U.S. this month. The therapy has now been approved in the U.S., EU, UK, Australia, Canada, and Switzerland. Demand potential is extensive: an estimated 1.2 billion women worldwide are expected to experience menopause by 2030, while millions receive breast cancer diagnoses each year, with about 70% presenting HR-positive tumours treated with endocrine therapies that commonly induce VMS.

“Menopause symptoms, including hot flashes, can greatly affect women’s quality of life,” said Nick Panay, a gynaecologist at Imperial College London and a principal investigator in one of the supporting clinical trials. “This approval is an important milestone in the area of menopause care as it expands therapeutic options for women experiencing distressing menopause symptoms with a novel targeted hormone-free treatment and facilitates healthcare professionals to achieve more personalised treatment,” he added.

A potential variable for the drug’s commercial trajectory is the FDA’s recent removal of black-box warnings related to breast cancer, cardiovascular disease, and dementia from hormone replacement therapies (HRT), a shift that could renew interest in hormonal treatments and influence uptake of newer non-hormonal options.