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

Victoria Johnson

Welcome back to our recap of this month's news and updates from the 

November brought a series of impactful FDA decisions spanning rare disease, pediatrics, neuromuscular disorders, and cardiometabolic care—each marking a meaningful expansion in therapeutic options for historically underserved patient populations. The month opened with the approval of doxycitine and doxribtimine (Kygevvi), the first treatment for thymidine kinase 2 deficiency, offering survival and functional benefits in an ultra-rare mitochondrial disorder with no prior therapies. Soon after, linaclotide became the first FDA-approved option for pediatric irritable bowel syndrome with constipation (IBS-C) in patients ≥7 years old, supported by efficacy signals consistent with adult outcomes. Mid-month, the agency issued significant safety-driven changes to ELEVIDYS, restricting its use to ambulatory patients with Duchenne muscular dystrophy (DMD) and adding a Boxed Warning following reports of acute liver failure in non-ambulatory recipients.

Regulators also advanced innovation in lipid and renal disease. The approval of plozasiran introduced the first apoC-III–targeting siRNA therapy for familial chylomicronemia syndrome, offering meaningful reductions in triglycerides, apoC-III, and pancreatitis risk with quarterly dosing. Closing the month, sibeprenlimab received accelerated approval for IgA nephropathy after demonstrating a more than 50% placebo-adjusted reduction in proteinuria at one year, adding momentum to a rapidly evolving treatment landscape ahead of confirmatory eGFR data. Collectively, these decisions signal an FDA increasingly focused on precision mechanisms and high-need populations across a diverse therapeutic spectrum. Here’s a concise overview of the key FDA decisions and updates from November 2025.

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA Approves Kygevvi, First Treatment for Thymidine Kinase 2 Deficiency

On November 3, 2025, UCB announced the FDA approved doxycitine and doxribtimine (Kygevvi) as the first therapy for thymidine kinase 2 deficiency (TK2d). Based on phase 2 clinical data, retrospective analyses, and an expanded access program, Kygevvi demonstrated improved survival and functional outcomes, including markedly lower mortality compared with untreated patients. The combined nucleoside therapy supports mitochondrial DNA replication and skeletal muscle function in this ultra-rare mitochondrial disorder.

FDA Approves Linaclotide (Linzess) Capsules for Pediatric IBS-C

On November 5, 2025, Ironwood Pharmaceuticals announced the FDA approved linaclotide (Linzess) for pediatric patients ≥7 years of age with irritable bowel syndrome with constipation (IBS-C), making it the first therapy authorized for IBS-C in this age group. Based on extrapolated adult efficacy data and a 12-week randomized pediatric trial, linaclotide 145 mcg once daily demonstrated reductions in abdominal pain and increases in spontaneous bowel movements consistent with adult outcomes. The safety profile was similar to that observed in adults, with diarrhea reported as the most common adverse event.

FDA Issues New Boxed Warning, Limiting ELEVIDYS to Ambulatory DMD

On November 14, 2025, the FDA announced new safety warnings and labeling revisions for delandistrogene moxeparvovec-rokl (Elevidys), restricting its indication to ambulatory patients with Duchenne muscular dystrophy.

Based on postmarketing safety reports, including fatal cases of acute liver failure in non-ambulatory recipients, the agency added a Boxed Warning for serious liver injury and mandated a postmarketing observational study with serial liver function monitoring. Updated guidance includes enhanced recommendations for hepatic and cardiac monitoring, cautions regarding corticosteroid-associated risks, and new contraindications for certain patient populations.

FDA Approves Plozasiran for Adults With Familial Chylomicronemia Syndrome

On November 18, 2025, Arrowhead Pharmaceuticals announced the FDA approved plozasiran (Redemplo) for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS). Based on the phase 3 PALISADE trial and supportive phase 2 SUMMIT data, plozasiran significantly lowered fasting triglycerides and apoC-III levels and reduced acute pancreatitis risk, with dosing once every 3 months. The small-interfering RNA therapeutic demonstrated a favorable tolerability profile and represents the first approved therapy targeting apoC-III reduction for FCS.

