This article, "Satralizumab Approved for Treatment of Neuromyelitis Optica Spectrum Disorder," was originally published in NeurologyLive.

The US Food and Drug Administration (FDA) has approved satralizumab for the treatment of adult patients with anti-aquaporin-4 (AQP4-IgG) antibody positive 

neuromyelitis optica spectrum disorder (NMOSD)

The approval, awarded to Genentech, is only the second targeted treatment for this patient population and the first eligible for at-home administration.

Marketed under the name Enspryng, the humanized monoclonal antibody targets and inhibits interleukin-6 receptor activity, which is thought to play a key role in the inflammation observed in 

Novel recycling technology allows for a longer duration of antibody circulation, translating into a subcutaneous dosing regimen of every 4 weeks after an initial loading dose.

The approval pins satralizumab against the only other targeted treatment in this space, Alexion’s eculizumab (Soliris), which was approved with the same indication on June 27, 2019.

“For people with NMOSD, relapses can cause devastating, irreversible and disabling neurological effects,” Jeffrey Bennett, MD, PhD, professor of neurology and ophthalmology at the University of Colorado, and an investigator for the satralizumab pivotal clinical trials, said in a statement. “Having an approved therapy that can be administered subcutaneously in the home and has demonstrated an impact on the frequency of relapses is an important advancement for patients.”

Satralizumab’s approval was based on robust data from the phase 3 SAkuraStar and SAkuraSky clinical trials, which combined included more than 170 patients who were randomly assigned to receive satralizumab 120 mg or placebo.

In SAkuraSky, patients added treatment to baseline immunosuppressive therapy.

All patients received a loading dose of the study drug at baseline, week 2, and week 4, followed by 4-week treatment intervals.

The primary end point in both studies was time to protocol-defined relapse. Notably, both studies included a population of patients who were AQP4-IgG antibody positive and negative to accurately reflect clinical practice.

In SAkuraStar, 30% of patients treated with 

 experienced relapse compared with 50% of those who received placebo (HR 0.45; 95% CI, 0.23–0.89; 

Among those who were AQP4-IgG antibody positive, a 74% reduction in relapse risk was observed.

In the overall satralizumab-treated population, 76.1% and 72.1% were relapse-free at 48 and 96 weeks, respectively, compared with 61.9% and 51.2% with placebo.

Data from the AQP4-IgG seropositive subgroup showed that 82.9% and 76.5% were relapse-free at 48 and 96 weeks compared with 55.4% and 41.1% with placebo, respectively.

In total, 92% (n = 58) of those in the satralizumab group experienced an adverse event compared with 75% (n = 24) of the placebo group. Serious AEs were similar between groups; only 1 event led to study drug discontinuation in the treatment group.

In SAkuraSky, the overall population saw a 62% reduction in the risk of relapse (HR 0.38, 95% CI, 0.16-0.88; 

 =.0184), while the group of AQP4-IgG seropositive patients experienced a 79% reduction in relapse risk (HR 0.21, 95% CI, 0.06-0.75; 

Results showed 88.9% and 77.6% of patients in the total population were relapse-free at 48 and 96 weeks, respectively, compared with 66% and 58.7% of patients in the placebo group.

Among AQP4-IgG seropositive patients, 91.5% treated with satralizumab were relapse-free at 48 and 96 weeks compared with 59.9% and 53.3% of patients in the placebo group.

The most common AEs observed were upper respiratory tract infection, nasopharyngitis, and headache.

Genentech reports that the drug will be available in the US within 2 weeks. It is already approved and available in Japan, Canada, and Switzerland, with applications under review in the European Union and China.

Satralizumab represents only the second targeted treatment for NMOSD and the first cleared for at-home administration.

FDA Approves New Neuromyelitis Optica Spectrum Disorder Treatment

Alicia Bigica

Ankeet Bhatt, MD, is a research scientist at Kaiser Permanente Northern California and an associate physician at Kaiser San Francisco Medical Center.

