2 S'porean couples are seeking a S$2.4 million cure for their babies. Why is it so expensive?

Over the past few days, two babies have made headlines in Singapore.

Baby Ginny is five months old, with large round eyes and a head full of hair.

Baby Faziq is nine months old and has a smile that could melt a heart of stone.

Both also suffer from severe Spinal Muscular Atrophy (SMA), a rare and debilitating disease. Both are searching for a cure.

The only catch?

The cure costs S$2.4 million.

But why is it so expensive?

What is Spinal Muscular Atrophy?

SMA is an inherited disease that destroys motor neurons — the cells in your body that control movement, swallowing, and breathing.

It's exceedingly rare, occurring just once in every 10,000 to 12,000 births.

In Singapore, this translates to around two to three new cases each year.

The disease is caused by mutations or deletions in the SMN1 gene, explained Stacey Tay, senior consultant of paediatric neurology at the National University Hospital (NUH).

Without this gene, the muscles in the body weaken and waste away over time.

Once symptoms begin, they are irreversible and incurable.

This eventually leads to functional decline and, often, premature death.

An elusive cure

This was what happened to Baby Faziq's older brother, Faris.

Faris was born in 2016 and diagnosed with SMA at around six months old.

Despite the clinical team's efforts, he passed away in 2022.

He was not yet six years old.

Things have changed since then. Today, there are three treatments available for SMA, said Tay.

The first is Risdiplam, a daily oral therapy that targets the SMN2 gene (similar to the faulty SMN1 gene).

The second is Nusinersen, which is functionally the same but must be administered into the spine every four months.

Both must be taken in the long term.

The final treatment available is Zolgensma, a one-time gene therapy that replaces the faulty gene entirely.

It was approved in Singapore in 2023 — one year after Faris's death.

The differences

Both Ginny and Faziq are now on Risdiplam, which costs around S$4,800 a month.

But they're hoping to raise funds for Zolgensma, which was once the world's most expensive drug (it has since been superseded by newer gene therapies).

Baby Ginny has thankfully reached her fundraising goal. Baby Faziq's fundraiser is currently at S$1.45 million.

Tay explained that Zolgensma has several advantages over Risdiplam and Nusinersen.

Both the latter therapies target the SMN2 gene, not the faulty SMN1 gene itself.

The two genes are 99 per cent similar.

But SMN2 only has about 10 to 15 per cent of the function of the SMN1 gene.

The more copies of SMN2 gene a person has, the milder their symptoms.

But it slows the progression of the disease and does not stop it entirely.

In contrast, Zolgensma replaces the faulty SMN1 gene, and hence, stops disease progression entirely.

Studies have shown that when given to patients with the most severe form of SMA, 92 per cent achieved head control, and 17 per cent were able to walk independently.

66.7 per cent were also able to stop feeding support.

Results are even better when patients are given the gene therapy before symptoms begin.

In such cases, survival rates — without respiratory and feeding support — reach 100 per cent.

Not covered

NUH has prescribed Zolgensma to several patients since 2019, said Tay.

That was when it was first approved by the U.S. Food and Drug Administration (FDA).

At the time, it was only available in Singapore through special access.

But it's since been fully approved by the Health Sciences Authority (HSA).

Even so, it remains unsubsidised by the government.

In comparison, Risdiplam is subsidised by the government and covered under MediShield Life and Medisave.

Risdiplam was added to the subsidy list in August 2024. It was the first — and so far, only — SMA treatment that is eligible for government subsidies in Singapore.

So then why not Zolgensma?

In response to Mothership's queries, a Ministry of Health (MOH) spokesperson said that Risdiplam has been evaluated to be both clinically- and cost-effective.

"However, [Zolgensma] is not eligible for subsidies, MediShield Life and MediSave coverage, as it is not cost-effective compared to Risdiplam.

This means that the additional cost is not worth any marginal benefits."

MOH said that it remains committed to ensuring that effective treatments for SMA are accessible to patients.

It will continue to review the Cell, Tissue, and Gene Therapy Product (CTGTP) List as new evidence becomes available.

The what list?

In Singapore, there's a list of medications that are eligible for government subsidies.

What goes on the list depends on recommendations by the Drug Advisory Committee, which are then assessed and accepted by MOH.

In October 2024, MOH launched a new list of drugs — the CTGTP List.

It's an MOH list of cell, tissue, and gene therapy products that have been assessed to be clinically and cost effective, and as such are eligible for government subsidies.

In an MOH statement at the time of its launch, it said:

"While CTGTPs have the potential to transform healthcare and treat serious diseases, they have high upfront costs.

Without financing support, patients may not be able to access these potentially effective treatments."

However, this must be done in a sustainable manner, given the high cost of CTGTPs and the uncertainty around their long-term effectiveness, MOH said.

"Hence, the government has introduced a pilot financing framework to focus support only on CTGTPs that have been assessed to be both clinically effective and cost-effective."

Gene therapies are expensive

Currently, there are only two products on MOH's CTGTP List.

The first is Kymriah, which is used to treat specific blood cancers.

The second is Yescarta, a cancer drug which was added in September 2025.

Both cost around S$481,000 per treatment. Expensive, but a fraction of what Zolgensma costs.

Tay explained that gene therapies tend to be expensive because of the high research and development costs involved.

They are also often complex to manufacture.

Furthermore, when they target rare diseases — like Zolgensma — the costs can run even higher.

"Because of the small number of patients, the costs cannot be spread over millions of patients as in the case of other traditional drugs," she said.

At S$2.4 million per dose, Zolgensma is decidedly out of reach for most people, including Ginny and Faziq's families.

Novartis, the company that owns it, says it is "consistently priced based on the value it provides to patients, caregivers, and health systems".

It added that it reduces the burden of SMA by replacing repeated, lifelong therapies with a single treatment.

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