RX Review: Leveraging siRNA in FCS Management

FDA Awards Accelerated Approval to Sibeprenlimab in IgA Nephropathy

On November 25, 2025, Otsuka Pharmaceutical announced the FDA approved sibeprenlimab (Voyxact) to reduce proteinuria in adults with primary IgA nephropathy at risk for disease progression. Based on results from the phase 3 VISIONARY trial, sibeprenlimab produced a 54.3% placebo-adjusted reduction in 24-hour urine protein-to-creatinine ratio after 12 months, with consistent effects across clinical subgroups. The APRIL-targeting antibody adds to the growing therapeutic landscape for IgA nephropathy and received accelerated approval pending full 2-year eGFR results expected in 2026.

Kidney Compass: Sibeprenlimab and the VISIONARY Trial, with Vlado Perkovic, MBBS, PhD, at ERA 2025

Articles

November saw significant FDA decisions enhancing therapeutic options for underserved populations, including rare diseases, pediatrics, and cardiometabolic care. Key approvals included Kygevvi for thymidine kinase 2 deficiency, linaclotide for pediatric IBS-C, and plozasiran for familial chylomicronemia syndrome. Safety updates for ELEVIDYS restricted its use to ambulatory Duchenne muscular dystrophy patients. Sibeprenlimab received accelerated approval for IgA nephropathy, marking progress in precision medicine. These decisions reflect the FDA's focus on precision mechanisms and high-need populations across diverse therapeutic areas.

This FDA News Month in Review provides a round-up of regulatory decisions from November 2025.

5 FDA Headlines You Missed in November 2025

The US Food and Drug Administration (FDA) has approved Axogen’s Biologics License Application (BLA) for acellular nerve allograft-arwx (Avance) for surgical implantation.

As described in a December 3, 2025, press release from the FDA, the peripheral nerve scaffold is approved for sensory nerve discontinuities (≤25mm) (breaks in the pathway of sensory nerves) in adults and pediatric patients aged 1 month and older. Under the Accelerated Approval pathway, acellular nerve allograft-arwx is also approved for larger sensory nerve discontinuities (>25mm), motor and mixed nerve discontinuities.

Of note, continued approval for these indications is contingent upon verification and description of clinical benefit in confirmatory studies.

“This approval represents a meaningful shift from our historical classification as a human tissue product and brings the product in line with FDA’s classification of Avance as a biologic. This milestone clarifies and strengthens our regulatory footing and confirms approval for use of Avance as an acceptable therapeutic option for treating peripheral nerve discontinuities in all of Axogen’s present nerve repair use cases,” Michael Dale, Axogen’s Chief Executive Officer, said in a statement.

Unlike some current treatments that require surgeons to remove healthy nerve tissue from another part of the patient's body and create a second surgical site, acellular nerve allograft-arwx is made using nerve tissue from deceased donors that has been specially processed to remove cells while preserving the natural structure that helps nerves regrow.

“Avance eliminates the need for patients to undergo additional surgery to harvest their own nerve tissue for repair,” said Vijay Kumar, MD, Acting Director of the Office of Therapeutic Products, CBER.

 “The agency has exercised regulatory flexibility in expanding the indication for Avance to motor and mixed nerve injuries, and sensory nerves with gaps exceeding 25 mm, through the Accelerated Approval pathway based on a surrogate endpoint that is reasonably likely to predict clinical benefit. The accelerated approval is subject to the requirement that the applicant conduct and submit the results of confirmatory clinical trials.”

The study of acellular nerve allograft-arwx’s efficacy compared implantation of acellular nerve allograft-arwx to collagen nerve cuffs. The study met its primary endpoint for the return of sensory function, showing acellular nerve allograft-arwx’s statistical non-inferiority to comparator nerve cuff.

