FDA approval for small molecule drug bodes well for Longevity

Roche, the world’s largest biotech company, has won approval from the FDA for an SMA drug.

The race to take a therapy from lab to patient for the treatment of Spinal Muscular Atrophy (SMA) now has a third runner. Roche’s SMA drug Evrysdi has won approval from the FDA in what is a landmark approval for patients, given that this is the first and only medicine for SMA that patients are able to take at home [1]. 

Longevity.Technology: Half of speciality medicine sales can be attributed to small molecule applications [2], and Roche’s Risdiplam (marketed as Evrysdi) has paradigm-shifting potential for the treatment of SMA, which not only shortens life, but makes it painful and debilitated.

While Evysdi now has approval, there are many other small molecule treatments – drugs which are easily able to enter cells because of their low molecular weight – currently coming through the research and clinical trial pipeline which are designed to improve health and lifespan by targeting conditions which typically affect the elderly or which cause symptoms which are more severe in older patients.

While many people with SMA are diagnosed as a young child, the condition also affects older adults and the elderly (Type 4, adult-onset SMA). SMA, which is a genetic condition, causes progressive muscle weakness through atrophy, scoliosis, muscle tremors and twitching. Over time, this can lead to a loss of control over walking, neck control, speech and even swallowing and breathing – profound obstacles to both lifespan and healthspan.

Up until now, however, treatment for SMA has been limited and expensive. In 2016, the drug Spinraza was approved, followed by Zolgensma three years later. However, both drugs have very high price tags. Zolgensma is one of the most expensive drugs in the world with a price tag of $2.1m, while Spinraza costs $750,000 for the first year and $375,000 for subsequent yearly treatment. Evrysdi, however, has made a quick migration from trials to being available to patients and will have an annual price of $340,000 [3].

Democratisation of healthcare through large patient populations is something the CEO of Juvenescence, Greg Bailey spoke to us about recently. Evrysid’s lower price point and at home administration show that big pharma may well be aware of this shift in drug positioning.

The Evrysdi approval was given following two clinical trials which showed clinically-meaningful improvements in motor function across participants with various ages and levels of disease severity, including Types 1, 2 and 3 SMA which affect different age groups. The trials included a placebo-controlled trial which incorporated adults with Types 2 and 3 SMA. 


The Evysdi approval was given following two clinical trials which showed clinically-meaningful improvements in motor function across participants with various ages and levels of disease severity.


The drug is designed to work by increasing production of the survival of motor neuron protein, which is vital to maintain healthy motor neurons and movement. It is administered daily at home in liquid form orally or via a feeding tube.

A recent review, for example, revealed 18 amyloid-targeting disease modification trials involving small molecule treatments [4]. There are also a number of assessments underway in a bid to see if certain small molecule drugs can be repurposed for the treatment of Covid-19 [5].

Speaking about the latest FDA approval for Evrysdi, Kenneth Hobby, president of Cure SMA, said: “Throughout their lives, many people with SMA may lose their ability to perform critical movements, which can impact the ability to independently participate in aspects of daily life and even be life altering. The approval of Evrysdi is an eagerly awaited milestone for our community.” 

[1] https://www.roche.com/media/releases/med-cor-2020-08-10b.htm
[2] https://themedicinemaker.com/manufacture/why-small-molecules-are-still-a-big-deal
[3] https://bit.ly/3anbwPR
[4] https://www.sciencedirect.com/science/article/pii/S2352873719300290
[5] https://pubs.acs.org/doi/10.1021/acs.oprd.0c00233

Image credit: JHVEPhoto / Shutterstock

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