Science

Costly gene therapy drug could enter U.S. market: report


WASHINGTON, May 8 — A drug that can cure the most fatal genetic deficiency in infants is expected to enter the U.S. market soon, but the potential seven-digit price per treatment has raised concerns over its feasibility, according to U.S. media report.

The Zolgensma therapy, developed by Swiss pharmaceutical company Novartis AG, is awaiting a decision by the U.S. Food and Drug Administration due this month, the Wall Street Journal reported on Tuesday.
Zolgensma can cure the genetically inherited disease known as spinal muscular atrophy (SMA), whose sufferers lack a gene essential for muscle control. If left untreated, those who suffer the most severe form of SMA typically die before reaching two years old, making the disease the most common genetic cause of infant death.

Currently, drug-makers and independent analysts differ on setting the installment price. Analysts believe the drug, if launched, will generate around 2 billion U.S. dollars a year at peak for Novartis, the journal said.

Novartis, based on its own analysis, said the installment for Zolgensma could be cost-effective in the range of four to five million dollars, while an analysis by the Institute for Clinical and Economic Review, an independent nonprofit organization, concluded that the initial price should be no higher than 1.5 million dollars.
Zolgensma’s high price worries both insurers and patients, but Novartis remained confident about the applicability of two new payment models, according to the report.

In one model, patients pay an installment at the beginning of the treatment and subsequent annual payment is contingent upon the therapy’s continued effectiveness. Insurers could also pay upfront but get a partial refund if treatment proves ineffective.
The number of newborn babies carrying SMA is between 400 and 500 in the United States each year, and some 300 of them have the most severe version, according to the report. – XINHUA