NVS Novartis AG

By Denise Roland 

Novartis AG launched a lottery-style program to give away doses of its pricey gene therapy for free, drawing criticism from patient groups that say that is an inappropriate way to distribute a lifesaving treatment aimed at babies.

Zolgensma, a one-shot cure for a deadly inherited disease whose victims cannot control their muscles, is so far sold only in the U.S. at a price of $2.1 million, making it the world's most expensive drug. The so-called global managed-access program is aimed at providing Zolgensma to a limited number of spinal muscular atrophy patients outside the U.S.

Under the program, doctors can submit requests for the treatment, with eligible patients entered into a draw every two weeks for free doses. AveXis, the Novartis unit that makes the drug, said it aimed to distribute around 100 free doses a year as long as production capacity allows. Around 1,600 children are thought to have the most severe form of SMA, known as Type 1, in Europe alone.

However, patient groups criticized the move, highlighting the thorny ethical issues that companies face when providing unapproved treatments for free.

"It's really too crude," said Kacper Rucinski, co-founder of U.K.-based patient-advocacy group TreatSMA. "They are making patients compete. Which will be the lucky one? That's not helpful."

Olga Germanenko, a board member of SMA Europe, an umbrella organization for patient-advocacy groups from across the continent, said the lottery made Zolgensma "look like a prize."

Patients would have instead welcomed a program that prioritized the patients or countries with the highest needs, said Mr. Rucinski. In some places, patients can already get a rival SMA treatment called Spinraza, made by Biogen Inc. Novartis's program doesn't prioritize countries where Spinraza isn't routinely available or patients who don't respond to Spinraza.

A spokesman for Novartis said the company was advised to take the lottery approach by a group of bioethics experts, who were concerned that creating complicated criteria could unfairly discriminate against some patients. "In the end we're dealing with a difficult ethical dilemma," he said. "Unfortunately, there is no perfect solution."

Novartis had faced high demand from patients outside the U.S. to provide the treatment free of charge in some cases, although local regulations may prevent some countries from adopting the program.

Managed-access programs are common, especially for life-threatening conditions where the treatment in question is considered a breakthrough. However, companies typically use a criteria based on medical need to decide who should receive the treatment.

Novartis said it couldn't provide Zolgensma to every eligible patient because of limited supply. The treatment is made at one plant in the U.S., though the company hopes to open up two more manufacturing sites within the next year.

Zolgensma is one of the first in a new wave of treatments known as gene therapies that promise to cure certain inherited diseases in a single treatment by providing a working copy of the faulty gene.

It went on sale in the U.S. earlier this year and is under review by regulators in Europe and Japan, with decisions expected next year. Once a country or region approves Zolgensma, Novartis will stop offering the lottery program there.

Despite its eye-catching price tag, Novartis has said Zolgensma has sold well since its launch in late May, overcoming concerns about whether insurers would cover the treatment as well as a data-manipulation scandal at the unit that makes it.

Novartis has defended Zolgensma's price on the grounds it costs less than Spinraza -- the rival treatment -- in the long term. Spinraza is a lifelong treatment that costs $750,000 for the first year and then $375,000 for each year thereafter.

Write to Denise Roland at Denise.Roland@wsj.com

(END) Dow Jones Newswires

December 19, 2019 10:19 ET (15:19 GMT)

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