Roche (RHHBY - Free Report) announced that the European Commission (“EC”) had approved the label expansion for Evrysdi (risdiplam) to treat spinal muscular atrophy (“SMA”) in babies aged under two months old.
Before the label expansion, Evrysdi was approved by the EC in 2021 to treat SMA patients aged two months or older. Following the latest approval, Evrysdi is approved in the European Union (EU) across all age groups, including newborns.
The approval was expected as last month the European Medicines Agency’s (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) recommended approving Evrysdi for use in infants from birth to below two months.
The CHMP and EC decisions are based on a positive interim analysis of data from the ongoing RAINBOWFISH study in pre-symptomatic babies with type 1 SMA from birth to six weeks.
Evrysdi is already approved in the United States to treat SMA in patients across all age groups.
Roche is currently evaluating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the phase II/III MANATEE study for treating SMA in patients aged between two and ten years. This study is currently enrolling participants.
Shares of Roche have lost 6.3% year to date against the industry’s 7.5% growth.
Currently, Roche’s Evrysdi is the preferred choice of therapy for SMA treatment. Unlike other marketed treatments like Biogen’s (BIIB - Free Report) Spinraza and Novartis’ (NVS - Free Report) Zolgensma, which take an invasive approach to treating patients, the Roche drug is administered as an oral solution. Also, Evrysdi is cost-efficient when compared to the other two marketed treatments.
Biogen’s Spinraza was the first marketed drug approved for SMA treatment. The drug received FDA approval in 2016 and EC approval in 2017. Biogen licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals (IONS - Free Report) . In return, Ionis receives royalties on annual worldwide net sales of Spinraza.
Evrysdi and Spinraza also compete with Novartis’ Zolgensma, the first and currently the only FDA-approved one-time gene therapy for SMA treatment. However, the Novartis gene therapy can currently be administered to SMA patients under two years and is also quite expensive. Also, recent reports suggest that patients receiving Novartis’ Zolgensma still require further treatment with other SMA drugs.
In June, Ionis/Biogen reported new interim results from a phase IV study highlighting Spinraza’s benefits in infants and toddlers with SMA who had previously received Zolgensma. The results showed that treatment with Spinraza improved motor function in most study participants.
Zacks Rank
Roche currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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Roche (RHHBY - Free Report) announced that the European Commission (“EC”) had approved the label expansion for Evrysdi (risdiplam) to treat spinal muscular atrophy (“SMA”) in babies aged under two months old.
Before the label expansion, Evrysdi was approved by the EC in 2021 to treat SMA patients aged two months or older. Following the latest approval, Evrysdi is approved in the European Union (EU) across all age groups, including newborns.
The approval was expected as last month the European Medicines Agency’s (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) recommended approving Evrysdi for use in infants from birth to below two months.
The CHMP and EC decisions are based on a positive interim analysis of data from the ongoing RAINBOWFISH study in pre-symptomatic babies with type 1 SMA from birth to six weeks.
Evrysdi is already approved in the United States to treat SMA in patients across all age groups.
Roche is currently evaluating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the phase II/III MANATEE study for treating SMA in patients aged between two and ten years. This study is currently enrolling participants.
Shares of Roche have lost 6.3% year to date against the industry’s 7.5% growth.
Currently, Roche’s Evrysdi is the preferred choice of therapy for SMA treatment. Unlike other marketed treatments like Biogen’s (BIIB - Free Report) Spinraza and Novartis’ (NVS - Free Report) Zolgensma, which take an invasive approach to treating patients, the Roche drug is administered as an oral solution. Also, Evrysdi is cost-efficient when compared to the other two marketed treatments.
Biogen’s Spinraza was the first marketed drug approved for SMA treatment. The drug received FDA approval in 2016 and EC approval in 2017. Biogen licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals (IONS - Free Report) . In return, Ionis receives royalties on annual worldwide net sales of Spinraza.
Evrysdi and Spinraza also compete with Novartis’ Zolgensma, the first and currently the only FDA-approved one-time gene therapy for SMA treatment. However, the Novartis gene therapy can currently be administered to SMA patients under two years and is also quite expensive. Also, recent reports suggest that patients receiving Novartis’ Zolgensma still require further treatment with other SMA drugs.
In June, Ionis/Biogen reported new interim results from a phase IV study highlighting Spinraza’s benefits in infants and toddlers with SMA who had previously received Zolgensma. The results showed that treatment with Spinraza improved motor function in most study participants.
Zacks Rank
Roche currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Zacks Names #1 Semiconductor Stock
It's only 1/9,000th the size of NVIDIA which skyrocketed more than +800% since we recommended it. NVIDIA is still strong, but our new top chip stock has much more room to boom.
With strong earnings growth and an expanding customer base, it's positioned to feed the rampant demand for Artificial Intelligence, Machine Learning, and Internet of Things. Global semiconductor manufacturing is projected to explode from $452 billion in 2021 to $803 billion by 2028.
See This Stock Now for Free >>
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