Cardiology and the Move to Combination Therapy, with Ankeet Bhatt, MD, MBA

 has advanced further and faster than many other specialties, with innovations and discoveries cropping up almost constantly. From new treatments themselves to the efficacy and safety of their delivery, the patient side of cardiology is expanding rapidly, and research on the disease is not showing signs of slowing down.

Cardiology's growth was showcased again this year at the 

American College of Cardiology (ACC) 2025 Annual Scientific Sessions

. With more than 4750 abstracts and 1300 expert faculty, the conference covered 53 late-breaking clinical trials in over 300 sessions from March 29-31.

A leading expert in the field, Ankeet Bhatt, MD, MBA, a research scientist at the Division of Research and an associate physician and practicing cardiologist and intensivist at the Kaiser Permanente San Francisco Medical Center, sat down with 

's editorial team to discuss what he expects to see from cardiology over the rest of 2025.

What are you looking forward to in cardiology for the rest of 2025?

 It's a great time to be in cardiovascular, renal, and metabolic health, because there's so much happening. I think we're going to learn a lot more about the class of medicines that are related to the glucagon-like peptide 1 GLP-1 receptor agonist (GLP-1 RA). There's multiple therapeutics coming on [at ACC] that are GLP-1-RAs plus. So I think we're going to learn a lot more about their modifying effect. 

I'm excited for the trend toward combination therapy, because I think we're reaching a point in which our patients are medically complex, and there's lots of available, highly efficacious therapies, but we need to be thinking more smartly about how to deliver those therapies in a way that's patient centric, and I see that as, really, a care delivery problem. But I think there's a lot of innovation that can happen there. 

And then I'm excited about heart failure still. I think we've made lots of progress in both heart failure with reduced and preserved ejection fraction (HFrEF and HFpEF). I think the latter half of the year is going to tell us more about certain populations, for example, those who have marginal blood pressure or renal function or other things. Are there available therapies outside of the advanced heart failure therapies for these patients? I'm looking forward to an exciting back half of 2025.

Relevant disclosures for Bhatt include Sanofi and Novo Nordisk.

Bhatt discusses his expectations for the future of cardiology in 2025, including GLP-1-RAs and the push towards combination therapy.

GLP-1 RAs and the Move to Combination Therapy in 2025, with Ankeet Bhatt, MD, MBA

Ankeet Bhatt, MD, MBA

Brittany Oliver, MD, FAAD is a board-certified dermatologist practicing in Kansas City, Missouri, with a special interest in pigmentary disorders and hair loss, and a member of the Skin of Color Society.

Skin of Color Savvy: The Art and Science of Treating Patients of Color

Skin of Color Savvy: News Update—Addressing Disparities, Black History Month, & Mentorship Opportunities

Skin of Color Savvy: The Art and Science of Treating Patients of Color 

series, Brittany Oliver, MD, FAAD, speaks about the pivotal role of mentorship in dermatology and the initiatives led by the 

 to foster professional development. Oliver emphasizes the profound impact of mentorship on career trajectories, highlighting how meaningful mentor-mentee relationships extend beyond networking to shape clinical expertise, confidence, and leadership in the field.

She discusses SOCS’s commitment to mentorship as one of its core pillars, detailing key programs such as the 

, and the newly launched Diversity in Clinical Research Mentorship Program. These initiatives aim to support early-career dermatologists, underrepresented minority (URM) investigators, and students pursuing careers in dermatology.

In honor of Black History Month, Oliver reflects on the influential mentors who shaped her journey, including Adam Friedman, MD, and SOCS founder Susan Taylor, MD, whose dedication to mentorship and advocacy has left a lasting impact on the specialty. She also shares her perspective on the importance of increasing diversity in medicine, encouraging dermatologists to engage in outreach, advocacy, and structural changes to address healthcare disparities.

