Latest news with #Fenfluramine
7NEWS
08-05-2025
- Health
- 7NEWS
From $10k to $31.70: Massive savings for severe epilepsy sufferers after drug added to PBS
A life-changing medication for those battling a rare form of epilepsy has been added to the Pharmaceutical Benefits Scheme (PBS). Fenfluramine, an additional treatment option for those with Dravet syndrome, is a ray of hope for families dealing with an already non-responsive epilepsy. By the time this drug is recommended to people with the syndrome, they are normally on at least two other medications. 'A lifelong condition': What is Dravet syndrome? Affecting one in 15,700 people, the Dravet syndrome is characterised by seizures that are hard to treat and significant developmental delays. The syndrome typically begins when children are 6 months to 12 months old, and the mortality rate is 15 per cent to 20 per cent due to SUDEP (sudden unexpected death in epilepsy) and related accidents such as drowning and infections. And it doesn't go away when they become adults. According to Professor Piero Perucca, 'while onset begins in infancy, it remains a lifelong condition, with reduced survival'. Adults look at 'persistent seizures, disabling cognitive, behavioural, and motor symptoms', with little effect from medication. Medication can help reduce symptoms, but it is not something that can be cured. 'Constant state of fight or flight' At just four months old, Raphael Lennard had his first seizure, which proved to be long and uncontrolled. 'The early days were really quite shocking and scary,' said Raphael's father, Ben Lennard. What followed were weekly hospital visits and a constant state of fear and uncertainty, only increasing during COVID-19, when restrictions at hospital meant only one parent could be with him at a time. Seizures were 10 minutes to 20 minutes long, with multiple rounds of drugs needed to stop them. 'It was happening every week — trying to make normal life work around that was just impossible, we were just in a constant state of fight or flight,' said Lennard. There were also developmental delays and reactions to other medications that suppressed appetite. 'There's still motor delays, he doesn't want to eat so he's not food driven like other kids, we can't reward him with chocolate because he just doesn't care,' Lennard said. 'Ungodly amounts': $10k every 60 days And the medication price before the PBS? $10,000 every 60 days. This is often after trying multiple other medications, and those with Dravet syndrome can look at being on at least three anti-epileptic drugs at a time. This doesn't even take into account the cost for specialists and hospital visits. Due to the frequency of seizures and time spent in hospital, Rachael was given compassionate access to fenfluramine, without the heavy price tag. 'It has been a game changer for us, the seizures are shorter, happening only four to six months,' said Lennard. 'For other families, just having this option at the start without having to go through all the different concoctions, without having to pay ungodly amounts — it's another weapon in the arsenal against epilepsy'. Impact on carers Because Dravet syndrome is diagnosed in infancy, the burden of care and advocacy often fall on parents and guardians. Not knowing when the next seizure will come can be terrifying for child and parent and the helplessness felt watching a loved one going through one. Both of Raphael's parents have had to cut their hours to part-time work as a result. Research shows that the burden of care can lead to persistent anxiety, poor mental health and sleep quality, loss of work and financial burdens. The care burden has also resulted in 55 per cent of carers accessing a mental health or family support service for the parents or siblings of the person living with the condition. It is a condition that affects the whole family. 'Stability in the chaos' With fenfluramine on the PBS, prescriptions will now cost a maximum of $31.70, which is huge drop from the previous price of $10,000. 'It could help so many families like ours find stability in the chaos,' Lennard said. Lennard said there were also benefits for the wider community as 'we're not accessing hospitals so much, we're not going to the GP every week to get new scripts'. 'It makes life a little bit easier.'
