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On February 25, 2021, the "New England Journal of Medicine" published the data results of the first part of the FIREFISH study of Risdiplam in children with type 1 SMA, and this article is hereby collated and reported.
Dose exploration data of Risdiplam in the treatment of children with type 1 SMA, Part 1 data of the FIREFISH study showed that 90% (19/21) of the children survived without permanent ventilation after 12 months of Risdiplam treatment.
The high-dose cohort Among them, 41% (7/17) of the children achieved sitting alone for at least 5 seconds without support, which is a key sports milestone that is not usually seen in the natural course of the disease.
Moreover, no children lost their swallowing function, and 86% (18/21) of the children were able to take food by mouth (with or without a feeding tube).
The study also found that in the high-dose cohort, 9 infants (53%, 9/17) achieved head erection, and 1 infant (6%, 1/17) achieved standing.
The SMN level of children with SMA was higher than the baseline 1.
9 times (median value).
In addition, the researchers used the CHOP-INTEND scale to assess the motor function of children with type 1 SMA.
The results showed that 11 children (52%, 11/21) had a total CHOP-INTEND score ≥ 40 points (range 0-64, the higher the score, the better the motor function).
Risdiplam increases the level of SMN protein in the whole body.
Risdiplam is a SMN2 gene splicing modifier, which can penetrate the blood-brain barrier, distribute throughout the body, increase the level of central SMN protein, and increase the protein level of peripheral organs.
It can act to maintain motor neuron health and exercise Ability is normal.
Risdiplam is a liquid preparation that can be administered orally at home or via a feeding tube every day.
It is the world's first oral disease correction drug approved by the FDA for the treatment of SMA.The FDA approved Risdiplam for the treatment of SMA patients 2 months of age and older.
Risdiplam was recognized by the European Medicines Agency (EMA) Priority Medicine (PRIME) program in 2018, FDA orphan drug certification in 2017, and EMA orphan drug certification in 2019.
In June 2020, the National Medical Products Administration of China officially granted Risdiplam the priority review qualification.
Currently, Risdiplam has been approved in 7 countries and submitted applications in 57 countries, including 27 EU countries, Norway and Iceland.
Risdiplam clinical trials extensively cover SMA patients Risdiplam has carried out 4 international multi-center clinical trials in SMA patients (Figure 1), including the RAINBOWFISH study, FIREFISH study, SUNFISH study, and JEWELFISH study.
The presymptomatic SMA, type 1 SMA, 2/ Patients with type 3 SMA and previously treated SMA patients are included.
The research data collected so far show that Risdiplam is effective and safe for SMA patients.
Figure 1 Risdiplam clinical trials extensively cover SMA patients.
Follow-up preview of other key clinical data of Risdiplam.
On March 15-18, the 2021 MDA conference will be held online.
During the conference, the latest developments in the field of SMA and Risdiplam will be reported, including "Evrysdi® (risdiplam) Experiences: Explore the Science and Hear From People Living With SpinalMuscular Atrophy", "SUNFISH Part 2: 24-Month Efficacyand Safety of Risdiplam in Patients with Type 2 or Non-Ambulant Type 3 SpinalMuscular Atrophy (SMA)".
stay tuned! References: 1.
Giovanni Baranello et al.
Risdiplam in Type 1 Spinal Muscular Atrophy.
N Engl J Med.
2021 Feb 24.
2.
https://clinicaltrials.
gov/ct2/show/results/NCT02913482 Source: SMA New Voice
Dose exploration data of Risdiplam in the treatment of children with type 1 SMA, Part 1 data of the FIREFISH study showed that 90% (19/21) of the children survived without permanent ventilation after 12 months of Risdiplam treatment.
The high-dose cohort Among them, 41% (7/17) of the children achieved sitting alone for at least 5 seconds without support, which is a key sports milestone that is not usually seen in the natural course of the disease.
Moreover, no children lost their swallowing function, and 86% (18/21) of the children were able to take food by mouth (with or without a feeding tube).
The study also found that in the high-dose cohort, 9 infants (53%, 9/17) achieved head erection, and 1 infant (6%, 1/17) achieved standing.
The SMN level of children with SMA was higher than the baseline 1.
9 times (median value).
In addition, the researchers used the CHOP-INTEND scale to assess the motor function of children with type 1 SMA.
The results showed that 11 children (52%, 11/21) had a total CHOP-INTEND score ≥ 40 points (range 0-64, the higher the score, the better the motor function).
Risdiplam increases the level of SMN protein in the whole body.
Risdiplam is a SMN2 gene splicing modifier, which can penetrate the blood-brain barrier, distribute throughout the body, increase the level of central SMN protein, and increase the protein level of peripheral organs.
It can act to maintain motor neuron health and exercise Ability is normal.
Risdiplam is a liquid preparation that can be administered orally at home or via a feeding tube every day.
It is the world's first oral disease correction drug approved by the FDA for the treatment of SMA.The FDA approved Risdiplam for the treatment of SMA patients 2 months of age and older.
Risdiplam was recognized by the European Medicines Agency (EMA) Priority Medicine (PRIME) program in 2018, FDA orphan drug certification in 2017, and EMA orphan drug certification in 2019.
In June 2020, the National Medical Products Administration of China officially granted Risdiplam the priority review qualification.
Currently, Risdiplam has been approved in 7 countries and submitted applications in 57 countries, including 27 EU countries, Norway and Iceland.
Risdiplam clinical trials extensively cover SMA patients Risdiplam has carried out 4 international multi-center clinical trials in SMA patients (Figure 1), including the RAINBOWFISH study, FIREFISH study, SUNFISH study, and JEWELFISH study.
The presymptomatic SMA, type 1 SMA, 2/ Patients with type 3 SMA and previously treated SMA patients are included.
The research data collected so far show that Risdiplam is effective and safe for SMA patients.
Figure 1 Risdiplam clinical trials extensively cover SMA patients.
Follow-up preview of other key clinical data of Risdiplam.
On March 15-18, the 2021 MDA conference will be held online.
During the conference, the latest developments in the field of SMA and Risdiplam will be reported, including "Evrysdi® (risdiplam) Experiences: Explore the Science and Hear From People Living With SpinalMuscular Atrophy", "SUNFISH Part 2: 24-Month Efficacyand Safety of Risdiplam in Patients with Type 2 or Non-Ambulant Type 3 SpinalMuscular Atrophy (SMA)".
stay tuned! References: 1.
Giovanni Baranello et al.
Risdiplam in Type 1 Spinal Muscular Atrophy.
N Engl J Med.
2021 Feb 24.
2.
https://clinicaltrials.
gov/ct2/show/results/NCT02913482 Source: SMA New Voice