Finally, in recognition of American Heart Month, the discussion turns to the intersection of dermatology and cardiovascular disease, particularly within communities of color. Oliver underscores the need for systemic solutions, early pipeline programs, and physician-led advocacy to improve representation and access to care. She calls on her colleagues to take an active role in addressing disparities, emphasizing the power of physicians to effect meaningful change beyond the clinic.

To learn more about SOCS’s mentorship programs and initiatives, visit 

The February 18, 2025 SOCS News Update highlights SOCS mentorship opportunities as well as the importance of mentors and role models.

Brittany Oliver, MD, FAAD

panel discussion on a head-to-head sequence study of rizankizumab vs ustekinumab in the treatment of CD, as well as approaches to choosing first-line treatment.

Sequence Study of Risankizumab vs Ustekinumab in CD

This is a video synopsis/summary of a panel discussion involving Marla Dubinsky, MD; David Rubin, MD; Miguel Regueiro, MD; Remo Panaccione, MD, FRCPC; and Sara Horst, MD, MPH.

The transcript discusses the findings of the sequence study, focusing on the comparison between two drugs, P41 (referred to as risankizumab) and P19 (referred to as IL (interleukin)-23 therapy). The study aimed to evaluate the efficacy of risankizumab in patients with Crohn's disease who had been exposed to TNF.

The results indicate that risankizumab-exposed patients were more likely to achieve clinical response, clinical remission, and endoscopic improvement compared to those on P40 (risankizumab with IL-12 and IL-23). The transcript emphasizes that the head-to-head comparison provides valuable insights, guiding clinicians in choosing the most effective therapy.

It highlights that risankizumab, along with its biosimilar, is an approved and effective option for Crohn's disease. The discussion touches upon the significance of the sequence study's second primary endpoint, endoscopic remission, and how it could be a pivotal moment in the field.

The conversation also touches on the evolution of clinical trials in Crohn's disease, emphasizing the importance of endoscopic outcomes and the progression from subjective measures to objective proof of efficacy. The participants express optimism about the potential impact of risankizumab, especially when used as a first-line therapy.

Furthermore, the transcript discusses the importance of early intervention in Crohn's disease, suggesting that initiating treatment early is more likely to result in a positive response and prevent further damage. The participants encourage setting higher expectations for patients and discuss the advantages of using risankizumab in biologic treatment-naive patients.

In conclusion, the discussion underscores the positive findings from the sequence study, positioning risankizumab as a promising option for Crohn's disease, particularly in terms of achieving endoscopic remission and improving patient outcomes. The participants stress the need for effective communication with patients about treatment options and the potential long-term benefits of early intervention.

Video synopsis is AI-generated and reviewed by HCPLive editorial staff.

David Rubin, MD, leads the panel discussion on a head-to-head sequence study of rizankizumab vs ustekinumab in the treatment of CD, as well as approaches to choosing first-line treatment.

David Rubin, MD

Dhiren Patel, PharmD, CDCES, BC-ADM, BCACP:

 I did want to mention about a recent approval of bempedoic acid. I know it exists as monotherapy and in combination. And so, maybe Dr Busch, what’s the role of that in your toolbox? When do you kind of use it? If you could talk to us a little bit about bempedoic acid.

