Incyte is looking to expand the use of its PD-1 blocker Zynyz to treat non-small cell lung cancer. The FDA’s rejection did not flag problems with the drug’s efficacy or safety.

Original title: “Incyte’s Lung Cancer Expansion Bid Thwarted by Issues at Novo’s Catalent-Acquired Site”

Phase 3 Mascot study reaches key milestone for potential first-in-class therapy

Original title: “Lundbeck completes patient randomisation early in global MSA trial”

EC decision introduces first systemic option for adults with advanced SCAC

Original title: “Incyte treatment for advanced anal cancer approved in Europe”

In February 2026, the U.S. FDA ... therapies for ultra-rare diseases, particularly genome editing and RNA-based treatments. The framework supports small-sample clinical evidence generation by focusing on demonstrating biological mechanisms, target validation, and real-world biomarker or clinical outcome improvements when randomized controlled trials are not ... In February 2026, the U.S. FDA issued draft guidance in 2026 introducing the “Plausible Mechanism Framework” to accelerate development and regulatory approval of individualized therapies for ultra-rare diseases, particularly genome editing and RNA-based treatments. The framework supports small-sample clinical evidence generation by focusing on demonstrating biological mechanisms, target validation, and real-world biomarker or clinical outcome improvements when randomized controlled trials are not feasible. Rare disease clinical trials are specialized research studies designed to evaluate the safety, efficacy, and therapeutic potential of new treatments for rare diseases affecting small patient populations. Growth is driven by increasing prevalence of rare diseases, advances in genomic diagnostics, rising investment in gene and cell therapies, orphan drug incentives, and expanding precision medicine research. Phase I trials account for 35.1% of the global rare disease clinical trials market because they represent the primary entry point for a large number of novel investigational therapies, particularly in gene therapy, biologics, and precision medicine-based treatments. China has registered around 780,000 rare disease cases in its national diagnosis and treatment system, reflecting the large patient base and significant unmet medical need that is encouraging clinical trial activity across multiple rare disease indications. The country’s growing focus on innovative biologics, gene therapies, and precision medicine is further accelerating rare disease drug development programs and early-stage clinical research initiatives.

Original title: “Rare Disease Clinical Trials Market Forecast and Trends 2033”

The regimen’s clearance in early multiple myeloma was the third approval under the agency’s controversial “national priority” voucher program and issued only 55 days after the review began.

Original title: “FDA issues speedy approval to J&J’s Tecvayli-Darzalex combo”

Zealand Pharma’s shares fell 32% in early morning trading Friday after its Roche-partnered asset petrelintide failed to meet investor expectations in a mid-stage clinical trial.

Original title: “Roche, Zealand’s Amylin Weight Loss Drug Fails To Clear Lilly’s Bar”

The FDA granted Johnson & Johnson a Commissioner's National Priority Voucher in December 2025 after the combination of Darzalex and Tecvayli improved progression-free survival by 83%. The new approval is for second-line treatment of the blood cancer.

Original title: “J&J Wins Third National Priority Approval for Multiple Myeloma Combo”

Catch up on any of the neurology headlines you may have missed in February 2026, compiled into 1 place by the NeurologyLive® team. The agency’s CRL outlined several reasons the gene therapy product was not approved, including concerns over whether the clinical trial eligibility criteria could adequately distinguish between neuronopathic and attenuated disease. The European Commission (EC) has approved the monoclonal antibody (mAb) inebilizumab (Uplizna; Amgen), on February 12, 2026, as an add-on to standard therapy in the treatment of adults with generalized myasthenia gravis (gMG) in adults who are antiacetylcholine receptor (AChR) and antimuscle specific tyrosine kinase (MuSK) antibody positive.5 · The EC’s decision was based on data from the randomized, double-blind, placebo-controlled phase 3 MINT clinical trial (NCT04524273). The coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds. Click the read more buttons for more details and information about each update. At the start of the month, on February 9, 2026, the FDA issued a CRL to Regenxbio’s biologics license application (BLA) for clemidsogene lanparvovec (RGX-121), an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat mucopolysaccharidosis type 2 (MPS II), also known as Hunter syndrome.1 The FDA was busy in February 2026, making a number of decisions on potential new therapeutic agents, including issuing a complete response letter (CRL), granting an expanded approval indication, accepting a new drug application (NDA), and granting a priority review. With all the treatments that have progressed through the pipeline of clinical development, the NeurologyLive® team has been hard at work covering all the agency movements to make sure you are up to date on the latest news in neurology.