The most common adverse reactions reported in clinical trials were procedural pain and increased sensitivity to sensory stimuli, such as touch, temperature, and pain. According to the FDA, patients should be monitored for procedural complications including pain, hyperesthesia, infection, implant site swelling, adhesions, hypertrophic scar formation, impaired motor or sensory function, bleeding and neuroma formation.

Of note, acellular nerve allograft-arwx is made from human cadaveric tissue and may carry a risk of transmitting infectious agents. No cases of transmission of viral diseases have ever been identified for acellular nerve allograft-arwx. However, the Agency warns all infections thought to be transmitted by acellular nerve allograft-arwx should be reported to the manufacturer.

“The approved BLA and successful transition of Avance to a biologic regulatory framework should give all stakeholders the assurance that Avance has been rigorously evaluated and determined to be safe, pure, and potent for its intended use, and that its benefits outweigh its known or potential risks,” Dale said in a statement.

US Food and Drug Administration. FDA Approves Nerve Scaffold for the Treatment of Sensory Nerve Discontinuity. December 3, 2025. Accessed December 4, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-approves-nerve-scaffold-treatment-sensory-nerve-discontinuity

Axogen. Axogen Announces FDA Approval of Biologics License Application for AVANCE® (acellular nerve allograft–arwx). December 3, 2025. Accessed December 4, 2025. 

https://ir.axogeninc.com/press-releases/detail/1012/axogen-announces-fda-approval-of-biologics-license

The FDA has approved Axogen's Biologics License Application for Avance, an acellular nerve allograft, for surgical implantation in sensory nerve discontinuities in both adults and pediatric patients. This approval, under the Accelerated Approval pathway, extends to larger sensory, motor, and mixed nerve discontinuities. Continued approval is contingent on confirmatory studies. Avance, derived from cadaveric tissue, eliminates the need for autografts, reducing surgical burden. Clinical trials demonstrated its non-inferiority to collagen nerve cuffs. Adverse reactions include procedural pain and increased sensitivity, with a potential risk of infectious agent transmission.

The accelerated approval expands Avance use to motor, mixed, and long-gap sensory nerve injuries, with required confirmatory trials to verify clinical benefit.

FDA Approves Acellular Nerve Allograft-arwx (Avance) for Sensory Nerve Discontinuity

Lingdolinurad (ABP-671) had acceptable safety and tolerability and reduced serum uric acid (sUA) in people with hyperuricemia or 

Findings from a phase 2a study evaluating ligdolinurad were presented at the 

American College of Rheumatology (ACR) Convergence 2025

, held October 24–29 in Chicago, Illinois, by Ullrich Schwertschlag, MD, PhD, FACP, Atom’s Senior Vice President of Clinical Development.

The study enrolled adult participants up to the age of 55 years without clinically significant kidney abnormalities on renal ultrasound and non-symptomatic hyperuricemia or gout, as evidence by 2 separate fasting sUA results of >7 mg/dL 24 hours apart. Participants were randomized to 5 multiple ascending oral dose groups including 1, 2, 4, 6, 12 mg of ABP-671 and placebo in a 7/2 ratio. The dose escalation dosing period started from 0.2 mg to 1 mg over a 9-day or 10-day period to mitigate any renal sUA overload and was then followed by a 7-day dosing period of ABP-671 or placebo. Investigators enrolled groups separately and conducted them sequentially.

Schwertschlag and colleagues found that treatment-emergent adverse events (TEAEs) were reported in 24 participants (53.3%), including 5 (71.4%), 4 (57.1%), 6 (85.7%), 4 (57.1%), 1 (14.3%), and 4 (40%) in the 1, 2, 4, 6, and 12 mg and placebo groups, respectively. There were no serious AEs, grade >3 TEAEs, or deaths. Notably, investigators did not observe AE incidence to be dose dependent. Participants followed a structured dose-escalation regimen before reaching their assigned target doses. In Group 1, dosing increased from 0.2 mg once daily (QD) on days 1–3 to 0.5 mg QD on days 4–10 and 1 mg QD on days 11–17. Group 2 received 0.2 mg QD on days 1–3, 0.5 mg QD on days 4–6, 1 mg QD on days 7–9, and 2 mg QD on days 10–16. Groups 3, 4, and 5 escalated to target doses of 4 mg, 6 mg, and 12 mg QD, respectively, administered from days 10–16.

