Galicia Becomes First in Spain to Approve Gene Therapy for Hemophilia B

The President of the Xunta, Alfonso Rueda, announced on Monday that the regional government of Galicia has approved an investment of €15.6 million for the purchase of the first gene therapy drugs for treating hemophilia B.

He emphasized that the tender for the supply of the drug Etranacogene Dezaparvovec, a gene therapy treatment for the hereditary blood coagulation disorder, affirms “the validity of public health” and positions Galicia as “the first community in Spain to treat hemophilia B with gene therapy.”

Galicia Invests €15.6 Million to Launch Groundbreaking Gene Therapy Treatment for Hemophilia B Patients

Rueda highlighted that prior to the development of this therapy, hemophilia B had no curative treatment. The initial contract for the drug’s supply will last 24 months, with the possibility of extending it for an additional two years.

Hemophilia B (HB) is a rare, genetically inherited disease with an incidence rate of five cases per 100,000 males. It is linked to the X chromosome and caused by a deficiency or absence of factor IX. Although it primarily affects men, women carrying the mutation may also show symptoms of the condition.

The General Directorate of the Basic Portfolio of Services of the National Health System and Pharmacy has approved the inclusion of this gene therapy in the national pharmaceutical provision. However, its application is limited to treating adults with severe or moderately severe hemophilia B. These patients must have no history of inhibitors to factor IX, must have received stable prophylactic treatment with extended-life recombinant factor IX for at least two years, and must present anti-capsid neutralizing antibody levels against AAV5 equal to or below 1:678.

Rueda noted that there are currently 51 individuals diagnosed with hemophilia B in Galicia—37 men and 14 women. Among these, it is expected that eight patients may be eligible to receive gene therapy, provided they meet all other clinical inclusion criteria specified in the Etranacogene Dezaparvovec Pharmacoclinical Protocol.

Galicia’s investment and treatment initiative mark a significant milestone in the application of gene therapy within Spain’s public healthcare system and represents a step forward in improving care for patients with rare genetic disorders.

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(Featured image by Sangharsh Lohakare via Unsplash)

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First published in diariofarma. A third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.

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