A new treatment for a devastating rare genetic disease has just been approved by the US.

Called Zolgensma, it is a cutting-edge, one-time treatment to treat spinal muscle atrophy at its genetic root called a “gene therapy.”

That means it should work longer than the typical drug, but that also means a much higher price tag.

Zolgensma, made by the AveXis unit of drugmaker Novartis, will cost $ 2,125 million.

Rare Genetic Disease.

A new treatment for a devastating rare genetic disease called spinal muscle atrophy was approved by the U.S. Food and Drug Administration on Friday.

Novartis’s Zolgensma treatment.
 Novartis’s AveXis

Zolgensma, the treatment made by the AveXis unit of Novartis, is a one-time therapy that works at the genetic level to treat the disease.

That means the effects of the drug should last a long time, although whether the treatment is permanent is not yet known.

Novartis priced the product at $2.125 million, or $425,000 a year if it paid over a five-year payout plan. The price tag of $2,125 million makes Zolgensma the world’s most expensive drug.

Novartis executives called the price tag fair and reasonable at a conference call with reporters on Friday, citing the value the treatment will provide for patients.

A lifetime of possibilities

“Zolgensma could create a lifetime of opportunities for the children and families affected by this devastating condition,” said Vas Narasimhan, CEO of Novartis in a statement.

According to the SMA Foundation, spinal muscle atrophy affects an estimated 10,000 to 25,000 people in the US.

The Swiss drug giant previously suggested that a price tag of up to $5 million could be appropriate for therapy because it is already very expensive to treat spinal muscle atrophy and Zolgensma is expected to have long-term benefits for those who get it.

Spinraza, for example, the first therapy for spinal muscle atrophy, costs up to $750,000 for the first year of therapy and $375,000 in the following years.

Spinal muscle atrophy is the leading genetic cause of infant death :

The condition is the leading genetic cause of death in infants, which affects muscle movement.

Babies born with spinal muscular atrophy have impaired muscle and head and muscle control, lacking the ability to sit without assistance.

Novartis expects babies born with spinal muscle atrophy under the age of 2 to be Zolgensma’s target population, executives said at the call.

The company said it would launch the drug immediately and expected approximately 1,100 patients at the launch to be eligible for it.

On Friday, Dr. Steve Pearson, chairman of the Institute for Clinical and Economic Review, a group that measures the cost-effectiveness of new drugs, said Zolgensma’s price tag “falls within the upper bound” of a cost-effective price.

The group found that, depending on how the figures are calculated, fair price ranges would be around $1 million to $2 million.

“Zolgensma is transforming the lives of families affected by this devastating disease dramatically,” Pearson said in a statement, adding: “Insurers were going to cover Zolgensma regardless of price, and Novartis spoke publicly about pricing approaching $5 million.

For patients and the entire health system, it is a positive outcome that Novartis instead chose to price Zolgensma at a level that more closely aligns with the benefits for these kids and their families.

Payers such as health insurers will have the option to pay for Zolgensma for up to five years, Novartis said, adding that there were more than 15 payers in “advanced discussions” about contracts with their AveXis unit.