Orsini Chosen as the Exclusive Specialty Pharmacy Provider for MIPLYFFA™ (arimoclomol), the First FDA-Approved Treatment for Niemann-Pick Disease Type C

ELK GROVE VILLAGE, Ill., Sept. 24, 2024 /PRNewswire/ -- Orsini today announced that Zevra Therapeutics, Inc. (Zevra) has selected them as the exclusive specialty pharmacy partner for MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. Visit https://zevra.com/documents/MIPLYFFA-Prescribing-Information.pdf to view the Full Prescribing Information, including Important Safety Information. MIPLYFFA is the second therapy from Zevra to join Orsini's portfolio, following the announcement of Orsini as the pharmacy partner for OLPRUVA® (sodium phenylbutyrate) in June 2024.

"Orsini is thrilled to expand our partnership with Zevra and to continue our work with them by bringing this groundbreaking therapy to those living with NPC," said Darin DeCarlo, Orsini's Chief Growth Officer. 

About Niemann-Pick Disease Type C
Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality.

About MIPLYFFA
MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.

About Orsini
Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™.

Orsini Specialty Pharmacy holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned 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 us at [email protected], or visit www.orsinispecialtypharmacy.com.

SOURCE Orsini