Yahoo
21-02-2025
- Health
- Yahoo
Seven things to know about epilepsy in children as new drug rolled out
Children in England living with a rare and severe form of epilepsy could soon benefit from a new drug being rolled out on the NHS. Fenfluramine has been recommended by the National Institute for Health and Care Excellence (NICE) for patients aged two and over with Lennox–Gastaut syndrome (LGS). LGS is a rare and severe form of epilepsy that starts in early childhood and is resistant to many treatments. It is thought that around 1-2% of the 60,000 children in England with epilepsy have LGS. The drug, the first non-cannabis-based treatment approved for this form of epilepsy, is taken as an oral liquid medicine daily and works by increasing the levels of serotonin in the brain to reduce seizures. Lisa Suchet, whose 10-year-old son has LGS, says the drug offers her son hope of "seizure freedom". "My son has suffered with seizures since he was five weeks of age, including drop seizures, which are the most devastating," she said. "They occur without warning, cause immediate collapse and the risk of head injury is high due to the sudden fall." Suchet says until now there have been so few effective drugs for seizure control. "Many don't work for all patients; many have debilitating side effects or interactions with other meds a patient might have to take," she explains. "That there is another option now available, which has shown effective results and minimal side effects is a huge comfort. It is essentially providing a chance of seizure freedom, which is essentially another chance at life really; for the patient and the family or carers who are impacted so significantly by this awful disease." Epilepsy is a neurological condition (affecting the brain and nervous system) where a person has a tendency to have seizures that start as a disruption of signals in the brain. Some children stop having seizures after a period of being on treatment, others, however, need treatment for long periods. The condition is more common than many people realise, with around one child in every 200 living with epilepsy. The NHS says that while there is sometimes a clear reason for epilepsy developing, for example a severe blow to the head, an infection of the brain such as meningitis, or brain damage due to lack of oxygen during a difficult birth, in other cases it isn't obvious. It's possible a child's epilepsy could be partly caused by their genes affecting how their brain works, as the NHS estimates around one in three people with epilepsy have a family member with it. Seizures are caused by a sudden burst of electrical activity in the brain, which temporarily disrupts the way messages are passed between brain cells. There are a number of different types of epileptic seizure, but the type a child has depends on which part of their brain is affected by the condition. The Epilepsy Society says there are two main types of seizure: focal seizures and generalised seizures. "Focal seizures start in only one side of the brain and generalised seizures affect both sides of the brain," the charity explains. "Generally, adults and children have the same types of seizure. However, some may be more common in childhood (for example, absence seizures which can be very brief and are often mistaken for 'daydreaming' or not paying attention)." The kind of symptoms a child experiences during a seizure can include: jerking of the body repetitive movements unusual sensations such as a strange taste in the mouth or a strange smell, or a rising feeling in the stomach. In some types of seizure, a child may be aware of what is happening. In other types, a child will be unconscious and have no memory of what happened afterwards. Some children may have seizures when they are sleeping (sometimes called "asleep" or 'nocturnal' seizures). The Epilepsy Society says some children's seizures can occur in response to triggers such as stress, excitement, boredom, missed medication, or lack of sleep. The charity recommends keeping a diary of your child's seizures, which can help to see if there are any patterns to when they happen and what may have triggered them. If a child experiences a tonic-clonic seizure (when their body goes stiff and the type of seizures most people associate with epilepsy) the Great Ormond Street Hospital recommends not trying to stop it. "Instead, move any furniture out of the way, try to loosen any tight clothing around the neck and stay with the child until the seizure has finished," the site explains. "As soon as uncontrolled movements finish, lay the child on their side in the recovery position." If a child has another type of seizure, it is recommended you sit them down quietly and stay with them until they are fully recovered and alert. Reassure the child calmly. The NHS recommends calling 999 for an ambulance if your child: is having a seizure for the first time has a seizure that lasts more than 5 minutes has lots of seizures in a row has breathing problems or has seriously injured themselves The number and