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Ryan Livingston joined the MJH Life Sciences team in March of 2025, shortly after graduating from TCNJ with a bachelor’s degree in English. He enjoys creative writing, reading, and scuba diving. He can be reached at rlivingston@mjhlifesciences.com.

Hematology has been relatively silent compared to other fields in recent months, with few new treatments developed and fewer approvals from the 

. The majority of conditions, despite still being prevalent, are relatively well-handled.

Q2 2025 largely followed this pattern, with the FDA handing out a few designations and no significant approvals. Trial results varied as well, with a handful of advances tempered by the destructive failure of birtamimab to reach its primary endpoint. Now that the end of Q2 has arrived, 

 has put together a recap of the biggest news and headlines from April to June 2025.

In this recap, we highlight 3 regulatory updates from the FDA, our 4 biggest trial announcement articles, and 2 key insights from prominent experts featured within our coverage.

FDA Grants Orphan Drug Designation to DIAG723 for Treatment of HHT

On June 18, DIAG723 received Orphan Drug designation from the FDA and a positive opinion from the European Medicines Agency (EMA), both for the treatment of hereditary hemorrhagic telangiectasia. Parent company Diagonal Therapeutics recently began a natural history study of adults with HHT earlier this year, partnering with the organization Cure HHT to characterize the variability of patient-reported outcomes, such as hematologic support, quality of life, and epistaxis.

FDA Grants Fast Track Designation to Mavorixafor for Chronic Neutropenia

On June 10, X4 Pharmaceuticals announced the receipt of Fast Track designation from the FDA for mavorixafor, a treatment for chronic neutropenia. Mavorixafor is currently being examined in the phase 3 4WARD trial, which aims to evaluate the efficacy, safety, and tolerability of oral, once-daily treatment with or without injectable G-CSF. The trial will last 52 weeks with a primary endpoint based on 2 separate outcome measures of annualized infection rate and positive ANC response.

VAS-101 (Vasceptor) Receives Orphan Drug Designation for Sickle Cell Disease

On June 12, Vascarta announced receipt of an Orphan Drug designation from the FDA for VAS-101, a treatment for sickle cell disease. A phase 1 clinical trial is currently underway to investigate the safety and tolerability of VAS-101, as well as its effects on impaired blood flow dynamics like adhesion molecule expression and erythrocyte fragility parameters. Investigators are also examining the mean change in red blood cell sickling kinetics and oxygen dissociation curves.

Q2 2025 Trial Announcements in Hematology

Ruxoprubart Shows Efficacy for PNH in Interim Phase 2 Trial Results

Ruxoprubart, a novel complement-targeting immunotherapy typically administered as a monotherapy, reached all primary efficacy endpoints in interim data released by parent company NovelMed. These endpoints included transfusion avoidance, increased hemoglobin levels, reduced lactate dehydrogenase, and increased PNH clone size. NovelMed has also indicated an interest in partnerships to further develop the treatment.

Birtamimab Misses Primary Phase 3 Endpoint for AL Amyloidosis

Prothena Corporation announced the failure of birtamimab in its phase 3 AFFIRM-AL clinical trial, accompanied by a plan to cut operating costs and shrink its workforce. An investigational, humanized monoclonal antibody, birtamimab was designed to selectively target and clear the amyloid leading to organ dysfunction and failure. Despite receiving Orphan Drug designation from both the FDA and EMA, as well as an agreement between Prothena and the FDA for a Special Protocol Assessment, the phase 3 trial was an overall failure.

Iptacopan (Fabhalta) Hits Primary Endpoint in Phase 3 APPULSE-PNH Trial

Novartis announced iptacopan’s success in APPULSE-PNH, a phase 3b trial investigating its safety and efficacy. A twice-daily oral monotherapy for the treatment of paroxysmal nocturnal hemoglobinuria, iptacopan exhibited no new safety signals and was well-tolerated by patients. In addition, it resolved extravascular hemolysis control and reduced absolute reticulocyte count in treated patients.

