PantherRx Rare and Orsini Specialty Pharmacy Chosen as Partners for OXLUMO

Alnylam^® Pharmaceuticals selected PantherRx Rare and Orsini Specialty Pharmacy as limited distribution partners for OXLUMO™ (lumasiran). OXLUMO is the first and only treatment approved for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

PH1 is an ultra-rare genetic disease characterized by the overproduction of oxalate, a waste product that usually is eliminated by the kidneys and excreted in the urine. It is estimated that PH1 affects one to three individuals per million in the United States and Europe.

Oxlumo received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. The application was also granted breakthrough therapy designation.

In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children.

Both PantherRx Rare and Orsini Specialty Pharmacy are leading specialty pharmacies focused on rare and ultra-rare diseases and gene therapies. In the United States, a rare disease is defined as a condition that affects fewer than 200,000 Americans.

There may be as many as 7,000 rare diseases. The total number of Americans living with a rare disease is estimated at between 25-30 million. This estimate has been used by the rare disease community for several decades to highlight that while individual diseases may be rare, the total number of people with a rare disease is large. 

PANTHERx Rare Pharmacy, the largest independent and fastest growing specialty pharmacy in the United States, transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions.

Providing patients with comprehensive and compassionate care since 1987, Orsini is a leading, independent specialty pharmacy focused on rare and ultra-rare diseases and gene therapies. Orsini’s high touch care model is centered around experienced, therapy-specific care teams who provide personalized care to patients based on their specific condition and treatment.

Takeaway: Both PantherRx Rare and Orsini Specialty Pharmacy are leading specialty pharmacies focused on rare and ultra-rare diseases and gene therapies