Hemophilia | Hemophilia Treatment | ROCTAVIAN

About the Disease

Hemophilia is an inherited bleeding disorder in which the blood does not clot due to insufficient clotting factors, causing spontaneous bleeding, pains, swelling or tightness in joints, blood in urine or stool, and nose bleeds. The most common types of Hemophilia are Hemophilia A & B.

Hemophilia A, also called factor VIII deficiency or classic Hemophilia, is an X-linked genetic disorder caused by missing or defective factor VIII, a clotting protein. Approximately 1 in 10,000 people have Hemophilia A. Individuals with the most severe form of hemophilia A make up approximately 50% of the Hemophilia A population.

Hemophilia B, also known as Christmas disease or factor IX deficiency, is a rare, degenerative disease that occurs in approximately 1 in 25,000 male births. People with this condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Hemophilia B can range from mild (factor IX levels between 5 and 40% of normal) to moderate (factor IX levels from 1 to 5% of normal) to severe (factor IX levels less than 1% of normal).

Current treatment for moderate to severe hemophilia includes lifelong prophylactic infusions of factor VIII and factor IX to replace or supplement low levels of the blood-clotting factor temporarily; however, patients may still experience spontaneous bleeding episodes, limited mobility, joint damage, or severe pain as a result of the disease. New gene therapy infusion treatment is now available to enable people living with Hemophilia to produce their own factor, which can help reduce the rate of annual bleeds and help reduce or eliminate the need for prophylactic therapy entirely.

About our Patient Care

Our dedicated Hemophilia Gene Therapy Care Team will work with the physician's office and insurance on a patient's behalf to give our patients 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. In addition, Orsini is one of the specialty pharmacies selected to provide patients with access to an FDA-approved, adeno-associated virus vector-based gene therapy.

As part of treatment, we provide:

Dedicated Hemophilia Gene Therapy Care Team

Hemophilia Gene Therapy Care Team, including a single point of contact, clinical education, side-effects management, and a direct phone, fax, and email address. Orsini will assist with administrative responsibilities associated with the therapy, including benefit investigation, prior authorization, and support in securing financial assistance.

Coordination of Care

Manage care coordination between prescribers, sites of care, and patients to ensure dispense and drug delivery align with the date and location of service. ROCTAVIAN is delivered via courier to an approved site of care for patient administration.

Financial Assistance

We successfully identify and assist in enrolling patients into foundation support programs to minimize a potential financial burden to patients, given the high cost of specialty medications.