 So bempedoic acid blocks the pathway to generate cholesterol in the liver higher up than where a statin does. Statins are HMG-CoA blockers, and this is an ATP-citrate lyase blocker. So basically [it] stops cholesterol synthesis within the liver. The liver is hungry for cholesterol and upregulates the LDL [low-density lipoprotein] receptors. Which is what Brown [Michael S. Brown] and Goldstein [Joseph L. Goldstein] won the Nobel prize for in the 80s. It works higher in the pathway, but interestingly, it is not activated in the muscle, even though it’s activated in the liver so that it doesn’t affect the muscle cholesterol. There’s very little myopathy with the drug. The potential side effects are few. It can raise uric acid a little bit, so you have to be cautious if you give it to someone who’s had gout in the past. And there’s very rare cases of muscle rupture, although that’s not clear that that will happen. There’s no outcome study yet, the outcome study is in progress, fully recruited. It’s called the CLEAR-outcomes [Cholesterol Lowering via Bempedoic acid, an ACL-Inhibiting Regimen] study. And interestingly about that outcome study, the patients are either on no statin or very low dose statin. Very different than all of the other trials. Where the IMPROVE-IT trial, they were all on a statin. In the PCSK9 studies, they were on statins. And obviously the statin outcome studies were on statin. This will be 1 of the first studies in someone on no or low dose statin where a drug that is not a statin should show benefit. The LDL drop is about 18% monotherapy. And when it’s with ezetimibe [Zetia] as a combination drug, it’s about 38%, 40%. And those are patients already on a statin. If they’re not on a statin, can’t tolerate a statin, the drops might even be more. So personally, if someone’s on ezetimibe and their LDLs is not at goal and they can’t afford or close to LDL goal, I’ll upgrade it to the next—bempedoic acid with ezetimibe together. And the cost of it, to quote what Jen said, it’s a $10 copay for commercial insurance. It’s almost the same price as Dr Bhatt said for generic ezetimibe, and you get an extra 20% LDL. Again, there’s no outcome yet, but there [is] no reason you wouldn’t expect a positive outcome because it upregulates LDL receptors like all the other drugs that have outcomes.

 That's really helpful. And Dr Bhatt, I know Dr Busch just mentioned the outcomes are pending, but where does this fit into your toolbox? And maybe if you could kind of give it to us from a cardiology perspective, I think that would be helpful for the audience.

Deepak L. Bhatt, MD, MPH, FACC, FAHA, FSCAI, FESC:

 Sure. I think it’s a good question. Data to date of bempedoic acid looked good in terms of safety and in terms of efficacy, especially for that statin intolerant patient and when combined with ezetimibe, it seems to be respectable reductions in LDL cholesterol. Of course, in the cardiovascular community, we’re often looking for outcome trials and there is an ongoing outcome trial. And I think many cardiologists are really waiting for that before jumping on board. I guess I would see in terms of algorithms there’s outcome trial data for the PCSK9 inhibitors, the 2 monoclonal antibodies that are approved. So at least in my own, my algorithms typically statin, ezetimibe, PCSK9 inhibitor. And if the patient’s intolerant to statins, then ezetimibe, then PCSK9 inhibitor. And frankly, ezetimibe may not be doing that much with the PCSK9 inhibitor on board, but I usually still leave it in there. So that’s my typical algorithm. I think that once there’s outcome data for bempedoic acid, I probably feel more comfortable in its broader use. Right now, I think its use is probably, at least what I’ve seen in cardiology practices, more limited to patients who are statin intolerant. Ezetimibe doesn’t do the trick alone in terms of getting their LDL to go and they’re resistant to actually taking an injectable agent i.e. PCSK9 inhibitor. I must say, however, I’ve been surprised how few patients have objected to the injectable nature of the PCSK9 inhibitors. There were concerns that that was going to be a major barrier, but I haven’t actually seen that play out in clinical practice. Whether it’s a sort of a plus in terms of improving adherence, I haven’t necessarily seen that either, at least with the currently available PCSK9 inhibitors. Though there is a longer acting version inclisiran [Leqvio] that’s a bit of a different technology to monoclonal antibody technology where it could be given every 6 months for example, or even in some patients potentially if a lower degree of LDL lowering is required every year. Now, every year in terms of dosing, to me that could actually improve adherence, every 6 months potentially as well. But when the dosing interval is shorter, I haven’t necessarily seen an improvement in adherence from that versus oral therapies. But again, I haven’t seen such a resistance to injectable therapies as Dr Busch provided the anecdote. When you tell a patient that their LDL is dropped by 50%, usually that provides a lot of positive reinforcement to continue the PCSK9 inhibitor. So anyway, I see a much more limited role right now for bempedoic acid until the cardiovascular outcome data are available.