type of seizures your child is having may change over time. But some children do outgrow their epilepsy by their mid to late teens. According to the Epilepsy Society this is called "spontaneous remission". There is currently no cure for epilepsy, but treatment can help most children with epilepsy have fewer seizures or stop having seizures completely. medicines called anti-epileptic drugs – these are the main treatment surgery to remove a small part of the brain that's causing the seizures a procedure to put a small electrical device inside the body that can help control seizures a special diet (ketogenic diet) that can help control seizures In very rare cases when medication does not help, and scans indicate a structural brain abnormality is the cause, surgery may be suggested. Read more about epilepsy: First UK child to have epilepsy device fitted in his brain has huge reduction in seizures (Independent, 2-min read) I had a seizure minutes before my final exams at uni (Yahoo Life UK, 11-min read) Epilepsy myths debunked as study reveals most Brits still believe misconceptions about the condition (Yahoo Life UK, 3-min read)
Yahoo
20-02-2025
- Health
- Yahoo
Seven things to know about epilepsy in children as new drug rolled out
Children in England living with a rare and severe form of epilepsy could soon benefit from a new drug being rolled out on the NHS. Fenfluramine has been recommended by the National Institute for Health and Care Excellence (NICE) for patients aged two and over with Lennox–Gastaut syndrome (LGS). LGS is a rare and severe form of epilepsy that starts in early childhood and is resistant to many treatments. It is thought that around 1-2% of the 60,000 children in England with epilepsy have LGS. See for yourself — The Yodel is the go-to source for daily news, entertainment and feel-good stories. By signing up, you agree to our Terms and Privacy Policy. The drug, the first non-cannabis-based treatment approved for this form of epilepsy, is taken as an oral liquid medicine daily and works by increasing the levels of serotonin in the brain to reduce seizures. Lisa Suchet, whose 10-year-old son has LGS, says the drug offers her son hope of "seizure freedom". "My son has suffered with seizures since he was five weeks of age, including drop seizures, which are the most devastating," she said. "They occur without warning, cause immediate collapse and the risk of head injury is high due to the sudden fall." Suchet says until now there have been so few effective drugs for seizure control. "Many don't work for all patients; many have debilitating side effects or interactions with other meds a patient might have to take," she explains. "That there is another option now available, which has shown effective results and minimal side effects is a huge comfort. It is essentially providing a chance of seizure freedom, which is essentially another chance at life really; for the patient and the family or carers who are impacted so significantly by this awful disease." Epilepsy is a neurological condition (affecting the brain and nervous system) where a person has a tendency to have seizures that start as a disruption of signals in the brain. Some children stop having seizures after a period of being on treatment, others, however, need treatment for long periods. The condition is more common than many people realise, with around one child in every 200 living with epilepsy. The NHS says that while there is sometimes a clear reason for epilepsy developing, for example a severe blow to the head, an infection of the brain such as meningitis, or brain damage due to lack of oxygen during a difficult birth, in other cases it isn't obvious. It's possible a child's epilepsy could be partly caused by their genes affecting how their brain works, as the NHS estimates around one in three people with epilepsy have a family member with it. Seizures are caused by a sudden burst of electrical activity in the brain, which temporarily disrupts the way messages are passed between brain cells. There are a number of different types of epileptic seizure, but the type a child has depends on which part of their brain is affected by the condition. The Epilepsy Society says there are two main types of seizure: focal seizures and generalised seizures. "Focal seizures start in only one side of the brain and generalised seizures affect both sides of the brain," the charity explains. "Generally, adults and children have the same types of seizure. However, some may be more common in childhood (for example, absence seizures which can be very brief and are often mistaken for 'daydreaming' or not paying attention)." The kind of symptoms a child experiences during a seizure can include: jerking of the body repetitive movements unusual sensations such as a strange taste in the mouth or a strange smell, or a rising feeling in the stomach. In some types of seizure, a child may be aware of what is happening. In other types, a child will be unconscious and have no memory of what happened afterwards. Some children may have seizures