Abigail Brooks is an assistant managing editor for 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

The US Food and Drug Administration has approved Amgen’s inebilizumab-cdon (Uplizna) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive, offering patients a new targeted treatment option that has the potential for long-term disease control with just 2 doses a year after 2 initial loading doses.

As described in a December 11, 2025, release from the Company, the approval marks inebilizumab-cdon’s third indication and is supported by data from the Myasthenia Gravis Inebilizumab Trial (MINT), the largest phase 3 biologic study to include both AChR+ and MuSK+ patients, and the first to successfully incorporate a steroid taper into its protocol.

"Managing a rare and chronic illness can mean facing unpredictable relapsing symptoms and demanding treatment schedules," Samantha Masterson, president and chief executive officer of the Myasthenia Gravis Foundation of America, said in a statement.

 "This approval marks an important milestone, offering durable efficacy and a dosing schedule that provides people living with generalized myasthenia gravis 6 months of treatment-free time between maintenance doses."

Inebilizumab-cdon is a humanized monoclonal antibody (mAb) that causes targeted and sustained depletion of key cells that contribute to underlying disease process (autoantibody-producing CD19+ B cells, including plasmablasts and some plasma cells). It has previously been approved by the FDA for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD) in June 2020, and for the treatment of adult patients with Immunoglobulin G4-related disease (IgG4-RD) in April 2025.

MINT is a randomized, double-blind, placebo-controlled, parallel-group trial designed to evaluate the efficacy and safety of inebilizumab-cdon in adults with gMG. The trial enrolled 238 adults with gMG, including 190 patients who are AChR+ and 48 patients who are MuSK+.

The primary endpoint was change from baseline in MG-ADL score at week 26 in the combined study population. Key secondary endpoints included change from baseline in QMG scores in the combined study population; change from baseline in MG-ADL score at week 26 for the AChR+ cohort and separately the MuSK+ cohort; and change from baseline in QMG score at Week 26 for the AChR+ cohort and separately the MuSK+ cohort. MINT also includes an optional 3-year open-label treatment period.

In MINT, at week 26, inebilizumab-cdon demonstrated a 1.9-point difference in the MG-ADL score compared with placebo (-4.2 vs -2.2; 

<.0001). Benefits in the AChR+ patient subgroup continued through week 52, the longest randomized-controlled period for a phase 3 trial in gMG, with an exploratory analysis of AChR+ patients showing a 2.8-point difference in MG-ADL for inebilizumab-cdon compared with placebo (-4.7 vs -1.9; 95% CI, −3.9 to −1.7).

"[Inebilizumab-cdon] showed strong efficacy at 26 weeks in both AChR+ and MuSK+ patients, with AChR+ patients continuing to improve through 52 weeks in MINT," said Richard Nowak, MD, MS, global principal investigator and director of the Myasthenia Gravis Clinic at Yale University.

 "MINT also uniquely required steroid tapering, recognizing that long-term steroid use adds to the overall burden of disease. This approval brings a new first-in-class approach to gMG, expanding treatment options for clinicians and patients."

Amgen. FDA APPROVES UPLIZNA® FOR ADULTS WITH GENERALIZED MYASTHENIA GRAVIS. December 11, 2025. Accessed December 12, 2025. 

https://www.amgen.com/newsroom/press-releases/2025/12/fda-approves-uplizna-for-adults-with-generalized-myasthenia-gravis

Nowak RJ, Benatar M, Ciafaloni M, et al. A Phase 3 Trial of Inebilizumab in Generalized Myasthenia Gravis. 

Articles

The FDA has approved Amgen's inebilizumab-cdon for treating generalized myasthenia gravis (gMG) in adults with specific antibodies, marking its third indication. This approval is based on the Myasthenia Gravis Inebilizumab Trial (MINT), the largest phase 3 study for AChR+ and MuSK+ patients, which demonstrated significant efficacy and safety. Inebilizumab-cdon, a humanized monoclonal antibody, offers a novel treatment approach with a biannual dosing schedule. The trial showed significant improvements in MG-ADL scores, particularly in AChR+ patients, and incorporated a steroid tapering protocol.

The approval makes inebilizumab-cdon the first and only CD19-targeted B cell therapy indicated for anti-AChR and anti-MuSK Ab+ generalized myasthenia gravis.

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FDA Approves Inebilizumab-cdon (Uplizna) for Generalized Myasthenia Gravis

Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. She joined MJH Life Sciences in September 2023 after graduating from The College of New Jersey with a bachelor’s degree in journalism and professional writing. In her free time, she enjoys creative writing and reading.

