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Zolgensma is a gene therapy medicine for treating spinal muscular atrophy, a serious condition of the nerves that causes muscle wasting and weakness.

Gene therapies like Zolgensma have such a high price tag because they have very expensive research costs. The cost to make them is also extremely high. The manufacturing and development cost required for the production of gene therapies is usually much higher compared with “traditional” drugs.

ZOLGENSMA is made up of a new, working SMN gene that is placed inside a delivery vehicle called a vector. The vector helps deliver the working SMN gene to motor neuron cells throughout the body. The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or AAV9.

Zolgensma is a gene therapy. This means it delivers genes (pieces of DNA). In clinical trials, children treated with Zolgensma show large improvements in motor function and life expectancy.

Meanwhile, Zolgensma has also recorded some blood system problems, including irregular platelet counts. An animal study of an experimental spinal-injection formula of Zolgensma previously also flagged a neuroinflammation problem.

As of November, judges had forced the Brazilian health system to pay for 102 Zolgensma treatments at an average cost equivalent to $1.6 million, ing to the Ministry of Health.

However, zolgensma is not a cure: Timing is critical. Although the treatments can rapidly increase SMN protein production after administration, some irreversible damage may have already occurred in the nervous system. This may happen even before a baby is born.

Under the deal with Novartis, the Brazilian government has agreed to pay for Zolgensma for no more than 250 babies over the next two years. If demand is higher than that, Novartis will chip in with 40 free treatments.