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Roche’s SMA treatment Evrysdi approved by European Commission

Published on 30/03/21 at 11:28am
Roche building

The European Commission has granted marketing authorisation to Roche and PTC Therapeutics’ Evrysdi, for the treatment of patients aged 2 months or older with spinal muscular atrophy (SMA).

The approval comes following two studies – one in infants with symptomatic Type 1 SMA, and another in children and young adults with Type 2 or 3 SMA.

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies, and when untreated is the leading genetic cause of infant mortality. An individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost, depending on the type of SMA they have.

The FIREFISH study, in 41 patients with Type 1 SMA, found a significant proportion of infants able to sit without support after 12 months of treatment. The SUNFISH study, in 180 patients aged 2 to 25 years old with Type 2 or 3 SMA, showed increased motor function in those on Evrysdi after 12 months, as compared to those on a placebo.

More than 3,000 patients have now been treated with Evrysdi in clinical trials, compassionate use programs and real-world settings, with patients ranging from birth to over 70 years old including those previously treated with other SMA therapies. 

The new treatment has entered into a competitive market, with Biogen’s Spinraza and Novartis’s Zolgensma both already approved in many countries, including the US and most of Europe (Evrysdi was granted FDA approval itself last year.)

However, Zolgensma is the most expensive drug in the world, coming in at £1.79 million for a single dose, whereas Spinraza, which has to be given by lumbar puncture every year throughout a patient’s life, costs £450,000 for the first year of treatment, with every following year costing £225,000.

Evrysdi, a once-daily oral treatment, is priced depending on patient weight. It costs roughly $100,000 (£72,595) a year for infants, and up to $340,000 (£246,823) a year once the patient weighs 44 pounds or more (around the weight of a six-year-old).

Stuart W Peltz PhD, CEO of PTC Therapeutics, said: ”We’re thrilled that Evrysdi, with proven efficacy and safety, has been rapidly approved in Europe so that the SMA community will now have access to a much needed convenient at-home treatment.

”Evrysdi was discovered using our novel splicing technology and is an incredibly innovative drug that was the first in demonstrating that a small molecule can selectively and specifically modulate splicing to treat the underlying cause of this devastating disease. We're excited to see this therapy become more available to patients around the world."

Jack Goddard

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