HEMGENIX®

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HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who:

Currently use Factor IX prophylaxis therapy, or
Have current or historical life-threatening hemorrhage, or
Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is an in vivo gene therapy that introduces a functional copy of the F9 gene, and after single intravenous infusion results in cell transduction and increase in circulating factor IX activity in patients with hemophilia B.

HEMGENIX consists of a liver-directed adeno-associated viral vector serotype 5 (AAV5) containing the factor IX-Padua gene cassette.

The AAV5 is nonreplicating and has a serotype-specific tropism for hepatocytes, where factor IX is naturally produced.
The highly active factor IX-Padua variant has been shown to generate 5-8 times higher mean endogenous factor IX activity than the wild-type gene. The factor IX-Padua gene is under the control of a liver-specific promoter.

With HEMGENIX, the factor IX-Padua gene is contained within the AAV5 vector to introduce a functional copy of the F9 gene into a patient’s hepatocytes.

AAV=adeno-associated virus; FIX=clotting factor IX.
1. D’Avola D, et al. J Hepatol. 2016;65:777–783; 2. Miesbach W, et al. Blood. 2018;131:1022–1031; 3. Pipe SW, et al. Poster presented at 61st ASH meeting, 2019.

HEMGENIX is provided as a customized kit containing 10-48 vials of HEMGENIX, corresponding to the dosing requirement for the individual patient, depending on their body weight. HEMGENIX vials contain no less than 10-mL of extractable volume and are for single use only.

HEMGENIX Example Dose Calculation

1. The recommended dose of HEMGENIX is 2 x 10¹³ genome copies per kg of body weight (or 2 mL/kg of body weight) administered as an IV infusion after dilution with 0.9% sodium chloride solution (normal saline)

Calculate the dose as follows:
Dose (in mL) = patient body weight (in kg) x 2

For example, for a patient with a body weight of 85 kg, the dose would be:
85 x 2 = 170 mL

A patient with a body weight of 128 kg would require:
128 x 2 = 256 mL

2. Number of HEMGENIX vials needed = HEMGENIX dose (in mL) divided by 10 (round up to the next whole number of vials). The total volume of the patient's HEMGENIX dose to be diluted may be less than the total volume of vials needed.

Patient Weight	HEMGENIX dose (mL) (body weight multiplied by 2)	Number of vials needed [HEMGENIX dose (mL) divided by 10, then rounded up]
85 kg	170 mL	17
128 kg	256 mL	26

3. Prior to dilution, withdraw the volume of the calculated dose from the 500 mL infusion bag(s) of 0.9% normal saline solution, depending on patient’s body weight

- For patients with a body weight of <120 kg, withdraw saline volume equal to the total HEMGENIX dose in one 500 mL 0.9% normal saline infusion bag
- For patients with a body weight of ≥120 kg, withdraw half of the dose equivalent volume from each of the two 500 mL 0.9% normal saline infusion bags

4. The volume of the required dose is then added to the infusion bag(s) to bring the total volume in each infusion bag back to 500 mL


Recommended dose = 85 kg x 2 mL/kg = 170 mL Dilute the 170 mL of HEMGENIX in one 500 mL 0.9% normal saline solution infusion bag	Recommended dose = 128 kg x 2 mL/kg = 256 mL Dilute the 256 mL of HEMGENIX in two 500 mL 0.9% normal saline solution infusion bags (128 mL in each bag)

Handling

	Provided in a sealed biohazard bag as a customized kit depending on the patient’s body weight, each kit contains 10 to 48 vials (10 mL each)
	Cold chain storage and handling required. Product is shipped at 2°C to 8°C (36°F to 46°F)
	Store HEMGENIX in a refrigerator at 2°C to 8°C (36°F to 46°F) in the original carton within the biohazard bag. Protect from light until time of dilution and administration. Do not freeze.
	After dilution, store in the infusion bag protected from light for up to 24 hours at 15°C to 25°C (59°F to 77°F) after the dose preparation