Orsini Specialty Pharmacy has recently been selected by Takeda Pharmaceuticals as the exclusive specialty pharmacy partner for ADZYNMA, a groundbreaking treatment for the rare blood clotting disorder known as congenital thrombotic thrombocytopenic purpura (cTTP). This partnership marks an important milestone in addressing the unmet medical needs of patients suffering from cTTP.
cTTP is an ultra-rare and debilitating condition caused by a deficiency in the ADAMTS13 enzyme, which is essential for blood clotting regulation. Without sufficient levels of this enzyme, individuals with cTTP are at risk of life-threatening acute events and experience chronic symptoms. In fact, when left untreated, acute TTP events have a mortality rate exceeding 90%.
ADZYNMA, the first-ever FDA-approved recombinant ADAMTS13 enzyme replacement therapy, aims to target the root cause of cTTP by addressing the ADAMTS13 enzyme deficiency. By providing prophylactic or on-demand enzyme replacement therapy (ERT), ADZYNMA offers hope to those living with cTTP who previously had no treatment options specifically indicated for their condition.
“We’re proud to offer hope to those living with cTTP, who have previously had no treatment options specifically indicated for them,” stated Darin DeCarlo, Chief Growth Officer of Orsini Specialty Pharmacy. Orsini is committed to its ongoing partnership with Takeda in their mission to address unmet medical needs and improve the quality of life for patients.
Orsini Specialty Pharmacy, a leader in rare diseases and gene therapies, has been providing comprehensive and compassionate care to patients since 1987. The company works closely with biopharma innovators, healthcare providers, and payors to ensure that patients and their families can access revolutionary treatments for rare diseases. With a high-touch care model and a focus on personalized patient care, Orsini aims to leave no patient behind in their journey towards improved health.
FAQ:
Q: What is cTTP?
A: Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme, putting individuals at risk of life-threatening acute events and chronic symptoms.
Q: What is ADZYNMA?
A: ADZYNMA is the first FDA-approved recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy for people with cTTP, designed to address the root cause of the condition and provide prophylactic or on-demand enzyme replacement therapy.
Sources:
– Orsini Specialty Pharmacy [www.orsinispecialtypharmacy.com]