Credit: The US Food & Drug Administration 

BioCryst announced on Friday, December 12, 2025, that the 

 approved berotralstat (ORLADEYO) oral granules for patients with 

This follows berotralstat’s 2020 FDA approval for the prevention of HAE attacks in adults and children aged ≥ 12 years.

“Pediatric patients living with HAE can experience significant burdens, not only from the disease itself but also from its treatments, affecting their physical, psychosocial, and developmental well-being,” said Raffi Tachdjian, MD, associate clinical professor of medicine & pediatrics at the David Geffen School of Medicine, University of California Los Angeles. “This new oral pellet formulation of ORLADEYO provides a more easily administered long-term-prophylactic option to help children with HAE better manage their disease, especially when the experience of infusions or injections is not ideal.”

This past year the FDA approved many treatments for HAE—

garadacimab-gxii (Andembry) in June, sebetralstat (Ekterly) in July, and donidalorsen (Dawnzera) in August

 Numerous experts said that, despite these approvals, a critical HAE care gap was the limited treatment options for children under 12 years.

“We are excited to make ORLADEYO oral pellets available to children living with HAE. The use of prophylactic therapy has been limited among younger children compared to adults, in part because of the lack of appropriate, less burdensome treatment options. ORLADEYO oral pellets have the potential to change how a new generation of children with HAE and their caregivers manage this condition, giving them more freedom to live a normal life,” said Charlie Gayer, president and chief commercial officer of BioCryst.

Until now, children under 12 with HAE could only take Berinert (plasma-derived esterase inhibitor (human)), Cinryze (C1 esterase inhibitor (human)), haegarda (c1 esterase inhibitor subcutaneous (human)), and lanadelumab-flyo (Takhzyro).

 Of these previously approved treatments, only Berinert and lanadelumab-flyo are approved to target HAE attacks in children as young as 2 years old.

 This berotralstat approval adds another treatment option for children experiencing HAE attacks.

Berotralstat, a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein, is the first oral therapy designed specifically to prevent attacks of HAE.

 Taking 1 capsule a day can prevent attacks by reducing the activity of plasma kallikrein.

The pediatric approval was based on positive interim data from APeX-P, the largest clinical trial evaluating prophylactic therapy for HAE in 29 patients aged 2 to 11 years (48.3% females; mean age, 8 years, mean HAE onset age, 2 years).

 The study, led by Jolanta Bernatoniene, MD, PhD, from Bristol Royal Hospital for Children in the UK, showed berotralstat provided early and sustained reductions in monthly attack rates. At a steady state, berotralstat reached peak concentration at a median of 3.9 hours (range, 0.9 – 6.0 hours).

 Investigators noted an increase in QT prolongation at dosages over the recommended 150 mg daily dose.

Berotralstat was well tolerated and had a consistent safety profile in children as compared to adults. Common adverse events included abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

“As we mark 5 years since ORLADEYO first transformed care for those with HAE ages 12 and older, this approval extends the benefits of oral prophylactic therapy to a vulnerable and important part of the HAE community, children ages 2 to less than 12. We couldn’t be more excited to bring this treatment option to these kids and their caregivers,” said Jon Stonehouse, chief executive officer of BioCryst. “ORLADEYO has been prescribed to more than 3,500 patients in the U.S. to date, and we’re honored to now bring an oral pellet formulation to children and their caregivers, answering the community’s heartfelt call for a long-term prophylactic treatment option that meets the unique needs of children."

BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to <12 Years. BioCryst. Published December 12, 2025. Accessed December 12, 2025.

 https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-fda-approval-orladeyor-berotralstat-oral 

Kunzmann K. FDA Approves Berotralstat as First Oral Hereditary Angioedema Prophylaxis. Hcplive. Published December 4, 2020. Accessed December 9, 2025. 

https://www.hcplive.com/view/fda-approves-berotralstat-oral-hereditary-angioedema-prophylaxis

Soteres D, Tachdjian R, Manning M. Rare Disease, Rapid Progress: FDA's HAE Approvals in 2025 Signal Changing Course. HCPLive. Published December 3, 2025. Accessed 9, 2025. 

https://www.hcplive.com/view/rare-disease-rapid-progress-fda-hae-approvals-in-2025-signal-changing-course

US Hereditary Angioedema Association. Haea.org. Published 2020. 

