Ultragenyx Pharmaceutical (RARE.O) announced on Thursday that it has set a U.S. list price of $3.95 million for its recently licensed gene therapy Fayuvi, introducing one of the most costly medications in the world as a one-time treatment for a rare and deadly children illness.
Fayuvi has been licensed by the US Food and Drug Administration for use in children with Sanfilippo syndrome Type A, a genetic disorder that gradually impairs the brain and neurological system.
The illness is brought on by a lack of the enzyme sulfamidase, which causes the body and brain to abnormally accumulate heparan sulfate, a complex sugar compound. Children gradually lose their motor, linguistic, and cognitive abilities as a result of this toxic accumulation.
During a conference call, Ultragenyx executives stated that the lifetime cost of caring for a child with the illness, who may be bedridden for years, can surpass $8 million. They claimed that this burden increases as the illness progresses.
Fayuvi may achieve peak global sales of $200 million to $250 million, according to J.P. Morgan analysts.
The gene therapy, which is administered as a single intravenous infusion, gives cells a functional copy of the SGSH gene, allowing the body to manufacture sulfamidase and eliminate the harmful accumulation.
According to the FDA, children who received the therapy in trials either maintained or improved their cognitive performance when compared to those who did not receive treatment.