Genetically Modified Cell Therapy BEAM-101 Successful in Sickle Cell Disease Trial

Announced by parent company Beam Therapeutics, BEAM-101, a genetically modified cell therapy for the treatment of sickle cell disease, has demonstrated its strong clinical profile in the BEACON phase 1/2 clinical trial. BEAM-101 is intended to facilitate the production of non- and anti-sickling fetal hemoglobin; among these results, investigators also found normalization of a variety of hemolysis markers, such as indirect bilirubin, haptoglobin, and lactate dehydrogenase.

Q2 2025 Expert Perspectives in Hematology

Pediatric Year in Review: New Therapies in Sickle Cell Disease, with Shaina Willen, MD

Shaina Willen, MD, a board-certified provider in Pediatric Hematology/Oncology and Pediatric Pulmonology from UC Davis Health, speaks on new therapies for sickle cell disease and the recent drastic increase in options for patients.

A Positive Outlook on Gene Therapy for Hemophilia B Post-Discontinuation, with Guy Young, MD

Guy Young, MD, director of the Hemostasis and Thrombosis program at the Children’s Hospital of Los Angeles and professor of pediatrics at the Keck School of Medicine of the University of Southern California, discusses the discontinuation of fidanacogene elaparvovec development and its implications for the future of gene therapy in treating hemophilia B.

Articles

Hematology has seen limited advancements recently, with few new treatments and FDA approvals. In Q2 2025, the FDA granted several designations, including Orphan Drug status for DIAG723 and VAS-101, and Fast Track for mavorixafor. Trial results were mixed, with ruxoprubart and iptacopan showing promise, while birtamimab failed its primary endpoint. BEAM-101 demonstrated success in sickle cell disease trials. Expert insights highlighted new therapies for sickle cell disease and the future of gene therapy for hemophilia B, despite the discontinuation of fidanacogene elaparvovec development.

A relatively quiet quarter for hematology, punctuated by both successes and failures in clinical trials and a handful of Orphan Drug and Fast Track designations.

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Paroxysmal Nocturnal Hemoglobinuria

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Sickle Cell

Sickle Cell condition center page, resources on the topics of medical news and expert insight into sickle cell disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on sickle cell research, treatment, and drug development.

Q2 2025 Recap: Hematology News and Updates

Victoria Johnson joined the MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and now HCPLive. You can reach her at vjohnson@mjhlifesciences.com

In June, new data offered important updates in the treatment of psoriatic arthritis, uncontrolled gout, fibromyalgia, and asthma, highlighting both advances in therapeutic options and lingering gaps in care. Notably, deucravacitinib demonstrated sustained joint and skin symptom improvements in adults with psoriatic arthritis in the pivotal POETYK PsA-1 and PsA-2 trials, while monthly high-dose pegloticase infusions with methotrexate showed feasibility for uncontrolled gout, potentially reducing treatment burden. In fibromyalgia, a comprehensive Cochrane analysis underscored the limited but meaningful role of duloxetine, milnacipran, and pregabalin, and TNX-102 SL emerged as a promising candidate to become the first new FDA-approved therapy for fibromyalgia in over 15 years. Finally, a large meta-analysis reinforced guideline recommendations against SABA monotherapy, linking its overuse with increased mortality and acute exacerbations in asthma, underscoring the need for ongoing optimization of asthma management strategies.

Check out this June 2025 rheumatology month in review for a recap of HCPLive’s coverage of the top pulmonary news and research from the past few weeks:

Phase 3 Deucravacitinib Trial Meets ACR20 End Point in PsA

The phase 3 POETYK PsA-1 trial demonstrated that deucravacitinib significantly improved joint and skin symptoms in adults with active psoriatic arthritis, meeting its primary endpoint with 54.2% of treated patients achieving ACR20 response at week 16 versus 34.1% on placebo. Key secondary endpoints were also met, including significant improvements in skin clearance, physical function, and quality of life measures, alongside evidence of inhibited radiographic progression. The safety profile of deucravacitinib was consistent with previous studies, with no new safety signals identified, supporting its potential as an oral, first-in-class TYK2 inhibitor for psoriatic arthritis management.

