Etranacogene dezaparvovec

Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.

The most common adverse reactions include liver enzyme elevations, headache, mild infusion-related reactions and flu-like symptoms.

Etranacogene dezaparvovec was approved for medical use in the United States in November 2022, and in the European Union in February 2023.

Medical uses

Etranacogene dezaparvovec is indicated for the treatment of adults with hemophilia B (congenital Factor IX deficiency) who use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

History

The safety and effectiveness of etranacogene dezaparvovec were evaluated by the US Food and Drug Administration (FDA) in two studies of 57 adult men 18 to 75 years of age with severe or moderately severe hemophilia B. Effectiveness was established based on decreases in the men’s annualized bleeding rate (ABR). In one study, which had 54 participants, the subjects had increases in Factor IX activity levels, a decreased need for routine Factor IX replacement prophylaxis, and a 54% reduction in ABR compared to baseline.

The FDA granted the application for etranacogene dezaparvovec priority review, orphan drug, and breakthrough therapy designations. In November 2022, the FDA granted approval of Hemgenix to CSL Behring LLC.

Society and culture

Economics

Etranacogene dezaparvovec costs US$3.5 million per dose. The manufacturer claims that the drug will reduce total health care costs because patients will have fewer bleeding incidents and need fewer clotting treatments.

Legal status

On 15 December 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Hemgenix, intended for the treatment of severe and moderately severe hemophilia B. The applicant for this medicinal product is CSL Behring GmbH. Etranacogene dezaparvovec was approved for medical use in the European Union in February 2023.

Further reading

• Nathwani AC, McIntosh J, Sheridan R (September 2022). "Liver Gene Therapy". Human Gene Therapy. 33 (17–18): 879–888. doi:10.1089/hum.2022.169. PMID 36082993.
• Von Drygalski A, Giermasz A, Castaman G, Key NS, Lattimore S, Leebeek FW, et al. (November 2019). "Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B". Blood Advances. 3 (21): 3241–3247. doi:10.1182/bloodadvances.2019000811. PMC 6855101. PMID 31698454.

External links

• "Etranacogene dezaparvovec". Drug Information Portal. U.S. National Library of Medicine.
• Clinical trial number NCT02396342 for "Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B" at ClinicalTrials.gov
• Clinical trial number NCT02396342 for "Dose Confirmation Trial of AAV5-hFIXco-Padua" at ClinicalTrials.gov
• Clinical trial number NCT02396342 for "HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients" at ClinicalTrials.gov