The FDA was busy in July 2025, making a number of decisions on potential new therapeutic agents including an approval, a clearance, a label update, a priority review, a complete response letter, and suspension of a distribution.
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. To give you a chance to catch up on any of the headlines you may have missed over the course of the last month, we’ve compiled all the updates into one place. The coverage includes the latest FDA approvals, new designations, submissions and resubmissions, and clinical trial initiations and holds.
Click the read more buttons for more details and information about each update.
FDA Grants Priority Review, Sets PDUFA Date for Hunter Syndrome Treatment Tividenofusp Alfa
Early in the month, on July 7, the FDA accepted Denali Therapeutics’ biologics license application (BLA) for tividenofusp alfa, an investigational agent, as a potential treatment for Hunter syndrome, a rare genetic lysosomal disease. Along with granting priority acceptance review, the agency assigned a PDUFA date of January 5, 2026, for the iduronate-2-sulfatase (IDS)-targeting therapy.1
The therapy’s submission, coming through the accelerated approval pathway, was based on data from a phase 1/2, open-label, single-arm study of 47 patients with the disease that lasted 24 weeks. In the study, treatment with the investigational agent led to reductions to normal and near-normal levels in central nervous system (CNS) and peripheral biomarkers of the disease after 24 weeks, as well as noted changes in cerebrospinal fluid heparan sulfate (CSF HS) and neurofilament light, biomarkers of neuroaxonal damage.2
“We are grateful to the FDA for their recognition of the urgent need for new therapies that could offer a significant improvement in the treatment of Hunter syndrome, as reflected by their priority review designation for our Biologics License Application for tividenofusp alfa,” Carole Ho, MD, chief medical officer and head of development at Denali, said in a statement.1 “If FDA-approved, tividenofusp alfa would mark the first significant advancement in nearly two decades for enzyme replacement therapy for individuals living with Hunter syndrome because of its potential for delivery to tissues throughout the brain and the body. This is also a pivotal milestone for our TransportVehicle platform, which continues to progress with the aim of treating a wide range of lysosomal storage diseases and neurodegenerative disorders.”
Exablate Neuro Gains FDA Approval for Staged Bilateral Focused Ultrasound in Advanced Parkinson Disease
A day later, on July 8, the FDA approved the use of Insightec’s Exablate Neuro platform for staged bilateral pallidothalamic tractotomy in patients with advanced Parkinson disease (PD), covering both the initial and subsequent procedures to enable comprehensive bilateral treatment. This expanded indication offers a therapeutic option for patients with severe motor symptoms of PD who have not experienced relief from previous therapies.3
Exablate Neuro uses MRI-guided focused ultrasound to precisely target areas in the pallidothalamic tract through an incisionless procedure that requires no implanted hardware, offering a lower risk profile than traditional surgical interventions. Insightec noted that it is planning a limited launch of the bilateral procedure at select centers in 2025, alongside ongoing efforts to establish routine reimbursement pathways and expand patient access.
“This milestone reflects our commitment to expanding access to safe, effective, and incisionless treatment options for people living with Parkinson’s disease,” Maurice R. Ferré, MD, CEO and chairman at Insightec, said in a statement.3 “With this new indication, we’re advancing care for movement disorders and opening the door to life-changing treatment for those who need it now and in the years ahead.”
FDA Approves Updated Label for Alzheimer Therapy Donanemab to Lower ARIA-E Risk
A day later, on July 9, the FDA approved an updated label for donanemab (Kisulna; Eli Lilly), an FDA-approved therapy for early-stage Alzheimer disease (AD), to now include a new recommended titration dosing schedule, which may lead to lowered risk of amyloid-related imaging abnormalities (ARIA).4
Donanemab, an amyloid-targeting treatment, was approved in 2024 for patients with mild dementia stage of early AD with confirmed amyloid pathology. At the time, the recommended dosage of the therapy was 700 mg administered intravenously over approximately 30 minutes every 4 weeks for the first 3 doses, followed by 1400 mg every 4 weeks. In the latest updated labeling, the new recommended dosing regimen involves a more gradual titration, while still preserving the drug’s effect on amyloid plaque removal and phosphorylated tau 217 (p-tau) reduction.