Higher Dose Pegloticase Q4W Feasible for Uncontrolled Gout

The phase 4 FORWARD trial suggests that once-monthly (Q4W) dosing of pegloticase at 16 mg or 30 mg, combined with methotrexate, can maintain serum urate control in most patients with uncontrolled gout, with 68-73% achieving sustained sUA responses through month 6. These findings indicate that less frequent pegloticase infusions could improve treatment logistics and adherence while maintaining comparable safety and efficacy to the established Q2W regimen. Importantly, infusion reaction rates were low and consistent with prior studies, supporting monthly pegloticase as a feasible alternative for patients struggling with the burden of biweekly therapy.

New Data Further Support Safety of Upadacitinib With 26-Week GC Taper for Giant Cell Arteritis

In the SELECT-GCA phase 3 trial, upadacitinib 15 mg daily with a 26-week glucocorticoid taper showed numerically lower rates of serious infections (7.9 events/100 patient-years) compared with placebo on a 52-week taper (12.7 E/100 PY), suggesting reduced infection risk with a shorter steroid course. Herpes zoster rates were higher with upadacitinib 15 mg but remained within expected ranges, while opportunistic infections were rare and mostly mild.

Inebilizumab Efficacy and Safety Consistent Across Demographic Subgroups of IgG4-RD

A post-hoc analysis of the phase 3 MITIGATE trial confirmed inebilizumab’s effectiveness in reducing IgG4-related disease flares across diverse demographic subgroups, including variations by age, sex, and race. The treatment consistently lowered annualized flare rates and increased rates of flare-free, corticosteroid-free remission at 52 weeks compared to placebo, regardless of baseline characteristics.

A New Fibromyalgia Therapy on the Horizon

New Data Support TNX-102 SL Efficacy Ahead of PDUFA

TNX-102 SL, a sublingual cyclobenzaprine formulation, showed significant efficacy in reducing pain and improving sleep disturbance in fibromyalgia patients in the phase 3 RESILIENT study, with benefits sustained over 14 weeks. The phase 1 PK study demonstrated that TNX-102 SL achieves higher dynamic peak cyclobenzaprine levels with reduced exposure to its active metabolite norcyclobenzaprine, which may contribute to more durable effects compared to oral formulations. If approved by its August 15, 2025 PDUFA date, TNX-102 SL could become the first new FDA-approved fibromyalgia therapy in over 15 years and a novel non-opioid analgesic option for this challenging chronic pain condition.

Other Hot Topics in Rheumatology Research

Rheumatoid Arthritis Burden Has Surged Over Last 30 Years

A large AI-driven analysis of rheumatoid arthritis (RA) trends across 953 global locations revealed a 13.2% rise in RA incidence since 1990, affecting 17.9 million people in 2021, with a shift toward younger populations. While age-standardized RA mortality dropped by 32.7% since 1980, global RA-related DALYs nearly doubled, highlighting an expanding burden despite medical advances. The study also pinpointed local hotspots—like West Berkshire, UK, with the highest incidence—and demonstrated that targeted interventions, such as smoking reduction, could substantially lower RA burden, emphasizing the importance of localized strategies beyond socioeconomic status alone.

Pharmacological Therapies Largely Ineffective Against Fibromyalgia Syndrome Pain

A comprehensive Cochrane overview of pharmacologic treatments for fibromyalgia found moderate to good evidence supporting duloxetine, milnacipran, and pregabalin for reducing moderate-to-severe pain in about 1 in 10 adults, with benefits sustained over 4-12 weeks. Among 21 included reviews, many therapies—such as NSAIDs, antipsychotics, and cannabinoids—had inadequate or no data, while amitriptyline and SSRIs showed signs of publication bias and mirtazapine had moderate evidence of no effect. Overall, while adverse events were relatively common, serious events were rare, underscoring the limited yet important role of these few agents in fibromyalgia pain management.

June's updates in rheumatology and pulmonary research reveal significant advancements and ongoing challenges in treating psoriatic arthritis, uncontrolled gout, fibromyalgia, and asthma. Deucravacitinib showed promising results in psoriatic arthritis trials, while monthly pegloticase infusions with methotrexate offer a feasible option for uncontrolled gout. TNX-102 SL emerges as a potential new fibromyalgia therapy, and a meta-analysis highlights the risks of SABA monotherapy in asthma. Additionally, rheumatoid arthritis incidence has increased, emphasizing the need for targeted interventions to manage the growing burden of this condition.