FDA Expands Lanadelumab for Preventing HAE Attacks in Younger Children. HCPLive. Published February 4, 2023. Accessed 9, 2025. 

https://www.hcplive.com/view/fda-expands-lanadelumab-preventing-hae-attacks-younger-children

U.S. FDA Approves CSL Behring’s Berinert® as the First and Only Pediatric Treatment for Hereditary Angioedema (HAE). Global Newsroom | CSL. Published July 18, 2016. Accessed December 9, 2025. 

https://newsroom.csl.com/2016-07-18-U-S-FDA-Approves-CSL-Behrings-Berinert-R-as-the-First-and-Only-Pediatric-Treatment-for-Hereditary-Angioedema-HAE 

Derman C. FDA Accepts BioCryst’s NDA for Berotralstat Oral Granules in Children With HAE. HCPLive. Published May 14, 2025. Accessed December 9, 2025. 

https://www.hcplive.com/view/fda-accepts-biocryst-s-nda-for-berotralstat-oral-granules-in-children-with-hae

Derman C. Oral Berotralstat Shows Strong Safety, Efficacy in Young Children with HAE. HCPLive. Published August 5, 2025. Accessed December 9, 2025. 

https://www.hcplive.com/view/oral-berotralstat-shows-strong-safety-efficacy-in-young-children-with-hae

The FDA has approved berotralstat (ORLADEYO) oral granules for children aged 2-11 with hereditary angioedema (HAE), expanding its 2020 approval for those aged 12 and older. This new formulation offers a less burdensome prophylactic option compared to infusions or injections, addressing a critical gap in pediatric HAE care. The approval is based on positive interim data from the APeX-P trial, which demonstrated significant reductions in attack rates. Berotralstat is well-tolerated in children, with common adverse events including abdominal pain and vomiting.

Berotralstat (ORLADEYO) oral granules become the first FDA-approved therapy specifically designed to prevent HAE attacks in children aged 2 – 11 years.

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Allergy condition center page is a comprehensive resource for clinical news and insights on allergic and inflammatory diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for food and seasonal allergies, as well as allergic asthma and related conditions.

FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE  

The US Food and Drug Administration has approved Acadia Pharmaceuticals’ trofinetide (Daybue Stix) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients ≥ 2 years of age.

As described in a December 12, 2025, release from the Company, the new formulation is expected to deliver the same efficacy and safety profile of trofinetide oral solution, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their trofinetide treatment.

“The new formulation gives us an additional option for treatment with DAYBUE, allowing us to better customize care for our patients,” said Jennifer Martelle Tu, MD, PhD, Director of Katie's Clinic for Rett Syndrome and Associate Professor of Neurology, UCSF Benioff Children's Hospitals, Oakland. “DAYBUE STIX is a powder for oral solution that caregivers can mix with a variety of water-based liquids providing flexibility to modify the taste and volume of their loved-one's dose. We know that this kind of adaptability is something many Rett families have been seeking.”

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. It became 

the only FDA-approved treatment for Rett syndrome in 2023. 

The mechanism by which it exerts therapeutic effects in patients with Rett syndrome is unknown, but in animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.

“DAYBUE, the first and only approved treatment for Rett syndrome, has provided thousands of patients an important therapeutic option,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “With the approval of DAYBUE STIX, we are building on our commitment to turn scientific promise into meaningful innovation by introducing a new formulation informed by invaluable feedback from patients, caregivers and healthcare providers.”

The efficacy and safety of this new formulation was assessed in the LAVENDER™ study in patients with Rett syndrome. The approval was informed by the results of a bioequivalence study, which demonstrated that both original trofinetide oral solution and the new trofinetide for oral solution powder formulation provide comparable exposure. This confirmed bioequivalence means patients can expect the same efficacy and safety established by the oral solution formulation when using trofinetide for oral solution powder formulation.

As described in the release from Acadia, DAYBUE STIX will be available on a limited basis starting in the first quarter of 2026 and more broadly early in the second quarter of 2026, and the current oral solution formulation will remain available.

Acadia Pharmaceuticals. Acadia Pharmaceuticals Announces FDA Approval of DAYBUE® STIX (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of Rett Syndrome. December 12, 2025. Accessed December 12, 2025. 

https://acadia.com/en-us/media/news-releases/acadia-pharmaceuticals-announces-fda-approval-daybuer-stix

FDA. FDA approves first treatment for Rett Syndrome. March 13, 2023. Accessed December 12, 2025. 

https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-rett-syndrome

The FDA has approved Acadia Pharmaceuticals' trofinetide (Daybue Stix) for oral solution, a new powder formulation for treating Rett syndrome in patients aged 2 and older. This formulation offers flexibility in dose volume and taste, enhancing treatment customization. Trofinetide, a synthetic analog of insulin-like growth factor 1, is the only FDA-approved treatment for Rett syndrome. The new formulation's efficacy and safety were confirmed through bioequivalence studies, ensuring comparable exposure to the original oral solution. Daybue Stix will be available in early 2026, alongside the existing oral solution.