“We are confident that this label update for Kisunla will significantly aid healthcare professionals in evaluating appropriate treatment options for their patients,” Brandy Matthews, MD, FAAN, vice president of Global & US Medical Affairs for Alzheimer’s Disease at Eli Lilly, said in a statement.4 “This update underscores our unwavering commitment to patient safety and the advancement of Alzheimer’s disease treatment by potentially mitigating the risk of ARIA-E.”
FDA Hands Capricor Complete Response Letter for Deramiocel as DMD Cardiomyopathy Treatment
A couple of days later, on July 11, the FDA gave Capricor Therapeutics a complete response letter (CRL) for its investigational cell-based candidate deramiocel as a potential treatment for cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The agency has reached out to Capricor to set up a Type A meeting to discuss the best path forward for the cell therapy.5
In the CRL, the FDA claimed it was unable to review the biologics license application (BLA) in its current form, citing that it did not meet requirement for substantial evidence of effectiveness. In addition, the agency noted outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application, most of which Capricor claimed it believed to have addressed in prior communications to the FDA.
The submission for deramiocel was based on data from the HOPE-2 trial (NCT), its open-label extension, and natural history comparisons from FDA-funded datasets. Following the CRL, Capricor plans to resubmit with additional data from the ongoing phase 3 HOPE-3 trial to provide more evidence of effectiveness.
“We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD,” Linda Marban, PhD, chief executive officer at Capricor, said in a statement regarding HOPE-3.5 “While this was an unexpected decision by the FDA, we remain committed to the DMD community to get Deramiocel through the approval process.”
FDA Shuts Down Sarepta’s Distribution of Gene Therapy Elevidys Following Patient Deaths
About a week later, on July 18, the FDA requested Sarepta Therapeutics’ to suspend the distribution of its approved gene therapy Elevidys for DMD as well as pause clinical trials testing the company’s gene therapy agents, following the reported deaths of 3 patients.6
The latest death, a 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3, was enrolled in the company’s phase 1 study testing SRP-9004, also known as patidistrogene bexoparvovec. According to several reports, this incidence occurred last month and was caused by liver failure, the same reason for the two previously reported deaths.7,8
According to the announcement, the FDA revoked Sarepta’s platform technology designation and met with the company to request a voluntary halt on all Elevidys shipments. Sarepta declined to comply with the agency’s request. The reasons for revoking were because of insufficient evidence—especially in light of new safety concerns—that the company’s AAVrh74 Platform Technology can be used across multiple drugs without compromising safety.
“Today, we’ve shown that this FDA takes swift action when patient safety is at risk,” FDA commissioner Marty Makary, MD, MPH, said in a statement.6 “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”
FDA Clears HyHub and HyHub Duo Devices for Easier Administration of Immune Globulin Infusion, Hyqvia
A week later, on July 21, the FDA granted 510(k) clearance to Takeda’s HyHub and HyHub Duo devices for patients aged 17 years and older which allow immune globulin (IG) infusion 10% (human) with recombinant human hyaluronidase (Hyqvia), an approved treatment for chronic inflammatory demyelinating polyneuropathy (CIDP), to be transferred from vials needle-free in home or clinical settings. The company expects the devices to available in the United States starting in the second half of fiscal year 2025.9
HyHub and HyHub Duo serve as docking stations for the dual vial units (DVUs) used in Hyqvia administration, which consists of 1 vial of immunoglobulin (IG) and 1 vial of hyaluronidase. These devices can reduce the number of preparation steps by up to half compared with using a pooling bag, depending on the device and number of DVUs used. In addition, HyHub and HyHub Duo help minimize the ancillary supplies needed for infusion preparation. Takeda noted that a dedicated carrier bag is also available to support room-to-room mobility.
“This milestone exemplifies our dedication to advancing innovative solutions that can enhance the treatment administration experience for people who rely on infusions of facilitated immunoglobulin like HYQVIA,” Kristina Allikmets, MD, PhD, senior vice president and head of Research & Development for the Plasma-Derived Therapies Business Unit at Takeda, said in a statement.9 “We designed HyHub and HyHub Duo, Takeda’s first customized devices for use with a plasma-derived therapy, with input from patients and caregivers, demonstrating our focus on leveraging technology and deep insights to offer a patient-centric ecosystem of support throughout the treatment journey.”
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