The rheumatology month in review emphasizes new research in RA burden, fibromyalgia treatment, and insights from the EULAR 2025 meeting.

Rheumatology

Rheumatology condition center page is a comprehensive resource for clinical news and insights on rheumatologic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for arthritis, gout, nr-AxSpA, and more.

Fibromyalgia

On the HCPLive fibromyalgia page, resources on the topics of medical news and expert insight into fibrositis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on fibromyalgia, and more.

Psoriatic Arthritis

On the HCPLive psoriatic arthritis page, resources on the topics of medical news and expert insight into PsA can be found. Content includes articles, interviews, videos, podcasts, and breaking news on arthritis, and more.

Gout

On the HCPLive gout page, resources on the topics of medical news and expert insight into gouty arthritis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on gout, and more.

Rheumatology Month in Review: June 2025

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.

 review, several key advances indicate treatment progress. The US Food and Drug Administration (FDA) accepted the new drug application (NDA) for Anaphylm, a needle-free epinephrine film, and approved garadacimab-gxii (ANDEMBRY) for hereditary angioedema (HAE) prevention. LYR-210, a six-month nasal implant, showed strong phase 3 results for chronic rhinosinusitis (CRS).

A meta-analysis confirmed the safety of biologics like omalizumab for IgE-mediated food allergies. Finally, new data support the “two-hit” hypothesis: early allergic sensitization combined with respiratory infections may impair lung function into adulthood. This month in review features the top 5 allergy news stories in June.

FDA Allergy Updates with Anaphylm NDA and Garadacimab Approval for HAE

FDA Accepts Epinephrine (Anaphylm) Sublingual Film NDA for Type 1 Allergic Reactions

The FDA has accepted Aquestive Therapeutics’ NDA for Anaphylm, a needle-free sublingual epinephrine film designed to treat severe allergic reactions, including anaphylaxis. If approved, it would be the first orally delivered epinephrine option in the US, with a PDUFA action date set for January 31, 2026.

Anaphylm dissolves quickly, is portable, and has shown rapid symptom relief in both adult and pediatric trials with no serious adverse events. Experts believe this device-free option could transform anaphylaxis care by addressing key barriers to epinephrine use: fear of injection and inconvenient storage.

FDA Approves Factor XIIa-Targeting Garadacimab-gxii (ANDEMBRY) for HAE Attacks

The FDA has approved garadacimab-gxii (ANDEMBRY), the first HAE treatment targeting factor XIIa, for patients aged ≥ 12 years. Delivered monthly via a fast, citrate-free subcutaneous autoinjector, the monoclonal antibody offers long-term prevention of HAE attacks.

In the phase 3 VANGUARD trial, the drug reduced attacks by ≥ 99% compared to placebo, with 62% of patients remaining attack-free. Common adverse events included nasopharyngitis and abdominal pain. Long-term data support its safety and efficacy.

LYR-210 Nasal Implant Succeeds in Phase 3 for CRS

LYR-210 Nasal Implant for CRS Brings Symptom Relief in Phase 3 ENLIGHTEN 2

The phase 3 ENLIGHTEN 2 trial showed that LYR-210, a bioabsorbable nasal implant delivering 6 months of continuous mometasone furoate, significantly improved CRS symptoms in patients without nasal polyps. By week 24, patients experienced relief in nasal obstruction, discharge, and facial pain, along with over a 20-point improvement in SNOT-22 scores, exceeding the threshold for clinical significance.

The implant was well-tolerated, with no serious adverse events. CT scans and secondary endpoints supported its benefit, and fewer LYR-210 patients required sinus surgery. Results mark a promising advance for patients with CRS unresponsive to existing therapies.

