Novartis pitches discounts on pricey gene therapy for deadly muscle disorder: Report

Novartis AG wants insurers to commit to coverage for patients identified with the rare and often deadly disease.

LOS ANGELES: Novartis AG is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal muscular atrophy (SMA), a treatment that could cost more than a million dollars, but the gesture comes with strings attached.

The Swiss drugmaker wants insurers to commit to coverage for patients identified with the rare and often deadly disease, according to Dave Lennon, head of Novartis’ AveXis unit.

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Novartis also seeks their support for widespread screening to identify newborns with SMA, and to ensure quick authorization for the gene therapy, with the aim of starting treatment within two weeks of diagnosis, he said. The U.S. Food and Drug Administration is due to decide this month on approval for Novartis’ Zolgensma. Novartis, which has made a huge bet on gene therapy for future growth, says the one-time treatment could be a cure for SMA and is pushing for a price in the range of $1.5 million to $5 million. That would make Zolgensma the most expensive new therapy to date.

SMA is the leading genetic cause of infant mortality, affecting one in every 10,000 live births. There are up to 500 new U.S. cases of SMA annually. About 60 per cent of patients have the most severe Type 1 form of the disease, which often leads to paralysis, impaired breathing and death by their second birthday.

Insurers have balked, saying a multimillion-dollar price tag is too high for society to bear. Their argument is supported by a U.S. drug price watchdog, which concluded Zolgensma is cost-effective at about $900,000.

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It is unusual for drugmakers to offer discounts on a novel and potentially life-saving treatment. But getting insurers to assure quick coverage for eligible patients, rather than subjecting them to a lengthy individual review, could help Novartis as it seeks to displace Biogen Inc’s SMA therapy Spinraza. “We are very much interested in making sure that payers (support) newborn screening, that they establish fast turnaround on coverage decisions, and that they have specific policies that cover gene therapy for SMA,” Lennon told Reuters.

“As we negotiate discounts, et cetera, with payers, and the contracts with those payers, we’re trying to make sure they put those elements in place,” Lennon said.

Both Zolgensma and Spinraza have shown the best results when infants with SMA are treated within the first few weeks of life before symptoms emerge, helping preserve the development of motor neurons necessary for walking and breathing. Only a small number of Zolgensma patients have been followed for up to four years, while Spinraza has been used by thousands of patients since it was approved in 2016.

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Spinraza is also costly. It is administered by spinal injection every four months at a price of $750,000 for the first year and $375,000 annually thereafter. Wall Street forecasts for Zolgensma assume many families will seek the potential one-time gene therapy first, and consider Spinraza if needed later on.

“Early treatment allows payers to reap much more benefit for the patients,” Lennon said.