#USA #Elk Grove Village #Hunter Syndrome #Denali Therapeutics #AVLAYAH

Orsini and Denali Therapeutics Join Forces for AVLAYAH™

In a significant development within the rare disease healthcare sector, Orsini has been designated as the exclusive specialty pharmacy and home infusion partner for Denali Therapeutics' new therapy, AVLAYAH™ (tividenofusp alfa-eknm). This groundbreaking therapy, approved by the FDA, serves as an intravenous enzyme replacement treatment tailored specifically for the pediatric management of Hunter syndrome, also known scientifically as mucopolysaccharidosis type II (MPS II).

Understanding Hunter Syndrome

Hunter syndrome is a rare, genetic metabolic disorder caused by inadequate production of enzymes necessary to decompose complex sugars into simpler forms. In affected individuals, glycosaminoglycans (GAGs)—sugar molecules that accumulate in the body—begin to build up in cells, blood, and connective tissues, leading to progressive cellular damage. As the disease advances, it impairs physical abilities, organ functioning, and often cognitive development. Such complexities highlight the critical need for effective therapies like AVLAYAH that target these harmful accumulations.

AVLAYAH uniquely penetrates the brain, making it particularly useful in addressing neurologic manifestations associated with Hunter syndrome. It is also worth mentioning that this therapy is now the twentieth enzyme replacement option and the fifth medication targeting MPS to be incorporated into Orsini's renowned Neurology Center of Excellence.

A Commitment to Patient Care

Orsini's commitment to advancing care in rare diseases spans more than 16 years. Darin DeCarlo, the Chief Commercial Officer at Orsini, expressed enthusiasm about the partnership, stating, “We're thrilled that this collaboration with Denali allows us to extend our ability to serve this rare disease community and continue to offer it best-in-class patient care.” Through this partnership, Orsini aims to not only facilitate access to AVLAYAH but also provide extensive home infusion support, ensuring that patients receive their necessary treatments in a comfortable, convenient setting under the guidance of healthcare professionals.

The Role of Innovative Pharmacy Solutions

Founded in 1987, Orsini has established itself as a leader within the rare disease and gene therapy landscape. Working in close collaboration with biopharmaceutical innovators, healthcare providers, and insurance payors, Orsini strives to simplify the path patients take to access transformative therapies for rare and complex conditions.

Through an integrated approach that combines pharmacy distribution, patient services, clinical management, and home infusion services, Orsini enhances the experience of patients and families in need. The organization's high-touch care model, supported by experienced therapy care teams, exemplifies its mission to ensure that “No Patient is Left Behind™.”

Furthermore, Orsini has received numerous accreditations, including recognition from the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC, and NABP. Notably, it holds URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC’s Distinction in Rare Diseases and Orphan Drugs, cementing its reputation in the field.

With the addition of AVLAYAH, Orsini not only broadens its service portfolio but also reinforces its dedication to those affected by rare diseases, promising them innovative, compassionate, and accessible care. For further details regarding Orsini and their offerings, visit www.orsini.com.