The Swiss drug manufacturer Novartis has been approved in the US for its Zolgensma gene therapy for the treatment of spinal muscular atrophy and has estimated a one-time treatment with a record $ 2.125 million.
The Food and Drug Administration approved Zolgensma on Friday for children under the age of two with SMA, including those that do not show symptoms. The approval is for babies with the most deadly form of hereditary disease, as well as for babies with types, which can later debilitating symptoms.
SMA is the most common genetic cause of death in infants. The disease often causes paralysis, difficulty in breathing and death in infants with the most severe form 1
"This may be a new standard for caring for babies with the severest form of SMA," Dr. Emmanuelle Tiongson, a Los The pediatric neurologist from Angeles, who provided Zolgensma as part of an extended patient access program.
Executives at Novartis have defended the award, claiming that one-off treatment is more valuable than expensive long-term care, costing hundreds of thousands of dollars a year.  The therapy uses a virus to provide a normal copy of the SMN1 gene to infants born with a defective gene.
A review by the Independent Institute for Clinical and Economic Verification (Icer) concluded Novartis' previous estimate of $ 5 million per patient for Zolgensma was exaggerated.
However, on Friday, Icer announced that the drug was expected to decline due to additional Novartis clinical data, broad FDA label and introductory price. Lower cost-effectiveness cap.
Novartis claims to have treated more than 150 patients with Zolgensma. Its CEO, Vas Narasimhan, described Zolgensma as a cure for SMA when delivered shortly after birth. However, the data showing durability only last for about five years.
Novartis expects European and Japanese approval later this year. Zolgensma will compete with Biogens Spinraza, the first approved treatment for SMA.
Spinraza, which was approved at the end of 2016, must be infused every four months. The list price of $ 750,000 for the first year and $ 375,000 a year was also rated as excessive by Icer.
Wall Street analysts forecast $ 2 billion in revenue for 2022, according to a survey by Refinitive. Spinraza sales reached $ 1.7 billion last year and are expected to reach $ 2.2 billion in 2022. USD rise.