The approval of a dye- and preservative-free powder formulation of trofinetide offers a new option for adult and pediatric patients with Rett syndrome.

FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for HCPLive. He is also the executive producer of two podcasts: Skin of Color Savvy and The Medical Sisterhood. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

Apremilast is effective as an oral systemic therapy for 

, new data suggest, with consistent clinical efficacy, symptoms improvements, and quality-of-life benefits.

These findings from the phase 3 DISCREET study (NCT03777436) followed 32 weeks of analysis of this drug’s impact on genital psoriasis. Apremilast is an oral phosphodiesterase-4 (PDE4)-inhibitor approved for patients living with psoriasis. The medication previously demonstrated safety and efficacy in treating genital psoriasis following the 16-week results of DISCREET.

This new 32-week analysis, authored by investigators such as Joseph F. Merola, MD, MMSc, of UT Southwestern Medical Center, explored the twice-daily use of apremilast in the same patient group following the primary findings released at the 16-week mark.

“Product labelling in the United States has been revised to include these data supporting the efficacy of apremilast in genital psoriasis,” Merola et al wrote.

 “We report 32-week safety and efficacy results as well as post-hoc analyses of efficacy results.”

In their analysis, Merola and coauthors described the design of the DISCREET trial as randomized, double-blind, and placebo-controlled. The investigators' research was conducted across multiple centers located in Canada, Belgium, France, Italy, the US, and Germany. While details of DISCREET's design and methodology were previously highlighted, the team noted participants were assigned in a 1:1 ratio to be treated either with apremilast or placebo through a centralized Interactive Response Technology system via permuted block randomization.

Adults with moderate to severe genital psoriasis, as indicated by a baseline modified static Physician’s Global Assessment of Genitalia (genital PGA or sPGA-G) score of 3 or higher, were deemed as eligible to take part in the trial. All of those involved as participants had either insufficient response to or intolerance of topical drugs. Additionally, these individuals would also have had moderate to severe chronic plaque psoriasis for at least half a year, defined as an overall static Physician’s Global Assessment (sPGA) score of 3 at least, and non-genital plaque psoriasis involving at least 1% of patients' body surface area (BSA).

In terms of treatment Merola et al administered apremilast 30 mg, taken orally, on a twice-daily basis or provided participants with a matched placebo for 16 weeks during the placebo-controlled phase of DISCREET. This was then followed by a 16-week extension phase, during which the investigators provided all subjects with apremilast. Individuals who had been on placebo switched to active therapy from Weeks 16 - 32 (placebo/apremilast cohort). At the same time, individuals originally assigned to the apremilast cohort continued treatment for the full 32 weeks (apremilast/apremilast group).

At screening, at baseline, and at the 2, 4, 8, 12, 16, 20, 24, and 32-week marks, study visits took place. Merola and colleagues' follow-up interaction took place 4 weeks following the last apremilast dose. There were 289 patients who were randomized, with 229 entering the study's apremilast extension period. Among these subjects, 110 were transitioned by Merola and coauthors from placebo to apremilast at the 16-week mark. 119 remained on continuous apremilast throughout.

By Week 32, the investigative team found attainment of a modified genital PGA response, defined as a score of 0 or 1 with a reduction of at least 2 points from baseline, was seen among 51.8% of those included in the placebo/apremilast group (95% CI: 42.6–60.9).

 It was also seen among 40.3% of the apremilast/apremilast group (95% CI: 32.0–49.3).

By the conclusion of the study's 32-week period, the team identified improvements in overall skin disease, genital psoriasis signs and symptoms, and quality-of-life measures. They also found these results to be comparable between subjects who initiated apremilast at randomization and those who began treatment at the 16-week mark.

Additionally, the Merola et al's safety findings revealed the most commonly reported treatment-emergent adverse events during apremilast exposure to be instances of diarrhea (25.4%), nausea (19.4%), and headaches (17.9%).

