Q1 2024
Newsletters and Drug Reports

Beqvez Gene Therapy Receives FDA Approval for Hemophilia B

On April 26, 2024 the US Food and Drug Administration (FDA) approved Beqvez™ (fidanacogene elaparvovec)

from Pfizer Inc. for the treatment of adults with moderate to severe hemophilia B 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 (AAVRh74var). Beqvez is a one-time

treatment designed to enable hemophilia B patients to produce FIX. It is the second gene therapy

for Hemophilia B and has a list price of $3.5 million, the same as Hemgenix® (etranacogene dezaparvovec),

which received FDA approval in 2022.

Sarepta wins full approval and label expansion for its DMD Gene Therapy.

Elevidys will be available to Duchenne Muscular Dystrophy (DMD)

patients who are at least 4 years old, regardless [biospace.com] of whether they can walk.

Elevidys’ current FDA approved label is limited to 4- and 5-year-olds.

Approximately 300 to 400 boys are born with DMD every year in the U.S.,

and the disease kills around 400 boys and young adults annually. The DMD

population somewhere around 14,000, including ambulatory and

non-ambulatory patients. The current wholesale acquisition cost for this

one time gene therapy is 3.2 million. Please click on the link below

for more details.

SIIA CGT Stakeholder Forum Summary Notes