Orsini Named Exclusive Specialty Pharmacy Provider for MIPLYFFA™ (arimoclomol)

Orsini has been chosen as the exclusive specialty pharmacy partner for MIPLYFFA™ (arimoclomol) by Zevra Therapeutics, Inc. This marks a significant milestone as it is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), a rare and progressive neurodegenerative disorder. MIPLYFFA is to be used in combination with miglustat and is approved for treating neurological manifestations of NPC in both adult and pediatric patients aged two years and above. This partnership follows the earlier collaboration between Orsini and Zevra for OLPRUVA® (sodium phenylbutyrate).

Darin DeCarlo, Orsini's Chief Growth Officer, expressed enthusiasm about extending their relationship with Zevra and emphasized the importance of making this innovative therapy accessible to those affected by NPC.

Niemann-Pick disease type C is a rare lysosomal storage disorder characterized by the body's inability to transport cholesterol and other lipids within cells, resulting in their accumulation. This accumulation predominantly affects neurons and leads to a range of physical and cognitive impairments, including issues with speech, cognition, swallowing, ambulation, and fine motor skills. The disorder is caused by mutations in the NPC1 or NPC2 genes and can affect both children and adults. Diagnosing NPC can be complex and time-consuming, and the disease often progresses irreversibly, leading to early mortality.

MIPLYFFA works by increasing the activation of transcription factors EB (TFEB) and E3 (TFE3), which in turn upregulates genes involved in lysosomal function. It has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts, although the clinical significance of this finding remains unclear. The FDA has granted MIPLYFFA several designations, including Breakthrough Therapy, Rare Pediatric Disease, Orphan Drug, and Fast Track, highlighting its potential importance in treating NPC. Additionally, the European Medicines Agency (EMA) has recognized MIPLYFFA as an Orphan Medicinal Product for NPC treatment.

Orsini, established in 1987, has a long-standing reputation for providing comprehensive and compassionate care, particularly in the realm of rare diseases and gene therapies. The company collaborates closely with biopharma innovators, healthcare providers, and payors to make advanced treatments accessible to patients and their families. Orsini’s integrated rare disease pharmacy solutions encompass pharmacy distribution, patient services, clinical management, and convenient home infusion services, designed to simplify patient access to cutting-edge therapies. Their care model ensures personalized attention through experienced therapy care teams, dedicated to leaving no patient behind.

Orsini Specialty Pharmacy is accredited by several prestigious organizations, including the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC, and NABP. The pharmacy has also been recognized with URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs.

The collaboration between Orsini and Zevra Therapeutics for MIPLYFFA marks a promising advancement for patients suffering from Niemann-Pick disease type C, offering hope for improved management of this challenging and life-threatening condition.