New Research on Biologics and Lung Impact in Allergy

Biologic Treatments Safe to Recommend for IgE-Mediated Food Allergy, Study Suggests

A new meta-analysis suggests biologic therapies like omalizumab are safe and effective for treating IgE-mediated food allergies. Reviewing 13 randomized controlled trials involving more than 1,000 patients, investigators found that biologics, used alone or with oral immunotherapy (OIT), significantly increased food tolerance and reduced allergic reactions without raising the risk of severe side effects.

Omalizumab, the most studied biologic, helped patients tolerate allergens like peanuts and cow’s milk. While results support the use of biologics in food allergy management, investigators highlighted the need for more long-term data, standardization, and cost-effectiveness analyses to fully guide clinical care.

Childhood Sensitization Plus Respiratory Infections Impair Lung Function by 25

A new study supports the “two-hit” hypothesis, showing that early allergic sensitization combined with respiratory infections in childhood can impair lung function into adulthood, up to age 25 years. The team followed participants from the Melbourne Atopy Cohort and found that sensitized children with frequent infections showed reduced lung function, while infections in non-sensitized children appeared to be protective against lower lung function.

The findings suggest that early-life sensitization makes lungs more vulnerable to damage from infections, while those without sensitization may benefit from immune maturation. The study underscores the importance of preventing respiratory infections in sensitized children to help preserve long-term lung health.

Recent advancements in allergy treatment include the FDA's acceptance of Anaphylm, a needle-free epinephrine film, and approval of garadacimab-gxii for hereditary angioedema prevention. LYR-210, a nasal implant, showed promising results for chronic rhinosinusitis. A meta-analysis confirmed the safety of biologics like omalizumab for IgE-mediated food allergies. Additionally, new research supports the "two-hit" hypothesis, indicating that early allergic sensitization combined with respiratory infections can impair lung function into adulthood, emphasizing the need for preventive measures in sensitized children.

This month in review captures 5 allergy news in June, from FDA approvals to phase 3 data.

Allergy

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.

Allergy Month in Review: June 2025

Abigail Brooks is an assistant 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.

June brought notable momentum across the field of 

, with key regulatory approvals, emerging data from clinical trials, and new insights into modifiable liver disease risk factors.

The US Food and Drug Administration (FDA)’s label expansion for glecaprevir/pibrentasvir (Mavyret) marked the first approval of a direct-acting antiviral for acute 

 treatment in both adults and children, while phase 2b results for pemvidutide highlighted its dual potential in 

metabolic dysfunction-associated steatohepatitis (MASH)

 resolution and weight loss. Promising phase 1b findings for a novel hepatitis B virus (HBV) capsid assembly modulator added to the month’s pipeline activity.

Meanwhile, new population-based studies offered fresh perspective on the downstream risks of weight cycling, dietary salt intake, and the broader benefits of successful HCV therapy, reinforcing the importance of holistic risk management in liver health.

Check out this June 2025 hepatology month in review for a recap of

’s coverage of the top hepatic news and research from the past few weeks:

Regulatory, Pipeline Updates in Hepatology

FDA Approves Glecaprevir/Pibrentasvir (Mavyret) Label Expansion for Acute HCV

On June 11, 2025, the FDA approved a label expansion for AbbVie’s glecaprevir/pibrentasvir (Mavyret), an oral pangenotypic direct acting antiviral (DAA) therapy. With the decision, glecaprevir/pibrentasvir became approved for the treatment of adults and pediatric patients ≥ 3 years of age with acute or chronic HCV infection without cirrhosis or with compensated cirrhosis.

The approval was supported by data from the phase 3, multicenter, single-arm prospective M20-350 study evaluating the safety and efficacy of glecaprevir/pibrentasvir 8-week treatment in adults with acute HCV infection. Of note, the decision made glecaprevir/pibrentasvir the first and only DAA therapy approved to treat patients with acute HCV in 8 weeks with a 96% cure rate.

Pemvidutide Shows Significant MASH Effects, Weight Loss at 24 Weeks in Phase 2b IMPACT Trial

According to a June 26, 2025, announcement from Altimmune, the phase 2b IMPACT trial of pemvidutide in patients with MASH met its primary endpoint with statistically significant MASH resolution without worsening of fibrosis in up to 59.1% of participants and fibrosis improvement without worsening of MASH in up to 34.5% of participants in intent-to-treat analyses as well as weight loss of up to 6.2% at 24 weeks with no plateauing. Of note, the data make pemvidutide the first product candidate to demonstrate significant MASH effects and weight loss at 24 weeks.

