Novartis said it believes its new gene therapy for spinal muscular atrophy, a genetic disease affecting voluntary muscle movement, will be worth more than $4 million per dose, according to STAT.
The drug in question is AVXS-101, which was proven effective in a 15-patient trial for patients with SMA, a rare genetic disorder that in its most severe form is usually fatal for patients before age 2. In the trial, infants with SMA who received AVXS-101 treatment had a 100 percent survival rate after 24 months.
The trial data prompted Novartis to purchase AveXis, the gene therapy's maker, for $8.7 billion in April.
If and when Novartis wins approval for AVSX-101 in the next year, the drugmaker believes it will be worth upward of $4 million, company executives said in a presentation to investors Nov. 5. However, the executives stressed that the actual price would be disclosed after receiving regulatory clearance.
"Four million dollars is a significant amount of money, but we believe this is a cost-effective point," Dave Lennon, president of AveXis, told STAT.
It is unclear if Novartis would be able to convince payers to spend millions of dollars up front with no guarantee of benefit — especially as drug prices remain a top concern in the U.S.
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