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Roche RHHBY announced that the European Commission (EC) has approved its spinal muscular atrophy drug (SMA) Evrysdi.
The drug is approved for the treatment of 5q SMA in patients who are two months older and above with a clinical diagnosis of SMA type 1, type 2 or type 3 or with one to four SMN2 copies.
SMA is a severe, progressive neuromuscular disease that causes muscle weakness and progressive loss of movement affecting one in 10,000 babies and is the leading genetic cause of infant mortality.
The regulatory nod was based on data from two clinical studies, namely FIREFISH in symptomatic type 1 infants, aged from two months to seven months and SUNFISH in symptomatic type 2 and 3 children and adults, aged between two years and 25 years. Evrysdi demonstrated a favorable efficacy and safety profile in both studies.
An approval was in the cards as the Committee for Medicinal Products for Human Use (CHMP) gave a positive recommendation for the same in February 2021.
The review was completed under the accelerated assessment pathway for medicines.
We note that Evrysdi was granted a PRIME designation by the European Medicines Agency (EMA) in 2018 and an Orphan Drug Designation in 2019.
Evrysdi can be administered daily at home in liquid form through mouth or by feeding tube.
Roche is developing Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics PTCT. It is already approved in the United States.
The approval will strengthen Roche’s neuroscience portfolio. While pricing will play a key role in the drug’s uptake, competition is stiff in the SMA market from the likes of Biogen’s BIIB Spinraza and Novartis’ NVS gene therapy Zolgensma.
Shares of Roche have declined 8.2% so far this year against the industry’s increase of 0.7%.
Roche is a leader in the oncology space. The company is looking to diversify and strengthen its portfolio beyond oncology and explore neuroscience among others. Approval of new drugs in other therapeutic areas will enable the company to establish a foothold in these spaces.
Roche currently carries a Zacks Rank #5 (Strong Sell).
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