Topline Phase 1b Data Support Once-Daily Oral ABI-4334’s Potential for Chronic HBV

On June 25, 2025, Assembly Biosciences announced positive topline efficacy, safety, and pharmacokinetic (PK) results from a phase 1b study evaluating ABI-4334, an investigational next-generation capsid assembly modulator (CAM), in patients with chronic HBV. A favorable safety and tolerability profile, as well as pharmacokinetics supporting once-daily oral dosing, continued to be observed in the final 400 mg dose cohort, similar to that previously reported for the 150 mg dose cohort.

Successful HCV Treatment with DAAs Linked to Lower Extrahepatic Manifestation Risk

Recent research suggests successful HCV treatment with direct-acting antivirals (DAAs) is associated with a reduced risk of several extrahepatic manifestations. Leveraging data from the BC Hepatitis Testers Cohort, the study found successful DAA treatment leading to sustained virologic response was linked to lower risks of 

, major adverse cardiac events, and neurocognitive disorders, but not 

Weight Cycling Increases Risk of MASLD, Other Cardiometabolic Diseases

Going through cycles of weight loss and subsequent regain, known as weight cycling, increases the risk of several cardiometabolic diseases compared with weight stability, according to findings from this study. Leveraging de-identified electronic health record data from > 80,000 adults treated at Vanderbilt University Medical Center, the study found that being a weight cycler significantly increased the risk of 

metabolic dysfunction-associated steatotic liver disease (MASLD)

Frequently Adding Salt to Food May Increase Liver Disease Risk, Study Finds

Leveraging data for nearly 500,000 UK Biobank participants, a recent study found frequently adding salt to foods may incur a greater risk of incident MASLD, cirrhosis, and hepatocellular carcinoma. Those who self-reported more frequent use of salt were at an increased risk of developing these liver-related disorders than those who said they less frequently added salt to their food, suggesting that reducing salt/sodium intake could be a promising strategy for public health initiatives to promote liver health.

June saw significant advancements in hepatology, including FDA approval of glecaprevir/pibrentasvir for acute HCV in adults and children, marking a milestone in antiviral therapy. Phase 2b results for pemvidutide showed promise in MASH resolution and weight loss, while phase 1b data for ABI-4334 highlighted its potential for chronic HBV. New studies emphasized the risks of weight cycling and high dietary salt intake on liver health, and the benefits of successful HCV therapy in reducing extrahepatic manifestations, underscoring the importance of comprehensive risk management.

The month in review spotlights hepatic regulatory news, new clinical trial data, and novel research about liver disease risk factors.

Hepatology

Hepatitis C

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MASH / MASLD

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Hepatology Month in Review: June 2025

Ophthalmology has been on a tremendous rise in recent years, with developments on almost every front and new treatments for a slew of diseases. Upcoming therapies and groundbreaking technology have paved the way for attempts to lighten the treatment burden on patients by extending dosing intervals; a number of medications have exhibited efficacy outside of their expected field in ophthalmology; and trial successes have come hard and fast.

Q2 2025 followed many of these patterns, with the 

 handing out approvals to several critically important medications and companies worldwide announcing successful advancements on first-of-their-kind drugs. Niches were filled, treatment burdens were lessened, and alternative therapies were discovered. To mark the end of Q2 2025, 

 created a recap of the biggest news from April to June 2025.

This recap collects 5 regulatory updates from the FDA, our 5 biggest trial announcement articles, and 3 critical insights from top experts featured prominently within our coverage.

Q2 2025 Regulatory Updates in Ophthalmology

FDA Issues Complete Response Letter to Aflibercept 8 mg for Extended Dosing

Announced on April 18, this CRL was specifically for parent company Regeneron’s proposed dosage extension up to 24-weeks. No safety or efficacy issues of aflibercept 8 mg in any of its approved dosing regimens or indications were found; the FDA disagreed instead with an attempt to extend dosing intervals longer than the current maximum of 16 weeks according to the label.