Original title: “FDA Action Update, February 2026: Complete Response Letter, Expanded Indication, and Acceptance | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

The U.S. Food and Drug Administration today approved teclistamab in combination with daratumumab hyaluronidase-fihj, Tec-Dara, to treat adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy.

Original title: “FDA Grants Third Approval Under the National Priority Voucher Program”

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine.

Original title: “UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms”

The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took issue with were filed in mid-2024.

Original title: “PepGen’s Mid-Stage Myotonic Dystrophy Study Hit With ‘Surprise’ Pause”

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and well tolerated by patients with Dravet syndrome for whom antiseizure medications are not effective. The most common TEAE in the Phase 1/2a trials was post-lumbar puncture syndrome (nearly 25%), while the most common event in the extension studies was cerebrospinal fluid (CSF) protein increase (45%). However, none of the patients with CSF protein increase had increased intracranial pressure or hydrocephalus. Of the serious TEAE, only one was considered treatment-related. "Our data support zorevunersen safety and tolerability, as well as improvement in overall clinical status, quality of life, and adaptive behavior following continued dosing in the extension studies," said Dr. Owen – a Lurie Children's patient who participated in the clinical trial and now continues in the open-label extension study – is a 12-year-old boy with Dravet syndrome, whose seizures were not controlled by medications. He also had intellectual disability and gait abnormalities. Brain microphysiological systems are reshaping in vitro neurotoxicity testing through functional validation and advanced disease modeling. Results, published in the New England Journal of Medicine, include significant seizure reduction and improvement in other symptoms of Dravet syndrome, such as language, motor, and behavior issues. Researchers also report sustained treatment benefits in ongoing open-label extension studies.

Original title: “Novel gene regulation therapy passes key clinical milestone”

The company also reported that ... the gene therapy product. Neurogene plans to have enrolled 8 patients in the low-dose cohort by the end of the year and expects to report updated data in the second half of next year. In the first half of 2025, the company also anticipates that it will release more information on its plan for a registrational clinical trial for NGN-401. ... NGN-401 had been selected as part of the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics ... The company also reported that it has reached an accord with the FDA regarding plans for scaling up manufacturing and its potency assay strategy for the gene therapy product. Neurogene plans to have enrolled 8 patients in the low-dose cohort by the end of the year and expects to report updated data in the second half of next year. In the first half of 2025, the company also anticipates that it will release more information on its plan for a registrational clinical trial for NGN-401. ... NGN-401 had been selected as part of the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.2 The program will provide sponsors with the opportunity for frequent advice and regular communication as needed with regard to clinical trial design and other development issues particular to the investigational product being evaluated. The company noted that most treatment-related AEs that occurred in this group have been responsive to steroids and resolved or are resolving. Notably, an SAE was reported in a third patient who was more recently treated in the high-dose cohort. Neurogene pointed out that the SAE is consistent with the known risks of AAV vector-based gene therapy. Alongside the announcement of the interim data, Neurogene stated that a third cohort in the trial, which will treat up to 3 patients aged 16 years or older at the high dose, has been initiated. Notably, Neurogene also stated that it has made the decision to discontinue the development of NGN-101, its investigational AAV vector-based gene therapy intended to treat neuronal ceroid lipofuscinosis (Batten) Subtype 5 (CLN5) disease, which is currently being assessed in · a phase 1/2 clinical trial (NCT05228145).1 Neurogene cited the FDA’s denial of its application for regenerative medicine advance therapy designation for NGN-101 as the reason for the decision, as the company considered this designation necessary for continued investment in the product’s development. Neurogene’s NGN-401, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Rett syndrome, has generated positive efficacy results in the low-dose cohort of aphase 1/2 pediatric clinical trial (NCT05898620), but an emerging treatment-related serious adverse event (SAE) was reported in a patient treated in the study’s high-dose cohort.1

Original title: “Neurogene’s Rett Syndrome Gene Therapy NGN-401 Demonstrates Efficacy in Low-Dose Cohort, But Serious Adverse Event in High-Dose Group Raises Concerns | CGTlive®”