The investigators found that sUA levels decreased in a dose dependent manner, with the max median decreases of sUA occurring at 3.00-9.00 hours post dose. Serum uric acid (sUA) levels declined in a clear dose-dependent manner, with the greatest median reductions observed between 3 and 9 hours postdose. Among participants with gout, maximum mean sUA decreases from baseline were 17.7% with placebo, 56.4% with 1 mg, 58.1% with 2 mg, 69.4% with 4 mg, 77.0% with 6 mg, and 79.2% with 12 mg. In participants with hyperuricemia, corresponding reductions were 19.9%, 50.1%, 64.1%, 73.2%, 81.2%, and 82.1%.

At 24 hours postdose, the proportion of participants reaching sUA thresholds <6.0 mg/dL, <5.0 mg/dL, and <4.0 mg/dL increased consistently with dose. For the 1-mg group, rates were 85.7%, 57.1%, and 14.3%, respectively; for 2 mg, 100%, 85.7%, and 14.3%; for 4 mg, 100%, 85.7%, and 14.3%; for 6 mg, 100%, 100%, and 57.1%; and for 12 mg, 100%, 100%, and 100%. No placebo recipients achieved any of these sUA thresholds.

Atom also presented phase 1 safety and pharmacokinetic data on its novel colchicine analogue ABP-745 at the ACR conference in healthy volunteers that demonstrated the drug was well-tolerated and exhibited dose proportional pharmacokinetics. Atom recently also enrolled the first patient in a multicenter Phase 2 acute gout flare trial that will compare ABP-745 with placebo and colchicine.

“The positive findings in both of these studies have contributed to successfully advancing our goal of providing meaningful relief to patients worldwide suffering from the damaging effects of chronic gout and the disease’s painful acute gout flares,” William Dongfang Shi, PhD, Founder and CEO of Atom, said.

 “Both ABP-671 and ABP-745 have the potential to give gout patients more effective and safer alternatives to existing gout therapies, which have insufficient efficacy and are often associated with serious or even life-threatening adverse effects.”

Schwertschlag U, Yang Y, Li J, et al. A Phase 2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of ABP-671 in Subjects with Hyperuricemia or Gout in China. Presented at: ACR Convergence 2025; October 24-29; Chicago, Illinois. Abstract #2012

Atom Therapeutics to Present Positive Data from Clinical Trials of Its Lead Novel Oral Drugs for Chronic Gout and Acute Gout Flares at ACR Convergence 2025. News release. Atom Therapeutics. October 21, 2025. https://aijourn.com/atom-therapeutics-to-present-positive-data-from-clinical-trials-of-its-lead-novel-oral-drugs-for-chronic-gout-and-acute-gout-flares-at-acr-convergence-2025/

Lingdolinurad (ABP-671) demonstrated acceptable safety and tolerability while effectively reducing serum uric acid (sUA) levels in individuals with hyperuricemia or gout. A phase 2a study presented at the ACR Convergence 2025 showed dose-dependent sUA reductions, with the highest decreases observed between 3 and 9 hours post-dose. The study involved multiple ascending oral dose groups, and no serious adverse events were reported. Additionally, Atom presented phase 1 data on ABP-745, a colchicine analogue, indicating good tolerability and dose-proportional pharmacokinetics, with ongoing trials for acute gout flares.

A new study reveals ligdolinurad effectively lowers uric acid levels in gout patients, showcasing promising safety and tolerability results.

