Novartis has got the U.S. approval for Zolgensma drug for the treatment of spinal solid decay (SMA), the main hereditary reason for death in newborn children, and estimated the one-time treatment at a record $2.125 million.
The FDA has approved Zolgensma for infants under the age of two with SMA without any symptoms. The disease can lead to breathing difficulties, paralysis and in some cases death within a few months. The approval based on the clinical trial of 36 patients aged 2 weeks to 8 months.
Novartis defended the statement of price saying that a one-time treatment is more important than costly long haul medications that cost a few hundred thousand dollars per year.
Novartis said it was offering health insurers the option of instalment payments for Zolgensma as well as refunds if the treatment does not work and upfront discounts for payers who commit to standardized coverage terms.
The company also offering insurers with options of instalments for Zolgensma just as refunds if the treatment does not work.
As per Novartis, the durability of a drug is only about five years. The treatment utilizes a virus to give an ordinary duplicate of the SMN1 gene to babies with a defective gene which delivered by implantation.
The company also anticipates approval from EU and Japan in the coming days.