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New Gene Therapy for SMA – ZOLGENSMA by Novartis

FDA approved 59 NMEs (New Molecular Entities) in 2018 and 12 up to now in 2019.2,3. A review of these approvals sheds light on what is happening in the Drug Development Scene.

Drug Development has changed in several ways in recent times. It is mostly going away from mainstream indications and towards specific ‘unmet needs’, rare diseases, orphan drugs, new classes such as Nibs (small molecule kinase inhibitors), Nabs (nanoparticle, Albumin based), Mibs (proteasome inhibitors), mAbs (monoclonal Antibodies based) and so on. The bottom line is that it is going away from general areas to highly specific, specialized areas.

USFDA has also started Patient Focused Drug Development program1. The purpose is to enhance patients’ consideration in the drug development process. More on this topic in another post.

One of the new FDA approvals has caused a lot of ripples in the market and medical circles. It is a drug developed by a Novartis company Avexis. It was mentioned briefly in my previous blog also.


The following excerpts are taken from the source below and describe in detail about it.

“Zolgensma: A Remarkable New Treatment, An ICER Analysis, And A Poorly Justified Price, ” Health Affairs Blog, June 18, 2019.DOI: 10.1377/hblog20190617.50453

“The drug is for the treatment of Spinal Muscular Atrophy (SMA), as disorder characterized by deterioration of motor neuron cells. It is a rare heritable condition caused by defects in both sets of alleles in the coding region for the survival motor neuron 1(SMN1) protein.

The clinical presentation of SMA is heterogenous, ranging from Type 1 disease that causes neuromuscular decline and death, typically before age 2, to Type 4, an adult onset condition associated with weakness and a normal life span. The incidence is small and fewer than 1,000 infants in the US are born each year with SMA4.

In May 2019, Novartis obtained USFDA approval for the gene therapy onasemnogene abeparvovec-xioi (Brand Name. ZOLGENSMA). Zolgensma transfects additional copies of the SMN1 gene into motor neuron cells using an adenovirus vector. In two single arm clinical trials, infants who had already developed symptomatic type 1 SMA and then received Zolgensma not only survived well past their predicted date of death, but experienced improvement in neuromuscular function. Zolgensma is approved for infants under the age of 2 with any type of SMA, whether symptomatic or not.


Zolgensma has a record shattering price; US$ 2.125 million for the single required infusion, payable in five equal annual installments. While SMA is rare, there are more gene therapies to come. One immutable drug pricing truth is that once a drug sets the pricing level in a category, future drugs will be priced in the same range or even higher. In other words, this category of treatments will get expensive for society soon. Drug costs threaten not only budgets but access: cost-related non-adherence is six times more likely to be reported by US patients than those of other high-income countries, where drug prices are generally lower.”

Pakistan vis-à-vis new, novel therapies

Our healthcare policies and drug registration policies have not been updated to include novel therapies. There is a long list of Biosimilar and Biological drugs which are pending due to absence of relevant regulations.

Pharmaceutical research has truly transformed. There is greater emphasis on targeted therapies, rather than generalized ones. Another important change is that the vanguard in new drug development is not the Big Pharma; it is small, research-only setups that are spearheading in this direction. Venture capital is fueling the research in new areas. As soon as a promising New Chemical Entity (NCE) is announced, it is taken up by one or the other Pharma company. Either the NCE is acquired, or in many cases, the entire company is acquired.

Pakistan Pharma landscape is now largely taken over by generics. While it provides economical options for treatment of a variety of diseases, it has no provision for novel therapies. Trying to develop generic versions of new research products is neither permitted nor possible technically. It is therefore important to facilitate registration of new research products. For exclusive products such as Zolgensma, some arrangement with the innovator company should be made by virtue of which the product could be made available on softer terms.

If such steps are not taken now, Pakistan will fall far behind in making new therapies available to its population.


  4. “Zolgensma: A Remarkable New Treatment, An ICER Analysis, And A Poorly Justified Price,” Health Affairs Blog, June 18, 2019.DOI: 10.1377/hblog20190617.50453

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