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Roche's (RHHBY) Evrysdi Gets FDA Nod for Label Extension
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Roche (RHHBY - Free Report) announced that the FDA approved a label extension of its spinal muscular atrophy (SMA) drug Evrysdi (risdiplam).
The drug is now approved for babies below two months with SMA.
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.
The recent regulatory nod was based on the interim RAINBOWFISH data, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones, such as sitting and standing with half walking after 12 months of treatment.
Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. Moreover, all infants were alive at the end of 12 months without permanent ventilation.
Evrysdi is now approved in the United States to treat SMA in children and adults of all ages. The medicine is administered orally everyday at home in liquid form or through a feeding tube.
The drug’s prescribing information is also updated to include the recent two-year pooled data from parts 1 and 2 of the FIREFISH study, which demonstrates long-term efficacy and safety in symptomatic infants with type 1 SMA along with the above-mentioned label expansion.
Roche collaborated with PTC Therapeutics (PTCT - Free Report) for the development of Evrysdi. The drug is marketed by Roche and was based on PTC Therapeutics’ splicing platform.
The drug enjoys a PRIME designation in the EU and an Orphan Drug status in the United States.
It is currently being evaluated in five multicentre studies in people with SMA.
Roche’s stock has lost 16.7% in the year so far against the industry’s growth of 6.5%.
Image Source: Zacks Investment Research
SMA is a severe, progressive neuromuscular disease that affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.
Other approved treatments include Biogen’s (BIIB - Free Report) Spinraza (nusinersen) and Novartis’ (NVS - Free Report) gene therapy Zolgensma
Biogen’s Spinraza generated sales of $472.5 million in the first quarter. The ASCEND study is evaluating the potential benefit of investigational higher dose of nusinersen in later-onset, non-ambulatory SMA patients, previously treated with Evrysdi.
Novartis’ Zolgensma is a one-time treatment of SMA. Sales were up 18% to $363 million in the first quarter of 2022, driven by an expanding access in Europe and the emerging growth markets.
Roche’s performance in the March quarter was encouraging on the back of its diagnostics division, which maintained its stellar performance on demand for conducting COVID-19 tests. The pharmaceuticals business was also stable and newer drugs continued to offset the decline in sales of the legacy drugs.
However, the outlook indicated that sales will decline in 2022 from the prior-year reported figure due to reduced demand for COVID-19 medicines and diagnostics.
Image: Bigstock
Roche's (RHHBY) Evrysdi Gets FDA Nod for Label Extension
Roche (RHHBY - Free Report) announced that the FDA approved a label extension of its spinal muscular atrophy (SMA) drug Evrysdi (risdiplam).
The drug is now approved for babies below two months with SMA.
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.
The recent regulatory nod was based on the interim RAINBOWFISH data, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones, such as sitting and standing with half walking after 12 months of treatment.
Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. Moreover, all infants were alive at the end of 12 months without permanent ventilation.
Evrysdi is now approved in the United States to treat SMA in children and adults of all ages. The medicine is administered orally everyday at home in liquid form or through a feeding tube.
The drug’s prescribing information is also updated to include the recent two-year pooled data from parts 1 and 2 of the FIREFISH study, which demonstrates long-term efficacy and safety in symptomatic infants with type 1 SMA along with the above-mentioned label expansion.
Roche collaborated with PTC Therapeutics (PTCT - Free Report) for the development of Evrysdi. The drug is marketed by Roche and was based on PTC Therapeutics’ splicing platform.
The drug enjoys a PRIME designation in the EU and an Orphan Drug status in the United States.
It is currently being evaluated in five multicentre studies in people with SMA.
Roche’s stock has lost 16.7% in the year so far against the industry’s growth of 6.5%.
Image Source: Zacks Investment Research
SMA is a severe, progressive neuromuscular disease that affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.
Other approved treatments include Biogen’s (BIIB - Free Report) Spinraza (nusinersen) and Novartis’ (NVS - Free Report) gene therapy Zolgensma
Biogen’s Spinraza generated sales of $472.5 million in the first quarter. The ASCEND study is evaluating the potential benefit of investigational higher dose of nusinersen in later-onset, non-ambulatory SMA patients, previously treated with Evrysdi.
Novartis’ Zolgensma is a one-time treatment of SMA. Sales were up 18% to $363 million in the first quarter of 2022, driven by an expanding access in Europe and the emerging growth markets.
Roche’s performance in the March quarter was encouraging on the back of its diagnostics division, which maintained its stellar performance on demand for conducting COVID-19 tests. The pharmaceuticals business was also stable and newer drugs continued to offset the decline in sales of the legacy drugs.
However, the outlook indicated that sales will decline in 2022 from the prior-year reported figure due to reduced demand for COVID-19 medicines and diagnostics.
Roche currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.