Lingdolinurad Demonstrates Dose-Dependent Serum Uric Acid Reductions

November delivered a wave of advances across asthma, COPD, and related inflammatory airway diseases, reflecting the accelerating shift toward precision medicine in respiratory care. New data showed pulmonary rehabilitation’s unexpected anti-inflammatory impact in asthma—particularly a 40% FeNO reduction among patients who began PR with high levels—while an open-label extension of tezepelumab demonstrated substantial steroid-sparing benefits, with nearly all OCS-dependent patients tapering to 5 mg or less and half discontinuing steroids entirely. The month also brought promising phase 2b results for rezpegaldesleukin, an IL-2 receptor pathway agonist that improved both atopic dermatitis and asthma control, underscoring its potential as a dual-benefit therapy for patients with overlapping disease.

Alongside these trial updates, expert discussions at CHEST 2025 and a recent 

 clinical forum highlighted the practical realities of implementing targeted therapies for asthma and COPD. Nicola Hanania, MD, MS, emphasized the growing importance of biomarkers such as blood eosinophils and FeNO in navigating an increasingly crowded biologic landscape, while Meilan Han, MD, and colleagues examined how real-world factors—including payer barriers, clinician familiarity, and multidisciplinary coordination—are shaping biologic adoption in COPD.

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

1. Pulmonary Rehabilitation Reduces Type 2 Inflammation in High Inflammation Asthma

Pulmonary rehabilitation (PR) was shown to meaningfully reduce airway inflammation in people with asthma who began treatment with elevated FeNO, with the high-FeNO group experiencing a 40% drop in levels during the 3-week program. Improvements in asthma control and exercise capacity occurred across all baseline FeNO categories, but the anti-inflammatory effect was exclusive to those with high initial FeNO.

2. Tezepelumab Drastically Reduces OCS Use for Asthma in Open-Label Trial

Nearly all patients with severe, OCS-dependent asthma in the WAYFINDER trial were able to lower their daily steroid dose to 5 mg or less after 52 weeks of open-label tezepelumab, and half fully discontinued OCS while maintaining asthma control. These improvements were consistent across inflammatory subgroups, though the absence of a control arm makes the magnitude of benefit harder to interpret.

3. Rezpegaldesleukin Shows Strong Phase 2b Results for Atopic Dermatitis, Asthma

Rezpegaldesleukin, an IL-2 receptor pathway agonist, improved both atopic dermatitis severity and asthma control in the phase 2b REZOLVE-AD trial, with the highest dose (24 μg/kg every 2 weeks) delivering the strongest skin responses. Patients with comorbid asthma also saw meaningful improvements, including clinically significant ACQ-5 reductions in 75% of those with uncontrolled asthma at baseline.

4. The Asthma and COPD Precision Medicine Era, with Nicola A. Hanania, MD, MS

Nicola A. Hanania, MD, MS, professor of medicine and director of the Asthma Clinical Research Center at Baylor College of Medicine, discussed some of the sessions and data presentations from CHEST 2025 that speak to these latest innovations in asthma and COPD. He highlighted the expanding role of targeted therapies, the difficulties clinicians face in selecting among six asthma biologics, and the growing importance of biomarkers such as blood eosinophils and FeNO in guiding therapy.

5. Personalized COPD Treatment: Lessons from Biologic Trials and Clinical Practice

 clinical forum led by Meilan Han, MD, clinicians reviewed new biologic data alongside the evolving framework for identifying the right biologic candidates through biomarkers such as blood eosinophil counts. Faculty also discussed real-world considerations shaping the adoption of biologics, noting differing comfort levels among clinicians, the need for payer navigation, and the importance of multidisciplinary collaboration to integrate these therapies into routine COPD care.

November saw significant advancements in asthma, COPD, and related inflammatory airway diseases, emphasizing the shift toward precision medicine. Pulmonary rehabilitation showed a notable anti-inflammatory effect in asthma patients with high FeNO levels. Tezepelumab demonstrated substantial steroid-sparing benefits, allowing many patients to reduce or discontinue OCS use. Rezpegaldesleukin showed promise in improving both atopic dermatitis and asthma control. Expert discussions highlighted the importance of biomarkers and real-world factors in adopting targeted therapies for asthma and COPD, underscoring the complexities of integrating biologics into clinical practice.

November 2025 saw new data on asthma therapies and new insights from experts on continuing trends in the field.