“Based on the consistent clinical efficacy, improvements in symptoms and QoL benefit seen in DISCREET, apremilast appears to be an effective oral therapy in patients with moderate to severe genital psoriasis,” the investigators wrote.

Merola JF, Guenther L, Yamauchi P, et al. Results from the 32-week, phase 3 DISCREET study of apremilast in patients with moderate to severe genital psoriasis. J Eur Acad Dermatol Venereol. 2025; 00: 1–11. 

Merola JF, Parish LC, Papp KA, et al. Efficacy and safety of apremilast in patients with moderate-to-severe genital psoriasis: Results from DISCREET, a phase 3 randomized, double-blind, placebo-controlled trial. J Am Acad Dermatol. 2024 Mar;90(3):485-493. 

Apremilast, an oral PDE4 inhibitor, has shown efficacy in treating moderate to severe genital psoriasis, as evidenced by the 32-week phase 3 DISCREET study. The study demonstrated significant clinical efficacy, symptom improvement, and quality-of-life benefits. Conducted across multiple centers, the trial involved a randomized, double-blind, placebo-controlled design. Participants initially on placebo switched to apremilast after 16 weeks, with notable improvements observed by week 32. Common adverse events included diarrhea, nausea, and headaches. These findings support apremilast as an effective oral therapy for genital psoriasis.

The phase 3 DISCREET study explored the safety and efficacy of apremilast in treating patients with moderate to severe genital psoriasis over 32 weeks.

Plaque Psoriasis

The HCPLive plaque psoriasis page is a resource for medical news and expert insights on psoriasis. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for psoriatic disease, and more.

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Apremilast Effective Treatment for Moderate to Severe Genital Psoriasis at 32 Weeks

Chronic lower respiratory diseases like COPD were the fourth leading clinical cause of death among Americans as of 2023, and lung cancer remains the deadliest cancer today.

 have continually ranked among the greater drivers of disease burden and mortality.

The signs and symptoms of each are clearly understood, and non-invasive screening tools including low dose computed tomography (CT) scans and spirometry tests are proven safe, effective options for patients who meet disease risk criteria.

All this accounts to very little in the effort to curb deaths from COPD or lung cancer. The American Lung Association’s 2025 State of Lung Cancer report showed that, despite invasive care being most effective in cases with early-stage diagnosis, only 28.1% of cases are diagnosed at a stage when five-year survival rates are approximately 65%. Instead, patients with lung cancer are 50% more likely to be diagnosed at a later stage when survival rate is approximately 10%.

The 2025 edition of the Global Initiative for Chronic Obstructive Lung Disease (GOLD) guide for health care professionals paints a similar situation in missed opportunities for timely COPD detection and intervention. The guide this year highlighted environmental factors beyond tobacco smoking that may heavily influence COPD risk, as well as precursor conditions like pre-COPD and Preserved Ratio Impaired Spirometry (PRISm).

Patients who are presenting without airflow obstruction, defined as FEV

 / FVC ≥0.7 post-bronchodilator but any combination of these factors:

Structural lung lesions such as emphysema

Physiological abnormalities such as low FEV

, gas trapping, hyperinflation, reduced lung diffusing capacity, or rapid FEV

Patients who are presenting without airflow obstruction but decreased lung function per FEV

Despite these broadening opportunities to flag COPD risk, as well as major pharmaceutical milestones that have 

introduced targeted therapies and biomarker-specific agents to the field

 the GOLD committee acknowledges that there remains a major challenge in getting new cases diagnosed.

“COPD is a common, preventable, and treatable disease, but extensive under-diagnosis and misdiagnosis leads to patients receiving no treatments or incorrect treatment,” they wrote. “Appropriate and earlier diagnosis of COPD can have a very significant public-health impact.”

Lung cancer screening criteria has similarly been broadened in recent years to encourage a greater rate of diagnostic opportunities. The US Preventive Services Task Force (USPSTF) redefined its standard for “high-risk” individuals in 2021 — moving the qualification from individuals ≥55 years old with ≥30 pack years of cigarette smoking, to those aged 50 – 80 years who smoked ≥1 pack of cigarettes daily for 20 years or who quit smoking within the last 15 years.

Again, the State of Lung Cancer report showed approximately only 1 in 6 high-risk individuals were screened last year.

Experts have significantly widened the eligibility for COPD and lung cancer screening and evidence advocating for the efficacy of timely treatment has only improved in that time. So, what is it going to take to get more patients through the door in 2026?

