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Types Of Hemophilia / Haemophilia

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Hemophilia A and Hemophilia B

There are two main types of hemophilia - Hemophilia A (due to factor VIII deficiency) and Hemophilia B (due to factor IX deficiency). They are clinically almost identical and are associated with spontaneous bleeding into joints and muscles and internal or external bleeding after injury or surgery.

After repeated bleeding episodes permanent damage may be caused to the joints and muscles that have been affected, particularly the ankles, knees and elbows.

Approximately 1 in 5,000 males is born with Hemophilia A, and 1 in 30,000 males is born with Hemophilia B. Hemophilia affects people of all races and ethnic origins globally. The conditions are both X-linked and virtually all sufferers of hemophilia are males. Female carriers may also bleed abnormally, because some have low levels of the relevant clotting factor.

People with hemophilia have a genetic mutation in the affected gene on the X chromosome, which results in reduced production of Factor VIII or IX and creates a bleeding tendency, because coagulation takes much longer than normal, thus making the clot weak and unstable

Approximately one third of patients with hemophilia have no family history of the disease, either because of new genetic mutations, or because previous affected generations either had daughters (who were carriers) or sons who died in early childhood from hemophilia or any other cause or who were not affected.

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This is very rare. The patient develops the condition during his/her lifetime and it does not have a genetic or heritable cause. It occurs when the body forms antibodies that attack one or more blood clotting factors, (usually factor VIII), thus preventing the blood clotting mechanism from working properly. Patients may be male or female and the pattern of bleeding is rather different from that of classical hemophilia, the joints being rarely affected. The disorder is particularly associated with old age and occasionally complicates pregnancy.

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Koate-dvi

Koate-dvi Generic Name & Formulations General Description

Antihemophilic Factor VIII (human) 250 IU, 500 IU, 1000 IU; per bottle; dried concentrate for IV infusion after reconstitution; contains albumin.

Pharmacological Class

Clotting factor.

How Supplied

Single-dose bottle—1 (w. Diluent)

Koate-dvi Indications Indications

Prevention and control of hemorrhagic episodes or in order to perform emergency or elective surgery in Hemophilia A patients.

Koate-dvi Dosage and Administration Adult

Dosage Required (IU) = Body Weight (kg) × Desired % Factor VIII Increase × 0.5. Individualize. Infuse over 5–10 minutes if tolerated. Use filter needle. Hemorrhage: Mild: 10 IU/kg as single dose. Moderate: 15–25 IU/kg, then 10–15 IU/kg every 8–12hrs if needed. Severe: initially 40–50 IU/kg, then 20–25 IU/kg every 8–12hrs. Major surgery: pre-op dose: 50 IU/kg, then verify Factor VIII level achieved prior to surgery; may repeat every 6–12hrs initially and for 10–14 days until healing complete.

Children

Not recommended.

Koate-dvi Contraindications

Not Applicable

Koate-dvi Boxed Warnings

Not Applicable

Koate-dvi Warnings/Precautions Warnings/Precautions

Not for von Willebrand's disease. Confirm Factor VIII deficiency prior to treatment. Contains human plasma; monitor for possible infection transmission. Large or frequent doses: monitor hematocrit for signs of progressive anemia. Monitor for development of Factor VIII inhibitors. Pregnancy (Cat.C).

Koate-dvi Pharmacokinetics

See Literature

Koate-dvi Interactions

Not Applicable

Koate-dvi Adverse Reactions Adverse Reactions

Allergic reactions, tingling sensations, blurred vision, headache, GI upset, jittery feeling, antibody formation.

Koate-dvi Clinical Trials

See Literature

Koate-dvi Note

Not Applicable

Koate-dvi Patient Counseling

See Literature

FDA Approves Pfizer's Second Hemophilia Drug With Six Months

On Friday, the FDA approved Pfizer Inc's (NYSE:PFE) Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency), or hemophilia B (congenital factor IX deficiency).

Hympavzi is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the U.S. For hemophilia A or B and the first hemophilia medicine approved in the U.S. To be administered via a pre-filled, auto-injector pen.

Hympavzi can offer a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration.

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.

Results from the Phase 3 BASIS trial (NCT03938792) supported the FDA approval of Hympavzi.

In the study, Hympavzi reduced the annualized bleeding rate (ABR) for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis (RP) and on-demand (OD) treatment, respectively, in patients with hemophilia A or B without inhibitors.

The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion for marstacimab for the routine prophylaxis of bleeding episodes in adults and adolescents with severe hemophilia A or hemophilia B.

Reuters, citing some analysts, highlights that Hympavzi sales are expected to reach $300 million by 2030.

Earlier this year, FDA approved Pfizer's Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid.

Beqvez is a one-time treatment designed to enable hemophilia B patients to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.

In July, Pfizer, in collaboration with Sangamo Therapeutics Inc, released results from the Phase 3 AFFINE study of giroctocogene fitelparvovec, an investigational gene therapy for adult patients with moderately severe to severe hemophilia A.

Price Action: PFE stock is down 0.14% at $29.12 during the premarket session at last check Monday.

Photo via Shutterstock

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