An expert panel discusses where bempedoic acid currently fits into the treatment of hypercholesterolemia. 

Bempedoic Acid for Hypercholesterolemia

Deepak L. Bhatt, MD, MPH, FACC, FAHA, FSCAI, FESC

This month’s Lungcast features a discussion on the ALA’s State of the Air report and how worsening air conditions affect people across the nation.

2025 State of the Air: Dangers of the Air We Breathe with George Thurston, ScD

Nearly half of Americans are breathing unhealthy air, according to the American Lung Association (ALA)’s latest State of the Air report, which analyzes ozone and particle pollution levels across the U.S. Drawing on federally verified data from 2021 to 2023, the report reveals that 156 million people—46% of the U.S. population—live in areas receiving a failing grade for at least one pollutant, with more than 42 million exposed to unhealthy levels of all three key measures: ozone, short-term particulate matter (PM₂.₅), and annual PM₂.₅. Worsening air quality trends, driven by extreme heat and wildfires, have pushed short-term particle pollution to the highest levels in 16 years, posing serious risks to respiratory and cardiovascular health. Yet, large data gaps remain—over 72 million people live in counties without any monitoring, leaving many at-risk communities invisible in national air quality assessments.

An internationally recognized leader in air quality, epidemiology and exposure assessment, George Thurston, ScD, a professor of medicine and population health at New York University School of Medicine headlines this important episode analyzing the Lung Association’s 2025 “State of the Air” report. In this episode, Thurston delves into the acute and accumulative health impacts on communities across the country. Listen in to learn more about the report’s key learnings, from adverse effects of exposure to ground-level ozone pollution to the decade-long worsening trend of particle pollution.

“You really have to have the evidence, and it's hard to have the evidence if you don't have people actually living at these lower levels. So that’s been very slow but positive progress towards cleaner air as we learn more and more about the effects of air pollution at even at lower and lower levels,” Thurston said.

Lungcast is a monthly respiratory news podcast series hosted by Albert Rizzo, MD, chief medical officer of the ALA, and produced by 

George Thurston, ScD

Anti-VEGF for RVO: The Gold Standard

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John W Kitchens, MD, bookends the discussion by highlighting the landscape of branch vein occlusion (BRVO) treatment options. 

Anti-VEGF for RVO: The Gold Standard 

John W. Kitchens, MD

In March 2020 the United States was in the early stages of the coronavirus disease 2019 (COVID-19) pandemic. We shut down the entire country and ground the economy to a halt to slow the spread of the virus. Think back to March and how much uncertainty we were living under.

Nine months later, the FDA approved 2 COVID-19 vaccines under emergency authorization. Before New Year’s Day, millions of Americans had received the vaccine, including frontline physicians and health care providers and nursing home patients, our most vulnerable citizens.

Nine months. Take a moment to let that sink in.

The mainstream media has crafted a narrative about the COVID-19 pandemic that’s almost entirely negative. For the purpose of ratings, they have described the US response to the pandemic as blundering from 1 mistake to the next. This narrative is false.

There is another way—a more accurate and underappreciate way—to tell the story of the past 9 months. It is a story of heroism, innovation, and precise science, performed under unbelievable pressure.

Let’s not mince words: The US and the world must appreciate the role of the pharmaceutical industry—the investigators, physicians, and business leaders—who are rescuing the world from COVID-19. It’s the medical breakthrough of our lifetime.

Instead of dwelling on why many in the media are ignoring this, let’s review some facts.

Since the discovery of COVID-19, here is what scientists have accomplished: They identified a novel virus; unlocked and sequenced its genetic code; created new therapies to save lives; and developed multiple safe and effective vaccines using messenger RNA technology, a technology hopefully applicable to future vaccine development. Margaret Liu, MD, a biomedical scientist and member of the 

, 1 from Pfizer/BioNTech and another from Moderna, and the AstraZeneca/Oxford vaccine has been approved for emergency use in the UK. In addition, there are 64 vaccines undergoing clinical trials at the moment, including 20 in phase 3 trials. In the U.S. and throughout the world, the pharmaceutical industry has answered the call and invested heavily in this effort.