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial results. The drug, called zorevunersen, holds promise, especially for people with the condition who don’t respond to existing treatments such as antiseizure medications. New gene therapy results bring hope for treating Dravet syndrome, a rare and often fatal seizure condition Over the course of 20 months, patients had between 59 percent and 91 percent fewer seizures than before they started the treatment. Most side effects were moderate or mild. The studies were designed to test the safety and tolerability of the medication at varying dosages. Another study, a phase 3 randomized control trial, is underway, and it will more rigorously test how well the medication treats Dravet syndrome’s core symptoms. But the data from the studies published today suggest that zorevunersen is treating the underlying cause of the disease. Doctors currently prescribe antiseizure drugs and therapeutic diets to manage the seizures that characterize the condition. But these treatments are often ineffective. “It’s very rare that [a patient becomes] seizure-free,” says senior author Helen Cross of University College London. “These results are incredibly promising, and the levels of improvements are unprecedented in this disease state,” says Veronica Hood, chief science officer of the Dravet Syndrome Foundation. “This level of improvement is meaningful in so many aspects of daily life.” · Eight-year-old Freddie Truelove of Huddersfield, England, has Dravet syndrome and has taken part in a trial of zorevunersen, an ASO gene therapy to treat the condition.

Original title: “Pioneering gene therapy may treat a deadly seizure disorder | Scientific American”

First‑in‑human phase 1/2a study launches in China and the US targeting advanced cancers

Original title: “HUTCHMED begins global trial of novel attc therapy hmpl‑a580 in solid tumours”

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.

Original title: “UniQure’s Delay, REGENXBIO’s Rejection Explained, Sarepta’s Ingram Steps Down, More”

The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease H | The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease Hunter syndrome, providing further details ... The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease H | The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease Hunter syndrome, providing further details into the agency’s issues with the biotech’s trial design. The Jefferies analysts caveated their note by pointing out that Denali’s submission is being evaluated by the Center for Drug Evaluation and Research or CDER, while Regenxbio’s gene therapy was reviewed by the Center for Biologics Evaluation and Research (CBER). CBER Director Vinay Prasad, M.D., has garnered a reputation for demanding randomized, double-blind, placebo-controlled trials that measure clinical outcomes over the use of biomarkers or single-arm studies that are popular—and sometimes necessary—in rare diseases. The agency's rationale should have little impact on Denali Therapeutics, which is awaiting a decision on its own Hunter syndrome candidate. Regenxbio and others developing new Hunter syndrome treatments, like Denali Therapeutics, are using the sugar as a biomarker in place of clinical outcomes. The target heparan sulfate level of 100 nanograms per milliliter set by Regenxbio, though, is “compromised by lack of comparability” between the 13 trial patients and the patients whose data forms the natural history control arm, the FDA said, especially in age and level of cognitive impairment.

Original title: “FDA details rationale for rejecting rare disease gene therapy from Regenxbio”

The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease H | The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease Hunter syndrome, providing further details ... The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease H | The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease Hunter syndrome, providing further details into the agency’s issues with the biotech’s trial design. The Jefferies analysts caveated their note by pointing out that Denali’s submission is being evaluated by the Center for Drug Evaluation and Research or CDER, while Regenxbio’s gene therapy was reviewed by the Center for Biologics Evaluation and Research (CBER). CBER Director Vinay Prasad, M.D., has garnered a reputation for demanding randomized, double-blind, placebo-controlled trials that measure clinical outcomes over the use of biomarkers or single-arm studies that are popular—and sometimes necessary—in rare diseases. The agency's rationale should have little impact on Denali Therapeutics, which is awaiting a decision on its own Hunter syndrome candidate. Regenxbio and others developing new Hunter syndrome treatments, like Denali Therapeutics, are using the sugar as a biomarker in place of clinical outcomes. The target heparan sulfate level of 100 nanograms per milliliter set by Regenxbio, though, is “compromised by lack of comparability” between the 13 trial patients and the patients whose data forms the natural history control arm, the FDA said, especially in age and level of cognitive impairment.

Original title: “FDA details rationale for rejecting rare disease gene therapy from Regenxbio”

A month after Amgen gave up on rocatinlimab — which was once seen as a threat to Dupixent — new findings have linked the drug’s mechanism to the onset of a type of skin cancer.

Original title: “Kyowa Kirin abandons touted eczema drug following safety review”