The most apparent issue in lung cancer and COPD screening is the disparate access to such opportunities in the current healthcare system. Nearly 6 million Americans live in a “pulmonology desert” as of 2025, defined as areas that are ≥1 hour drive from the nearest pulmonologist, according to healthcare services provider GoodRx.

 These individuals predominately reside in Midwest states like Wyoming, where only 9.7% of high-risk individuals were screened for lung cancer in the last year. These residents have an approximate 50% decreased likelihood of 5-year survival compared with states like Rhode Island and Connecticut, which do not experience such challenges with clinician access.

Given that spirometry is the primary diagnostic tool in COPD — with results supported by patient questionnaires regarding symptoms and quality of life measures — the lack of in-person care opportunities persists the limit of timely COPD diagnoses in these deserts as well. 

Lung cancer screening has also been hampered by a slow buy-in to new processes. Despite being recommended as a standard strategy, low dose CT scans are a relatively newer procedure, and systemic buy-in is very gradual. As Douglas Wood, MD, the Henry N. Harkins Professor and Chair of the Department of Surgery at University of Washington, told 

, the procedure has been recommended and covered by payers for only the last decade. That’s hardly time to expect widespread adoption.

“If we’re just pragmatic, it does take time for the culture, policies, and procedures to get people to shift,” Wood said.

Regarding both low dose CT scans for lung cancer and lung function testing for COPD, multiple experts have stressed to 

 the need for more robust data supporting the approaches and their association with significant treatment outcomes — not for their own sake, but to assuage any lingering doubts among their colleagues.

This is a highly common issue not just in pulmonary medicine, but all of medicine in general, Graham Barr, MD, DrPH, Chief of the Division of General Medicine at NewYork-Presbyterian and Columbia University Irving Medical Center, told 

. Screening tools require “well-performed, randomized clinical trials” to achieve buy-in from clinicians, he explained.

“And we have that — two very good ones for lung cancer screening showing not just mortality reduction from lung cancer deaths, but all-cause mortality reduction too,” Barr said. “And that’s unique [among cancer screening tools].”

Whereas low dose CT scans have quickly accumulated top-down recommendation, supporting clinical evidence and cost coverage, pulmonary function testing remains a circumstantial tool in COPD screening. The USPSTF does not recommend its use to diagnose asymptomatic COPD, and debates persist on its cost-effectiveness relative to the screening setting.

Many experts want to see COPD screening become more widely adopted at the primary care level. But there’s concerns about the knowledge and perception of COPD among many clinicians.

The most common argument supporting the increase of respiratory disease screening is perhaps the simplest one to make: these rates would be unacceptable for any other disease. Barr noted the significantly better rates of breast cancer and cervical cancer screening for high-risk individuals relative to the more deadly lung cancer. Meredith McCormack, MD, director of the Division of Pulmonary & Critical Care Medicine at Johns Hopkins University School of Medicine, made the point to 

 that electrocardiograms are standard practice at cardiology visits — but spirometry testing is infrequent in her field.

The reality is that there is a significant need for these tools, and there are data and supports advocating for their use. Clinicians just are not using them.

From McCormack’s perspective, a crucial barrier in pulmonary function testing is getting clinicians more comfortable with conducting and analyzing results.

“The test can feel complicated, and part of what makes it feel complicated is some of the nuances of how we interpret the findings,” McCormack said. “We’re trying to make that simpler and show how to interpret a person’s numbers.”

Francesca Polverino, MD, PhD, professor of pulmonary medicine at Baylor College of Medicine, told 

 that advanced COPD education is much needed among their primary care peers. There’s a persistent mindset that it is a more homogeneous condition than it truly is — and ironically, that single-track mindset of what COPD is limits the field’s capability to learn more about it.

“The [primary care physicians] need to understand that COPD can occur in absence of cigarette smoke, it can occur in younger people, and there is a lot more beyond oral corticosteroids [to treat] because usually when we get a patient after an exacerbation who had been to the PCP, the patient has already been through a couple of rounds of oral corticosteroids already, which is a problem for the pulmonologist,” Polverino explained.

What limits clinicians from screening for lung cancer and COPD most, though, is the stigma attached to both diseases. Cigarette smoking is the most common trait of respiratory disease, and there remains a persistent perception among clinicians that COPD and lung cancer are self-inflicted — and as such, there’s less drive to identify and intervene on new cases.

An understated impact of the stigma surrounding COPD and lung cancer is that it causes clinicians to miss cases among younger, non-smoking patients. Polverino advocated for her primary care colleagues to more actively seek out the early signs of COPD in younger patients, including dyspnea, an inability to walk, chronic cough, chest tightness, and excess mucus.