This was the fastest vaccine development program in history, and it’s not even close. David Pride, MD, PhD, a microbiologist at the University of California, San Diego, 

 that vaccines typically take 10-15 years to develop. Until the COVID-19 pandemic, the fastest development timeline was 4 years, for the mumps vaccine.

Many government systems moved quickly to lessen the burden of onerous regulations and provide funding so that vaccines could be developed quickly but with rigorous standards. Perhaps it should be a lesson to all of us that regulation and innovation can be calibrated more effectively during “normal” times as industry races to develop new therapies for other epidemics—cancer, diabetes, heart disease, and more.

The next step of the process—distribution of the vaccine—will be as challenging as the development phase, if not more so. But again, the pharmaceutical industry is rising to the occasion. Factories worldwide are working overdrive to produce hundreds of millions of vaccine doses.

Less than a month after the Pfizer vaccine was approved, more than 15.4 million doses of vaccine have been distributed throughout the country, and more than 4.6 million individuals have received their first dose, 

. Many patients are already receiving their second dose.

Although 15.4 million doses are impressive, some expected 20 million doses. But that is moving the goal line, as 6 months ago many observers didn’t think a vaccine would be available until 2021.

Members of our COVID Coalition told us that the holidays slowed the rollout considerably. Nancy Messonnier, MD, a physician with the National Center for Immunization and Respiratory Diseases at the CDC, 

 in administered vaccines during the first few days of 2021.

Every day, more people will be vaccinated. After health care workers and our most vulnerable citizens, other frontline workers will be next. Teachers will be vaccinated so our children can return to school. And soon, all Americans will be able to receive the vaccine at their doctor’s office or at a CVS or Walgreens.

Remember, we accomplished this in 9 months, with the help, dedication, and expertise of our pharmaceutical industry heroes. Next time you turn on the TV and see negativity, turn it off and imagine instead where we will be in 9 months.

Mike Hennessy Sr is the founder and chairman of MJH Life Sciences.

The US and the world must appreciate the role of the pharmaceutical industry who are rescuing the world from COVID-19—the medical breakthrough of our lifetime.

How Pharmaceutical Innovation is Saving the World from COVID-19

Mike Hennessy Sr, Founder and Chairman, MJH Life Sciences

Rishi Kakar, MD, is the chief scientific officer and medical director of Segal Trials. 

Panelist introductions for an editorial program titled: Embracing New Therapies in Schizophrenia Management

Embracing New Therapies in Schizophrenia Management: Introductions

On September 26, 2024, the US Food and Drug Administration (FDA) approved 

 (Cobenfy), backed by evidence in phase 3 EMERGENT trials. The approval marks the start of a new era in the management of 

, as the agent, formerly known as KarXT, marks the first new pharmacological approach to treating schizophrenia since the 1970s.

Xanomeline and tropism chloride capsules act as a dual M1/M4 muscarinic acetylcholine receptor agonist in the central nervous system. Unlike previous schizophrenia treatments, this drug does not directly regulate the dopamine receptor—only indirectly modulates. 

To commemorate and offer perspective on what this approval means for the healthcare community, 

 hosted a Special Report where leading subject matter experts discussed the approval of xanomeline and tropism chloride capsule for schizophrenia. In this opening segment, moderator Sam Clark, MD, PhD, introduces panelists Rishi Kakar, MD, lead investigator of the EMERGENT trials, and Andrew J. Cutler, MD. The remainder of this panel discussion, which can be found below, focused not only on the approval of xanomeline and tropism chloride capsules but also on the latest updates and the most pressing unmet needs in schizophrenia management.

