PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) TREATMENT
About the Disease
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder in which red blood cells break apart prematurely. It is an acquired hematopoietic stem cell disorder, and some hematopoietic stem cells in individuals with PNH are defective and consequently produce defective blood cells. These defective red blood cells of PNH are extremely susceptible to premature destruction by a particular part of a person’s own immune system called the complement system, leading to episodes of hemoglobin in the urine. Individuals with hemoglobinuria may exhibit dark-colored or blood colored urine, which may not always be visible to the eye. In addition to hemolysis, individuals with PNH are also susceptible to developing repeated, potentially life-threatening blood clots.
Affected individuals also have some degree of underlying bone marrow dysfunction.
About our Patient Care
Our dedicated Care Team will work with the physician's office and insurance on a patient's behalf so that our patients have more time to focus on their treatment and quality of life. We are here to listen, provide
counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.
As part of treatment, we provide:
Dedicated Care Team
We provide prescribers and patients with a dedicated care team for each of our disease state programs. This includes a primary pharmacy contact, clinical education, side-effects management, scheduled refill calls, and a direct phone, fax and email address.
In-Home Patient- and Family-Centric Care
We administer a nationwide nursing network, allowing patients to pursue in-home treatment or receive training on self-administrated drugs. Our nurses are ideally positioned to establish a partnership with patients and their families in order to provide clinical education and coaching that strengthen the patient's capacity to achieve therapy goals.
Shipping and Coordination of Care
We provide free overnight delivery of medications and necessary medical supplies and manage the coordination of care between prescribers, sites of care, nurses and patients to ensure dispenses and drug delivery align with the date and location of service.
Financial Assistance
We successfully identify and assist enrolling patients into any and all manufacturer co-pay and foundation support programs in order to minimize a potential financial burden to patients given the high-cost of specialty medications.
24/7/365 Access to a Pharmacist
We provide prescribers and patients access to a pharmacist 24/7/365 to ensure patients are always cared for.
Supported Treatments
Click on the specialty drug name below to expand and see more information.
Soliris (eculizumab)
Brand Name | Soliris
Manufacturer | Alexion
Route of Administration | Intravenous (IV) infusion
Approved Indication | Treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis
Self-Administration | No
Product Website | info.soliris.net/home/
Ultomiris® (ravulizumab-CWVZ)
Brand Name | Ultomiris®
Manufacturer | Alexion®
Route of Administration | Infusion
Approved Indication | Treatment of adults and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) (1)
Self-Administration | No
Product Website | https://www.ultomiris.com/