Orsini is proud to announce its partnership with Zevra Therapeutics, Inc. (Zevra) as the exclusive specialty pharmacy provider for MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC), a rare and progressive neurodegenerative disease. MIPLYFFA is indicated for use in combination with miglustat to treat neurological manifestations of NPC in adult and pediatric patients aged 2 and above.
Orsini's Continued Partnership with Zevra
Orsini is thrilled to extend its collaboration with Zevra by offering this groundbreaking therapy to individuals with NPC, says Darin DeCarlo, Orsini's Chief Growth Officer.
Understanding Niemann-Pick Disease Type C
Niemann-Pick disease type C (NPC) is an extremely rare, progressive lysosomal storage disorder that affects the body's ability to transport cholesterol and lipids within cells, resulting in their accumulation in various cell types, including neurons. The disease stems from mutations in the NPC1 or NPC2 genes, responsible for producing lysosomal proteins. NPC can impact both children and adults, leading to physical and cognitive impairments, with neurological deficits affecting speech, cognition, swallowing, mobility, and fine motor skills. Diagnosis can be challenging and delayed, with irreversible disease progression often leading to premature death.
Insights into MIPLYFFA
MIPLYFFA (arimoclomol) enhances the activation of transcription factors EB (TFEB) and E3 (TFE3), promoting the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. It has demonstrated reduced unesterified cholesterol levels in human NPC fibroblasts' lysosomes, although the clinical significance remains unclear. FDA has granted MIPLYFFA Breakthrough Therapy, Rare Pediatric Disease, Orphan Drug, and Fast Track designations for NPC treatment. The European Medicines Agency has also designated it as an Orphan Medicinal Product for NPC treatment.
About Orsini
Orsini has been delivering comprehensive and compassionate patient care since 1987, focusing on rare diseases and gene therapies. Working closely with biopharma companies, healthcare providers, and payors, Orsini ensures access to innovative treatments for rare diseases. It offers integrated rare disease pharmacy solutions, including distribution, patient services, clinical management, and home infusion services. Orsini's patient-centric care model emphasizes personalized support through experienced therapy care teams, ensuring no patient is left behind. Orsini's accreditations include ACHC, The Joint Commission, URAC, and NABP. It has been recognized with URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, contact Orsini at 847-734-7373 ext. 505, email media@orsinihc.com, or visit www.orsinispecialtypharmacy.com. SOURCE: Orsini