FDA Approves Prednisolone Acetate Ophthalmic Suspension for Ocular Inflammation

On June 12, 2025, the FDA approved the sterile, topical anti-inflammatory agent for the treatment of steroid-responsive ocular inflammation. The medication is a self-administered eye drop intended for use 2-4 times daily. In addition to this approval, parent company Amneal Pharmaceuticals announced a planned launch for the third quarter of 2025.

FDA Approves Acoltremon Ophthalmic Solution (TRYPTYR) for Dry Eye Disease

On May 28, 2025, the FDA approved acoltremon, a first-in-class transient receptor potential melastatin 8 channel receptor agonist which stimulates corneal sensory nerves, to treat dry eye disease. Both pivotal phase 3 trials, COMET-2 and COMET-3, displayed rapid onset and sustained tear production, as well as a substantial percentage of patients with a ≥10mm increase in unanesthetized Schirmer’s score across both trials compared to vehicle.

FDA Approves Susvimo for Treatment of Diabetic Retinopathy

Announced on May 22, 2025, this approval marked the third approved indication for Susvimo, along with diabetic macular edema. The medication was made available to US retina specialists from the day that parent company Genentech announced the FDA approval. Susvimo is now the first and only FDA-approved continuous delivery treatment able to maintain vision in people with diabetic retinopathy with only 1 refill every 9 months.

FDA Grants IND Clearance to Immunoglobulin Eye Drops for Dry Eye Disease

On May 21, 2025, the FDA granted Investigational New Drug clearance to Selagine, Inc.’s immunoglobulin drops, an anti-inflammatory and immunomodulatory biologic drug for the treatment of dry eye disease. Selagine and partner Grifols, one of the leading producers of plasma-derived medicines, expect that the IG drops will reach retail pharmacies in early 2029.

Q2 2025 Trial Announcements in Ophthalmology

VVN461 Trial Topline Results Announced for Non-Infectious Anterior Uveitis

Announced April 24, 2025, VivaVision announced that both VVN461-1.0% and VVN461-0.5% showed non-inferior efficacy against a prednisolone acetate comparator cohort in treating NIAU. Although VivaVision is based in China, the company has announced its intention to request a type C meeting with the FDA regarding phase 3 trials and an eventual BLA.

Pegcetacoplan Reduces Rate of Geographic Atrophy Growth, 1-Year Trial Data Shows

On April 23, 2025, 12-month results from the ongoing GALE open-label extension study indicated pegcetacoplan’s ability to reduce the mean rate of geographic atrophy growth. Initiated in response to positive results from the 2-year OAKS and DERBY trials of pegcetacoplan in patients with GA secondary to AMD, GALE is expected to significantly advance the evidence for the medication’s long-term safety and efficacy by its culmination.

Oral Zervimesine Reduces Geographic Atrophy Lesion Growth in Phase 2 Trial

Announced by Cognition Therapeutics on May 8, 2025, the phase 2 MAGNIFY trial resulted in oral zervimesine reducing lesion growth in patients with geographic atrophy secondary to age-related macular degeneration. Simultaneously being developed for Alzheimer’s and dementia with Lewy bodies, the once-daily oral pill may overcome the limitations of current treatment options, which require regular intravitreal injections.

Veligrotug Shows Durability for Thyroid Eye Disease in Phase 3 Trial Results

Long-term data announced on May 20, 2025, by Viridian Therapeutics, Inc. indicated the positive long-term durability of veligrotug in treating thyroid eye disease. Veligrotug, already granted the Breakthrough Therapy designation by the FDA prior, is also on track with its BLA planned for the second half of 2025.

Opthea Announces Termination of ShORe and COAST Trials of Sozinibercept

On April 2, 2025, Opthea announced the termination of their ShORe and COAST trials after sozinibercept failed to achieve vision improvement benchmarks in treating wet AMD. COAST failed on March 24, which led Opthea to accelerate topline data of the ShORe trial. However, when this trial also missed its benchmarks, Opthea shuttered the trials.

