PITTSBURGH, Dec. 23, 2024 /PRNewswire/ -- PANTHERx Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce that it was selected by Ionis Pharmaceuticals, Inc. for the distribution of TRYNGOLZA (olezarsen), the first and only treatment approved by the U.S. Food and Drug Administration (FDA) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

TRYNGOLZA is a GalNAc3-conjugated antisense oligonucleotide therapy that selectively binds and degrades apoC-III mRNA to reduce hepatic production of apoC-III, a protein that regulates triglyceride metabolism.1

FCS is a rare, genetic disorder of fat metabolism characterized by extremely high plasma triglyceride levels. Most cases of FCS result from mutations in the LPL gene, leading to insufficient or impaired lipoprotein lipase (LPL) enzyme function.2 Lipoprotein lipase is responsible for breaking down chylomicrons, large lipid-containing particles primarily composed of triglycerides. In FCS, impaired ability to break down chylomicrons leads to an accumulation of triglycerides in the blood, placing patients at risk for acute pancreatitis, which can lead to long-term pancreatic dysfunction and may be fatal if left untreated.3 Additionally, this build-up of triglycerides can cause symptoms that produce significant physical and emotional burden for patients, such as enlargement of the liver and spleen (hepatosplenomegaly), chronic fatigue, severe abdominal pain, depression, and memory impairment. 3,4

"This partnership with Ionis will help to redefine life for adults with FCS by streamlining the access and support process. We are looking forward to serving the needs of the patients dealing with this illness, by streamlining the process associated with getting this medication from the people who created it to the people who need it most," Rob Snyder, Executive Chair, PANTHERx Rare Pharmacy.

About PANTHERx Rare

PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans.3 Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. 3 Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.

PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence.4 As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories.

1. TRYNGOLZA (olezarzen) [package insert]. Ionis Pharmaceuticals, Inc. 2024.
2. Goldberg RB, Chait A. A Comprehensive Update on the Chylomicronemia Syndrome. Front Endocrinol (Lausanne). 2020;11:593931.
3. Olezarsen (Apo-CIII), Familial Chylomicronemia Syndrome. IONIS Pharmaceuticals, Inc. Published August 1, 2024. Accessed October 10, 2024.
4. Paragh G, Németh Á, Harangi M, et al. Causes, clinical findings and therapeutic options in chylomicronemia syndrome, a special form of hypertriglyceridemia. Lipids Health Dis. 2022;21(1):21.

For more information, please contact [email protected]

SOURCE PANTHERx Rare Pharmacy