“[There is] an aspect of lung cancer that it is the fault of the individual, even though there's many individuals who get lung cancer that never had a smoking history,” Wood said. “The stigma associated with lung cancer is another problem with getting it to be accepted at the level that we have for breast or colon cancer screening.”

Wood added that the patient demographic most affected by lung cancer and COPD is one that is not “well advocated for.” This includes older individuals, minorities, and those from lower socioeconomic status. This is why screening recommendation groups have moved toward a race neutral approach — described by McCormack as a “one size fits all” standard for pulmonary function rather than defining standards by race-specific averages.

Another driver of COPD and lung cancer under-diagnosis is, ironically, the high mortality rate associated with each. Wood described a “nihilism” among his colleagues that, even if lung cancer was detected during an early stage, the likelihood of survival is not promising enough to warrant the procedure and follow-up care. Coupled with the blame placed on patients for their role in smoking, there’s little resolve to intervene.

Polverino added that there’s a lack of urgency surrounding these conditions. Despite its high mortality rate, COPD “doesn’t kill you fast enough,” she explained — whereas a major adverse cardiovascular event could escalate care and improve a patient’s adherence to lifestyle modifications, COPD is chronic and debilitating over years of worsening condition.

Ironically, this singular and defeatist perspective of lung cancer and COPD is what most perpetuates their status as slow marches to death. As many experts have stressed to 

 over the years, there is burgeoning opportunity to better understand and manage disease like COPD if it were only prioritized.

, the identification of COPD biomarkers helped introduce the first targeted biologics to clinically relevant patients: dupilumab and mepolizumab are indicated and proven to benefit specific patients based on blood eosinophil counts, complementary to maintenance therapy. These agents have helped reduced the rate and burden of exacerbations in patients with COPD — and have encouraged clinicians like Polverino to continue seeking more widely implicative biomarkers of the disease.

“There are big changes coming in the next 10 years,” she said. “COPD is going into the spotlight again, after it was considered an untreatable disease.”

The 2026 GOLD Report, presented last month, acknowledged the role of targeted biologic intervention in COPD going forward. Most notably, the new report discussed the opportunity to define “disease stability” in patients with COPD relative to the adoption of more endotype-based diagnostics and targeted treatment initiation following the first exacerbation.

Claus Vogelmeier, MD, chair of the GOLD science committee, told 

 his expectation is treatment escalation with a growing armamentarium of therapies could provide patients this new standard of disease stability. What’s needed, though, is timely diagnosis.

“If we are able to identify patients earlier in a state where not most of the lung is destroyed, and then select the right patient based on their endotype and give them the drugs that are targeted to this endotype...then we have a very high likelihood that we are able to change the natural cause the disease, and that that was not possible 20-30 years ago,” Vogelmeier said. “We didn't have the management strategies, we didn't have the assessment strategies, we weren't able to diagnose earlier. I think this is the time to try."

McCormack perceives an opportunity to altogether prevent COPD development in at-risk individuals with a greater adoption of pulmonary function testing at the primary care level. She described an evolved system where baseline testing is conducted at a younger age and monitored regularly throughout an individual’s life to interpret their likelihood of respiratory disease — and to help inform the greater global standard for lung health and timely interventions.

“That’s something to aspire to: where people could be trending their values relating to their historic values, rather than trying to understand their values relative to other healthy people,” McCormack said.

Similarly to GOLD, McCormack also sees an opportunity to incorporate the numerous determinants of respiratory health to provide a fuller picture of an individual’s risk: merging data from spirometry, CT scans, endotype tests, as well as notable lifestyle factors including smoking, vaping device use, and harmful air exposure to profile a person’s lung health.

“[Spirometry] is a fairly simple maneuver, and we should continue to use that value but also integrate it with other information we have about that individual so we can make specific, personalized medical decisions,” McCormack said.

It is also understated how widely beneficial CT scans can be to fulfilling that specific patient profile, Barr said. Screening for lung cancer often helps diagnose emphysema in patients who have never received a spirometry.

“Lung CT gives us a massive amount of information that going forward is going to be very helpful for guiding therapy and patient selection,” Barr said. “We should be using this as part of our diagnostic algorithm…and it’ll help a lot with the management of our COPD patients.”

The situation at hand is not without cause for optimism. The State of Lung Cancer report did note that the national survival rate has increased from 26% to 29.7% over the last 5 years, and again, the therapy pipeline for COPD looks very promising over the next decade.

