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SOUTH SAN FRANCISCO, California: – (WORK IN ARAR WIRE) -Roche Group (SIX: RO, ROG; OTCQX: RHHBY) member Genentech announced today that New England Journal of Medicine: (NEJM) releases data from FIREFISH Part 2, a key global study assessing the effectiveness and safety of Evrysdi®: (risdiplam) in children 1-7 months of age with type 1 spinal muscular atrophy (SMA). The study came to an end with the fact that 29% of newborns (12/41) sat for at least 5 seconds before 12 months without support *, which does not matter in the normal course of the disease. Evrysdi’s security in the FIREFISH Part 2 study was consistent with its well-known security profile.

“Without treatment, children with type 1 SMA are unlikely to survive after the age of two,” says Professor Lauren Serves, Ph.D. “Possible motor events, such as sitting, rolling, or swallowing, are essential elements that can help these children achieve optimal results with Euriside, potentially reducing the need for ventilation and increasing survival.”

At the time of data analysis, the mean duration of treatment with Eurisid was 15.2 months and the mean age was 20.7 months. At 12 months, 93% of newborns (38/41) were alive and 85% (35/41) were out of constant ventilation. The median age without treatment, natural death, or permanent ventilation was 13.5 months. Ninety percent (37/41) had an increase of CHOP-INTEND ** points by at least 4 points, 56% (23/41) had a score higher than 40; The average growth was 20 points.

In addition, the study encountered one of its secondary endpoints: 78% (32/41) of HINE-2 ***-responsive infants who rated motor performance through head control, sitting, voluntary grip, hitting, and rolling. crawl, stand և walk. Newborns are classified as HINE-2 responders if they show more motor signs of improvement than worsening.

“These data have been published New England Journal of Medicine: “The validated results of Part 1 of the FIREFISH study showed that Evrysdi can help children with SMA reach their destination for at least five seconds without support,” said Levi Garraway, MD, Ph.D. Global product development. “These results are further confirmed by recent 24-month data showing that Eurisdi has continued to improve motor function, doubling the number of possible newborns without support from the 12th month. We will continue to work closely with the և SMA community of governments. Bring as many people as possible to Eurisdi. ”

Evrysdi security in the FIREFISH Part 2 study was consistent with its well-known security profile. The most common adverse events were upper respiratory tract infection (68%), pneumonia (39%), pyrexia (39%), constipation (20%), diarrhea (10%), and maculopapular rash (10%). The most common adverse events were pneumonia (32%), bronchiolitis (5%), hypotension (5%), and respiratory failure (5%). Three newborns had fatal complications during the first 3 months of treatment. None of them were attributed to Eurisdi by the investigator.

In February 2021, the 12-month results of the discovery of the first dose of the FIREFISH study were published in the NEJM.

Genentech is leading Evrysdi Clinical Development in partnership with SMA և PTC Therapeutics.

* As assessed by the Bayley Child and Toddler Development Gross Engine (BSID-III)

** Philadelphia Children’s Hospital: Children’s test for neuromuscular disorders

*** Hammersmith Pediatric Neurological Examination 2:

About Eurisdi®: (risdiplam)

Evrysdi is a survival motor neuron 2 (SMN2) coupling modifier designed to treat SMA caused by mutations in the 5Q chromosome, leading to survival motor neuron (SMN) protein deficiency. Evrysdi is taken daily at home in liquid form by mouth or feeding tube.

Evrysdi is intended for the treatment of SMA by increasing and maintaining SMN protein production. SMN protein is found throughout the body և may be used to maintain the movement of healthy motor neurons:.

In 2019, Evrysdi was awarded an orphan appointment by the European Medicines Agency (EMA), PRIME was appointed by the EMA in 2018, and Orphan Drugs was awarded by the US Food and Drug Administration in 2017. Evrysdi has been established in 54 countries, represented in 33 countries.

Eurisdi is currently being evaluated in four multicenter trials for people with SMA.

  • FIREFISH (NCT02913482) – an open label, a two-part key clinical trial in infants with type 1 SMA. Part 1 was a dose-enhancement study in 21 infants whose primary purpose was to assess the safety profile of Eurisdi in infants and to determine the dose for the second part. SMA is treated for 2 years, followed by an extension of the open label. The registration of the second part was completed in November 2018. The main purpose of Part 2 was to evaluate efficacy as measured by the proportion of unassisted infants after 12 months of treatment as measured by Bailey’s Toddler Development-Third Edition (BSID-III) gross motor (defined as 5 seconds without support). ): The study reached its main point.

  • SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind, placebo-controlled study of people with 2 or 3 SMA types aged 2-25 years. In Part 1 (n = 51) the dose of confirming Part 2 is determined. Section 2 (n = 180) evaluates motor functions using the total score of Motor Function Measure 32 (MFM-32) for 12 months. The MFM-32 is a certified scale used in people with neurological disorders, including SMA, to assess good և gross motor function. The study reached its main point.

  • JEWELFISH (NCT03032172) – an open-label study designed to evaluate the safety, tolerability, pharmacokinetics (PK) և pharmacology (PD) of people who have received an SMA (Inclusion Criteria) from 6 months to 60 years of age. Approved SMA therapies. At least 90 days before receiving Eurisd. The study completed the collection (n = 174).

  • RAINBOWFISH (NCT03779334) – an open, unilateral, multicenter study examining the efficacy, safety, Evrysdi, PK և children (, n = 25), genetically diagnosed SMAs from birth to 6 weeks of age (first dose). no symptoms yet. The study is currently being recruited.

About SMA:

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in every 10,000 children է is the leading genetic cause of infant mortality. SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene, leading to SMN protein deficiency. This protein is found throughout the body:’s essential for the function of the nerves that control muscle: movements. Without it, nerve cells can not function properly, which over time leads to muscle weakness. Depending on the type of SMA, an individual ուժ’s ability to walk, eat or breathe can be significantly reduced or lost.

What is Eurisdi?

Evrysdi is a prescription drug used to treat spinal muscular atrophy (SMA) in adults երեխաների children over 2 months of age.

It is not known whether Evrysdi is safe և effective for children under 2 months.

Important safety information

  • Patients should inform their healthcare provider about all of their medical conditions before taking Evrysdi, including if they:

    • are pregnant or planning to become pregnant? If patients are pregnant or planning to become pregnant, they should consult their healthcare provider before taking this medicine. Eurisid can harm an unborn baby.

    • is a woman who can get pregnant.

      • Before patients start treatment with Evrysdi, their healthcare provider can check for pregnancy. Because Evrysdi can harm an unborn baby, his or her healthcare provider will decide if using Evrysdi is right for them during this time.

      • Patients should talk to their healthcare provider about birth control techniques that may be right for them. Patients should use birth control during treatment for at least 1 month after stopping Evrysdi

    • are adult males who plan to have children. If patients are concerned about this, they should definitely consult a healthcare professional

    • breastfeed or plan to breastfeed. It is unknown at this time what he will do after leaving the post. If patients plan to breastfeed, they should discuss with their healthcare provider the best way to breastfeed during treatment with Evrysdi.

  • Patients should inform their healthcare provider about all medications they have taken, including prescription, over-the-counter, vitamins, and herbal supplements. Patients should keep a list to show their healthcare provider և pharmacist when receiving new medicine

  • Patients should receive Evrysdi from a pharmacy as a liquid that can be given by mouth or through a feeding tube. The liquid solution is prepared by the patient’s pharmacist. If the medicine is in a bottle, do not use it. Patients should consult their pharmacist for replacement

  • Avoid getting into Evrysdi skin or eyes. If Eurisdine appears on the skin, wash the area with soap and water. If Eurisdine enters someone’s eyes, rinse their eyes with water

  • The most common side effects of Evrysdi include:

    • For SMAs starting later.

    • In case of SMA in childhood.

      • fever

      • diarrhea

      • rash

      • runny nose, sneezing, sore throat և cough (upper respiratory infection)

      • lung infection

      • constipation

      • vomiting

These are not all possible side effects of Evrysdi. For more information on the risks and benefits of Evrysdi հիվանդ, patients should contact their healthcare provider or pharmacist.

Patients can report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. Patients can also report side effects to Genentech by calling 1-888-835-2555.

See the full prescription for additional safety information.

Genentech Neurology

Neuroscience is a major area of ​​development for Genentech և Roche Research:. Our goal is to pursue innovative science to develop new treatments that will help improve the lives of people with chronic and potentially devastating diseases.

Genentech և Roche is investigating more than a dozen drugs for neurological disorders, including multiple sclerosis, neuromyelitis optic spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to meet the most challenging challenges in neurology today.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures, and manufactures commercial drugs for the treatment of patients with serious life-threatening illnesses. The Roche Group is headquartered in Southern San Francisco, California. For more information about the company, visit http://www.gene.com.

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