Fabhalta is prescribed for adults diagnosed with paroxysmal nocturnal hemoglobinuria (PNH). It is used to treat this medical condition.

Fabhalta is a novel therapeutic agent that specifically targets the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder characterized by the destruction of red blood cells. The active ingredient in Fabhalta is iptacopan, which operates through a unique mechanism to address the underlying pathology associated with PNH. In patients with this condition, aberrant blood cell destruction leads to a host of serious complications including severe anemia, hemolytic symptoms, and thrombosis.

The mechanism of action of iptacopan involves the inhibition of the complement system, a part of the immune response that can erroneously attack the body’s own red blood cells in PNH. By modulating this pathway, Fabhalta effectively reduces hemolysis and improves hemoglobin levels in patients. Moreover, the therapy aims to alleviate the debilitating symptoms associated with PNH, such as fatigue, weakness, and increased risk of clotting complications.

Fabhalta is available in a capsule form, which is essential for the effective delivery of iptacopan to the bloodstream. The capsules are pale yellow and opaque, making them easy to identify. Each capsule is marked with specific identifiers: "lnp200" and "nvr," ensuring proper recognition and minimizing potential medication errors. The formulation is designed to enhance patient compliance, allowing for a more manageable treatment regimen.

Fabhalta is available in capsule form with a strength of 200 mg of Iptacopan. The capsules are pale yellow, opaque, and made of hard gelatin. They are marked with "LNP200" on the body and "NVR" on the cap.

Dosage and Administration for Fabhalta:

• Recommended Vaccination and Prophylaxis for Encapsulated Bacterial Infections:

  • Vaccinate patients against encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis (serogroups A, C, W, Y, and B), and Haemophilus influenzae type B, based on current ACIP recommendations. Administer vaccinations at least 2 weeks before starting Fabhalta.

  • In cases where urgent Fabhalta therapy is necessary for a patient not up to date with the recommended vaccines, provide antibacterial drug prophylaxis. Administer the required vaccines as soon as possible.

  • Healthcare providers prescribing Fabhalta must enroll in the Fabhalta Risk Evaluation and Mitigation Strategy (REMS) [see Warnings and Precautions].

• Recommended Dosage:

  • The recommended dosage of Fabhalta is 200 mg orally twice daily, regardless of food intake.

  • Swallow the capsules whole, without opening, breaking, or chewing them.

  • If a dose is missed, advise the patient to take one dose of Fabhalta as soon as possible. They should resume the regular dosing schedule even if it's close to the next scheduled dose.

• Patients Switching from Anti-C5 (eculizumab, ravulizumab) to Fabhalta:

  • To minimize the risk of hemolysis when transitioning from other PNH therapies:

    • For patients switching from eculizumab, start Fabhalta no later than 1 week after the last eculizumab dose.

    • For patients switching from ravulizumab, initiate Fabhalta no later than 6 weeks after the last ravulizumab dose.

    • Information regarding the initiation timeframe for Fabhalta after other PNH therapies is currently unavailable.

Transitioning to Fabhalta from Other PNH Therapies

The transition from established therapies for Paroxysmal Nocturnal Hemoglobinuria (PNH), specifically anti-C5 therapies like eculizumab and ravulizumab, to Fabhalta is a multifaceted process that requires careful consideration. These transitions are critical as they aim to minimize the risks associated with treatment changes, including the potential for hemolysis, a significant complication that can arise during this period.

When switching to Fabhalta, it is essential to adhere to the recommended timeframes after the last administration of eculizumab or ravulizumab. Typically, healthcare providers recommend initiating Fabhalta treatment at least two weeks after the final dose of eculizumab. This timeframe allows for the gradual clearance of the previous medication from the patient’s system while also reducing the risk of hemolysis, which can occur due to an imbalance in complement activity post-discontinuation of the anti-C5 therapy.

For patients transitioning from ravulizumab, the timing may differ slightly due to its longer dosing interval. Here, it is generally advisable to start Fabhalta approximately four weeks after the last dose of ravulizumab. This distinction is important as it ensures that patients receive optimal therapeutic support while mitigating any adverse events linked to abrupt changes in their treatment regimen.

During this transition period, healthcare providers should closely monitor patients for any signs of hemolysis, alongside other potential complications. This vigilance is key, as any emerging symptoms should prompt immediate clinical intervention. Furthermore, patient education regarding the signs of hemolysis and the importance of adhering to the prescribed transition plan is vital for ensuring optimum outcomes during the switch to Fabhalta.

In conclusion, the successful transition to Fabhalta from other therapies requires careful planning and consideration to minimize risks, ensuring that patients continue to receive effective treatment for their PNH.

Safety Precautions and Risk Management with Fabhalta

The administration of Fabhalta, indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), necessitates stringent safety precautions. Healthcare providers must ensure that they or the medical facility is enrolled in the Fabhalta Risk Evaluation and Mitigation Strategy (REMS). This program is specifically designed to mitigate the risks associated with Fabhalta use, promoting patient safety and minimizing adverse effects. By adhering to the REMS guidelines, healthcare professionals can effectively monitor patient responses to the medication while ensuring that patients are well-informed about potential complications.

Prior to initiating treatment with Fabhalta, it is imperative to evaluate the patient’s medical history, as certain contraindications may contribute to heightened risk factors. Conditions such as active malignancies or hypersensitivity to any components of the medication should be carefully considered. Additionally, the potential for severe side effects, including thrombosis or infusion-related reactions, necessitates further discussion between patients and healthcare providers. Proper patient selection and risk assessment can greatly influence treatment outcomes and enhance the therapeutic efficacy of Fabhalta.

Regular monitoring during treatment is essential to identify any adverse reactions promptly. Clinicians should promote systematic evaluations of hematological parameters and assess for signs of hemolysis or other complications that may arise. Furthermore, patients are advised to report any unusual symptoms or changes in health status while on Fabhalta therapy. Clear communication about the importance of following prescribed monitoring schedules cannot be overstated, as diligent observation can prevent serious repercussions.

In conclusion, adherence to safety precautions surrounding Fabhalta treatment for PNH is critical. It is essential for patients to engage in open discussions with their healthcare providers regarding risks, benefits, and necessary safety protocols associated with this medication. This proactive approach ensures that the engagement with Fabhalta is both safe and effective, ultimately leading to improved patient outcomes.