FDA Approves World's Most Expensive Treatment

Henrietta Brewer
May 29, 2019

The Food and Drug Administration approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms.

The regulatory approval allows Zolgensma to be used on babies with the deadliest form of the disease as well as ones whose lives could be impacted later.

"The job now is trying to negotiate with insurers that this would be a long-term savings", said Tiongson, who provided Zolgensma to patients under an expanded access program.


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. ICER's president, Dr. Steven D. Pearson, called the treatment's price "a positive outcome for patients and the entire health system".

But on Friday, 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.

Follow-up result presented by Novartis last month showed the 10 patients who continued with follow-up study maintained all motor function gains from treatment, such as sitting without support, and were alive at three to five years of age. That's where Novartis' Zolgensma comes into the picture. Gene and cell therapies leverage the biology to reverse diseases ranging from congenital blindness to pediatric leukemia. It is delivered by infusion. Dr Al-Azmi expressed his confidence that the Ministry of Health will provide the new drug whenever it becomes available, especially since the treatment is now being done through a drug that is valued at thousands of dollars and has not achieved the desired results. The genetic disease affects about one in every 10,000 births, with at least 50 percent to 70 percent diagnosed with Type I form. Instead of a one-time treatment, it must be given every four months.


Spinraza is priced at $750,000 for the first year, and $350,000 afterwards. The treatment targets a defective gene that weakens a child's muscles so dramatically that they become unable to move, and eventually unable to swallow or breathe. Novartis is looking into whether the death of one severely ill baby treated with Zolgensma was related to the therapy. Out-of-pocket costs for patients will vary based on insurance coverage.

Analysts forecast the gene therapy could hit peak annual sales of between $1.8 - $2.6bn. Life expectancy does not generally exceed 2 years. Roche is developing risdiplam, an oral drug, for the condition and plans to file for approval later this year.

Prior to FDA approval, Novartis mentioned that it could be pricing its new treatment somewhere between $1.5 million to $5 million.


On the possibility of the ministry providing the new drug for the disease of spinal muscular atrophy (SMA), Al-Bader explained that once the requirements and conditions of the purchase are met, the ministry will spare no efforts in providing it, as there is a system and mechanism governing this either by the medical departments or when the presentation to the control authorities. Jerry Mendell, a neurologist at Nationwide Children's.

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