Mechanism of Action of Xanomeline and Trospium Chloride Capsules

Applying EMERGENT Trial Results to Real-World Clinical Practice for Schizophrenia

Balancing the Benefits and Risks of Xanomeline and Trospium Chloride Capsules

Unmet Needs, Future Research in Schizophrenia and Muscarinic Agonists

(Moderator) is a former practicing psychiatrist and is the founder and chief executive officer of Terran Biosciences.

is a clinical associate professor in the Department of Psychiatry and Behavioral Sciences at SUNY Upstate Medical University and the chief medical officer of the Neuroscience Education Institute.

is a psychiatrist based out of southern Florida as well as the medical director and chief scientific officer of Segal Trials. Kakar served as the principal investigator on the pivotal EMERGENT trials.

Relevant disclosures for Cutler include Karuna, Bristol Myers Squibb, and 

. Relevant disclosures for Kakar include Karuna, Bristol Myers Squibb, and others. Relevant disclosures for Clark include Terran Biosciences.

The FDA's approval of xanomeline and tropism chloride capsules (Cobenfy) on September 26, 2024, introduces a novel pharmacological approach to schizophrenia treatment, the first since the 1970s. This dual M1/M4 muscarinic acetylcholine receptor agonist indirectly modulates dopamine receptors, differing from traditional treatments. A Special Report by HCPLive Psychiatry featured experts discussing the drug's approval, its mechanism, and its application in clinical practice. The panel also addressed unmet needs and future research directions in schizophrenia management, highlighting the significance of this advancement.

In part 1 of 5, panel moderator gives an overview of the Special Report focusing on the approval of Xanomeline and tropism chloride capsules for schizophrenia.

Rishi Kakar, MD

Pongdee also discussed the changing treatment landscape for indolent systemic mastocytosis and questions that will need to be answered in the field. 

Looking Deeper Into ISM and KIT Mutations, with Thanai Pongdee, MD

Indolent systemic mastocytosis (ISM) is a rare, clonal mast cell disease caused by 

 D816V mutations. Patients typically experience chronic debilitating symptoms including allergic type symptoms, gastrointestinal symptoms, and fatigue, and it often takes years for a correct diagnosis to be made.

Thanai Pongdee, MD, allergist-immunologist at Mayo Clinic, is actively engaged in ISM research and coauthored a number of abstracts that were presented at the 

2025 American Academy of Allergy, Asthma, and Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress

, February 28-March 3, in San Diego, California.

One abstract Pongdee presented focused on using more sensitive duplex sequencing (DS) to detect 

mutations that were undetectable by droplet digital PCR (ddPCR) in patients that received avapritinib in the PIONEER (NCT03731260), and associations with response.

 Pongdee and colleagues found that 70% of participants with undetectable 

 D816V by ddPCR had KIT mutations, including non-D816V mutations, detected by DS.

spoke with Pongdee to learn more about the findings from the analysis. He also discussed the changing treatment landscape of ISM and questions that remain to be answered past the introduction of novel therapies.

“Once drugs are approved, then what? What's the course of disease after that? We really don't know that, so that'll be interesting to watch over time, and [effects on] some of the longer-term issues with mastocytosis, [including higher risk of] osteoporosis or bone disease,” Pongdee said. “Can these drugs affect or alter the course?”

Pongdee’s disclosures include GSK, Blueprint Medicines, and Sanofi.

Systemic mastocytosis. Mayo Clinic. Updated November 20, 2020. Accessed March 11, 2025. 

https://www.mayoclinic.org/diseases-conditions/systemic-mastocytosis/symptoms-causes/syc-20352859

Pongdee T, Vachhani P, Tashi T, et al. Responses to Avapritinib in Patients Without Detectable KIT Mutations by ddPCR in Peripheral Blood Highlight Diagnostic Challenges and Opportunities in Indolent Systemic Mastocytosis. Presented at: 2025 AAAAI/WAO Joint Congress, February 28-March 3. Abstract 965

Authors