New Hope at Home: Orsini to Exclusively Deliver Breakthrough Hunter Syndrome Drug

ELK GROVE VILLAGE, Ill. – April 20, 2026 – A new era in the treatment of a devastating pediatric rare disease has begun with the announcement of an exclusive partnership that promises to bring a breakthrough therapy directly into patients' homes. Denali Therapeutics has selected Orsini, a leader in rare disease pharmacy solutions, as the sole specialty pharmacy and home infusion provider for its newly FDA-approved drug, AVLAYAH™ (tividenofusp alfa-eknm).

This strategic alliance marks a pivotal moment for children with Hunter syndrome (mucopolysaccharidosis type II, or MPS II), a progressive and life-limiting genetic disorder. AVLAYAH™ is the first therapy engineered to cross the blood-brain barrier to treat the severe neurological decline associated with the disease. By pairing this innovative drug with Orsini's comprehensive home infusion services, the partnership aims to revolutionize care, reducing the immense burden on families and improving quality of life for young patients.

A Scientific Leap Across the Blood-Brain Barrier

Hunter syndrome is caused by a missing or malfunctioning enzyme, iduronate 2-sulfatase (IDS). Without it, complex sugar molecules called glycosaminoglycans (GAGs) build up in cells throughout the body, leading to widespread damage affecting organs, physical abilities, and, crucially, cognitive development. While conventional enzyme replacement therapies (ERTs) have been available for nearly two decades to address the body's somatic symptoms, they cannot penetrate the brain, leaving the progressive neurological damage unchecked.

AVLAYAH™ represents a significant scientific advancement designed to overcome this fundamental challenge. Utilizing Denali's proprietary Transport Vehicle (TV) technology, the therapy fuses the necessary IDS enzyme to an antibody that acts like a Trojan horse, binding to receptors that shuttle it across the formidable blood-brain barrier. This allows the therapy to reach the central nervous system and address the root cause of neurological decline.

The FDA granted AVLAYAH™ an accelerated approval on March 25, 2026, a decision based on compelling biomarker data from a Phase 1/2 clinical trial. The study showed a remarkable average reduction of 91% in a key disease biomarker, cerebrospinal fluid heparan sulfate (CSF HS), by the 24-week mark. Furthermore, 93% of pediatric patients treated with the drug achieved CSF HS levels within the normal range. While continued approval is contingent on a confirmatory trial, these results offer profound hope for halting or slowing the neurological devastation of Hunter syndrome.

Due to the risk of severe hypersensitivity reactions, the drug includes a Boxed Warning. Treatment must be initiated in a monitored healthcare setting, but the new partnership ensures that subsequent weekly intravenous infusions can transition to the patient's home under the supervision of trained professionals.

Redefining Care with At-Home Infusion

For families managing a rare pediatric disease, the logistical and emotional challenges are immense. Frequent, time-consuming trips to a hospital or infusion center for multi-hour treatments add a significant strain to daily life. The partnership between Denali and Orsini directly addresses this issue by making complex care more accessible and patient-centric.

Orsini, with its extensive experience in rare diseases, will manage the distribution and administration of AVLAYAH™ through its accredited home infusion network. This 'high-touch' model provides not just the drug, but a complete support system. Therapy care teams, composed of experienced nurses and patient support staff, will coordinate with physicians, handle insurance and reimbursement complexities, and provide personalized clinical management for each child.

Bringing these intricate infusions into the home can transform the patient experience. It allows children to receive their life-sustaining treatment in a familiar, comfortable environment, surrounded by family, minimizing disruption to school and childhood activities. This approach aligns with a broader trend in healthcare toward decentralized, patient-focused care models, particularly for chronic and complex conditions where quality of life is a paramount concern.

“Orsini has been serving MPS patients for over 16 years, and we're proud to add AVLAYAH to our portfolio,” said Darin DeCarlo, Orsini's Chief Commercial Officer, in the original announcement. “We're thrilled that this partnership with Denali allows us to extend our ability to serve this rare disease community and continue to offer it best-in-class patient care.”

The Strategic Imperative of Specialty Pharmacy

The exclusive nature of this partnership highlights the increasingly critical role of specialty pharmacies in the commercialization of advanced therapeutics. For a disease as rare and complex as Hunter syndrome, a mass-market distribution model is ineffective. A specialized partner like Orsini provides the necessary infrastructure, expertise, and patient relationships to ensure a successful and safe therapy launch.

This alliance allows for a streamlined and controlled channel from manufacturing to administration. Orsini's established Neurology Center of Excellence—of which AVLAYAH™ is the twentieth ERT—and its multiple accreditations from bodies like The Joint Commission and URAC provide biopharma innovators like Denali with confidence in quality and compliance. For payors and providers, it creates a single, accountable point of contact for a high-cost, high-complexity therapy.

By handling everything from patient services to clinical management and home infusion, Orsini simplifies the process for families navigating a difficult diagnosis. This integrated approach embodies the company's motto, “No Patient is Left Behind™,” ensuring that access to a potentially life-altering treatment like AVLAYAH™ is as seamless as possible for those who need it most. As more gene therapies and complex biologics enter the market, these strategic alliances will become even more essential to bridging the gap between scientific innovation and patient access.