But as it stands in 2025, lung cancer and COPD remain among the deadliest diseases in the US. That fact is clearly known. There are tools to detect these diseases, evidence supporting their effect, recommendations for their use, and supports to use them. They just need to be used.

“It’s sort of staggering that we’re still in this position,” Barr said. “We have to straighten this out.”

Ahmad FB, Cisewski JA, Anderson RN. Leading Causes of Death in the US, 2019-2023. JAMA. 2024;332(12):957-958. doi:10.1001/jama.2024.15563

American Lung Association. State of Lung Cancer. 2024. Accessed December 10, 2025. https://www.lung.org/research/state-of-lung-cancer

Venkatesan P. GOLD COPD report: 2025 update. Lancet Respir Med. 2025;13(1):e7-e8. doi:10.1016/S2213-2600(24)00413-2

Johnson V, Campbell P. Rewriting Airway Disease With Trait-Based Care: 2025’s Convergence of Asthma and COPD Management. 

. Published online December 1, 2025. https://www.hcplive.com/view/rewriting-airway-disease-with-trait-based-care-convergence-asthma-copd-management

US Preventive Services Task Force, Krist AH, Davidson KW, et al. Screening for Lung Cancer: US Preventive Services Task Force Recommendation Statement. JAMA. 2021;325(10):962-970. doi:10.1001/jama.2021.1117

Guttentag S. Mapping Pulmonology Deserts in 2025: Where Are Americans Having Trouble Getting Lung Care? 

. Published online September 25, 2025. https://www.goodrx.com/healthcare-access/research/pulmonology-deserts-update

Kunzmann K. What Ails Lung Cancer Screening, with Douglas Wood, MD. 

. Published online October 20, 2025. https://www.hcplive.com/view/what-ails-lung-cancer-screening-douglas-wood-md

Kunzmann K. Bettering CT Scan Rates in Pulmonology, with Graham Barr, MD, DrPH. 

. Published online December 2, 2025. https://www.hcplive.com/view/bettering-ct-scan-rates-pulmonology-graham-barr-md-drph

US Preventive Services Task Force. Screening for Chronic Obstructive Pulmonary Disease: US Preventive Services Task Force Reaffirmation Recommendation Statement. JAMA. 2022;327(18):1806–1811. doi:10.1001/jama.2022.5692

Kunzmann K. The Case for More Spirometry, Pulmonary Function Testing, with Meredith McCormack, MD. 

. Published online October 20, 2025. https://www.hcplive.com/view/the-case-for-more-spirometry-pulmonary-function-testing-meredith-mccormack-md

Kunzmann K. The Practical Hurdles in COPD Detection, with Francesca Polverino, MD, PhD. 

. Published online November 14, 2025. https://www.hcplive.com/view/practical-hurdles-copd-detection-francesca-polverino-md-phd

Venkatesan P. GOLD COPD report: 2026 update. Lancet Respir Med. Published online November 26, 2025. doi:10.1016/S2213-2600(25)00432-1

Kunzmann K. Breaking Down the 2026 GOLD Report, with Claus Vogelmeier, MD. 

. Published online November 14, 2025. https://www.hcplive.com/view/breaking-down-2026-gold-report-claus-vogelmeier-md

Chronic lower respiratory diseases, including COPD and lung cancer, remain leading causes of death in the US, despite advancements in screening and treatment. Disparities in healthcare access, clinician buy-in, and persistent stigmas contribute to underdiagnosis and late-stage detection. Efforts to broaden screening criteria and introduce targeted therapies have been made, yet systemic and cultural barriers persist. Experts emphasize the need for earlier diagnosis, improved clinician education, and a shift in perception to enhance patient outcomes. The potential for personalized medicine and targeted interventions offers hope for future advancements.

2025 data show the US is still failing to adequately identify and initiate care for deadly lung disease. Experts discuss what it will take to turn the tide on this public health issue.

This Year In Medicine

Pulmonology

Infectious Disease condition center page is a comprehensive resource for clinical news and insights on respiratory health. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for asthma, COPD, interstitial lung disease, COVID-19, and more.

COPD

COPD condition center page, resources on the topics of medical news and expert insight into chronic obstructive pulmonary disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on COPD research, treatment, and drug development.

Lung Cancer

Getting Through the Door: How COPD, Lung Cancer Screening Remains Minimal

Alex Hillenbrand is an assistant editor for 

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Getting Through the Door: How COPD, Lung Cancer Screening Remains Minimal