Phase 2 LUNA Trial Results for Ixo-vec to Treat Neovascular AMD with Dante Pieramici, MD

Dante Pieramici, MD, assistant clinical professor of ophthalmology at the Doheny Eye Center in Southern California, discusses the results of the phase 2 LUNA trial of ixoberogene Soroparvovec intravitreal gene therapy for the treatment of neovascular age-related macular degeneration.

Changing Dosage Regimens in Patients with Diabetic Macular Edema, with Mark Barakat, MD

Mark Barakat, MD, founder and director of research at the Retina Macula Institute of Arizona, discusses his post-hoc analysis examining the shortening or extending of aflibercept 8 mg dosage for patients with DME through week 96 of the PHOTON trial.

Switching to Aflibercept 8 mg in Treatment-Experienced Patients with Ted Leng, MD

Theodore Leng, MD, Director of Clinical and Translational Research and Director of Ophthalmic Diagnostics at the Stanford University School of Medicine, discusses the process of switching to aflibercept 8mg from other anti-VEGF agents in patients with neovascular age-related macular degeneration.

Switching to Aflibercept 8 mg in Treatment-Experienced Patients with Theodore Leng, MD, FACS

Ophthalmology has seen significant advancements, with new therapies and technologies reducing treatment burdens and expanding medication efficacy. In Q2 2025, the FDA approved several key medications, including prednisolone acetate for ocular inflammation and acoltremon for dry eye disease. Susvimo received approval for diabetic retinopathy, and immunoglobulin eye drops gained IND clearance. Notable trial results included pegcetacoplan's impact on geographic atrophy and veligrotug's durability for thyroid eye disease. Expert insights highlighted dosage regimen changes and treatment switches in diabetic macular edema and neovascular AMD.

Our Q2 2025 recap for ophthalmology highlights 5 regulatory updates, 5 important trial announcements, and top perspectives in eyecare.

Ophthalmology

Ophthalmology page, resources on the topics of medical news and expert insight into ophthalmic disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on eye disease research, treatment, and drug development.

Diabetic Macular Edema

The HCPLive diabetic macular edema page is a comprehensive resource for clinical news and insights on DME. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for the leading cause of blindness, and more.

Age-Related Macular Degeneration

age-related macular degeneration condition center page is a comprehensive resource for clinical news and insights on retina disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for patients with AMD.

Uveitis

The HCPLive uveitis page is a comprehensive resource for clinical news and insights on chorioretinitis. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for eye inflammation, and more.

Geographic Atrophy

Q2 2025 Recap: Ophthalmology News and Updates

June 2025 saw more research emerge in asthma therapeutics and management as well as rarer pulmonary diseases.

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.

Asthma

The HCPLive asthma page is a comprehensive resource for clinical news and insights on asthmatic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for allergic and severe asthma, and more.

Pulmonology Month in Review: June 2025

Tim Smith joined the MJH Life Sciences team as an assistant editor for HCPLive in August 2022. He graduated from UC Berkeley with a degree in political science, working in multimedia journalism as a staff writer prior to joining MJH. In his spare time, he enjoys reading, watching TV, listening to podcasts, and rock climbing. You can contact him at 

This FDA news recap highlights some of the most notable approvals and actions by the organization in dermatology during Q2 2025.

Dermatology

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

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.

5 FDA Approvals in Dermatology: Q2 Recap

The month in review spotlights perspectives on collaborative GI care, updates in C diff, and new research about disorders of gut brain interaction.

Gastroenterology

Gastroenterology condition center page is a comprehensive resource for clinical news and insights on digestive and GI conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for 

, IBS and IBD, Crohn's disease, and more.

Irritable Bowel Syndrome

On the HCPLive irritable bowel syndrome page, resources on the topics of medical news and expert insight into IBS can be found. Content includes articles, interviews, videos, podcasts, and breaking news on IBS, IBD, and more.

Latest News

Q2 2025 Recap: Hematology News and Updates

Rheumatology Month in Review: June 2025

Allergy Month in Review: June 2025

Hepatology Month in Review: June 2025

Q2 2025 Recap: Ophthalmology News and Updates