Health

Novartis Rs 14-Cr Gene Therapy

by Barkha-Rawat
May 27, 2019 14:45

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Novartis Rs 14-Cr Gene Therapy. Swiss drugmaker Novartis won US approval for its GENE THERAPY ZOLGENSMA FOR SPINAL MUSCULAR ATROPHY (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million (around Rs 14 crore). The Food and Drug Administration approved Zolgensma for children under the age of 2 with SMA, including those not yet showing symptoms. Novartis executives defended the price, saying that a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year. A review in April by an independent U.S. group, the Institute for Clinical and Economic Review (ICER), concluded Novartis’ value estimate for Zolgensma was excessive.

“This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr. Emmanuelle Tiongson, a pediatric neurologist at Children’s Hospital Los Angeles who has provided Zolgensma to patients under an expanded access programme. “The job now is trying to negotiate with insurers that this would be a long-term savings.”

But yesterday, ICER said that based on Novartis’ additional clinical data, the broad FDA label and its launch price, it believed that the drug fell within the upper bound of its range for cost-effectiveness. Novartis said it was offering health insurers the option of installment payments for Zolgensma as well as refunds if the treatment does not work and upfront discounts for payers who commit to standardized coverage terms.