when they are sleeping (sometimes called "asleep" or 'nocturnal' seizures). The Epilepsy Society says some children's seizures can occur in response to triggers such as stress, excitement, boredom, missed medication, or lack of sleep. The charity recommends keeping a diary of your child's seizures, which can help to see if there are any patterns to when they happen and what may have triggered them. If a child experiences a tonic-clonic seizure (when their body goes stiff and the type of seizures most people associate with epilepsy) the Great Ormond Street Hospital recommends not trying to stop it. "Instead, move any furniture out of the way, try to loosen any tight clothing around the neck and stay with the child until the seizure has finished," the site explains. "As soon as uncontrolled movements finish, lay the child on their side in the recovery position." If a child has another type of seizure, it is recommended you sit them down quietly and stay with them until they are fully recovered and alert. Reassure the child calmly. The NHS recommends calling 999 for an ambulance if your child: is having a seizure for the first time has a seizure that lasts more than 5 minutes has lots of seizures in a row has breathing problems or has seriously injured themselves The number and type of seizures your child is having may change over time. But some children do outgrow their epilepsy by their mid to late teens. According to the Epilepsy Society this is called "spontaneous remission". There is currently no cure for epilepsy, but treatment can help most children with epilepsy have fewer seizures or stop having seizures completely. medicines called anti-epileptic drugs – these are the main treatment surgery to remove a small part of the brain that's causing the seizures a procedure to put a small electrical device inside the body that can help control seizures a special diet (ketogenic diet) that can help control seizures In very rare cases when medication does not help, and scans indicate a structural brain abnormality is the cause, surgery may be suggested. Read more about epilepsy: First UK child to have epilepsy device fitted in his brain has huge reduction in seizures (Independent, 2-min read) I had a seizure minutes before my final exams at uni (Yahoo Life UK, 11-min read) Epilepsy myths debunked as study reveals most Brits still believe misconceptions about the condition (Yahoo Life UK, 3-min read)
The Independent
20-02-2025
- Health
- The Independent
Hope for children with severe form of epilepsy as NHS rolls out new drug
Hundreds of children in England with a rare and severe form of epilepsy are set to benefit from a new drug being rolled out on the NHS. Fenfluramine has been recommended by the National Institute for Health and Care Excellence (Nice) for patients aged two and over with Lennox–Gastaut syndrome (LGS). It is the first non-cannabis-based treatment approved for this form of epilepsy, according to NHS officials. For children and families living with Lennox–Gastaut syndrome, every day can be challenging, facing unpredictable and life-limiting seizures, and this new treatment option on the NHS will now offer new hope, giving many the chance for greater stability and a better quality of life Professor Stephen Powis The drug is taken as an oral liquid medicine daily and works by increasing the levels of serotonin in the brain to reduce seizures. LGS is a rare and severe form of epilepsy that starts in early childhood and is resistant to many treatments. It is thought that around 1-2% of the 60,000 children in England with epilepsy have LGS. Professor Stephen Powis, national medical director at NHS England said the recommendation offers patients and their families 'new hope'. 'For children and families living with Lennox–Gastaut syndrome, every day can be challenging, facing unpredictable and life-limiting seizures, and this new treatment option on the NHS will now offer new hope, giving many the chance for greater stability and a better quality of life. 'It's fantastic news for hundreds of families that there's a proven, evidence-based new medicine that can be taken at home to help control and reduce their child's seizures and, for example, lower the risk of them experiencing injuries and needing to go to hospital. 'Fenfluramine will offer a vital alternative for those who can't tolerate existing cannabis-based treatment and the fast-tracking of this treatment to be available from today is another example of the NHS' commitment to ensuring access to the best therapies that deliver real benefits to patients as well as value for the taxpayer.' Previously, the only Nice-recommended treatment for LGS was the cannabidiol Epidyolex with the medication clobazam, which was approved in 2019. Clinical trials suggest fenfluramine can slash the frequency of seizures that cause patients to lose consciousness and muscle control, known as drop seizures, by 26.5% on average. NHS England is using its Innovative Medicines Fund to fast-track the treatment to eligible patients. It is essentially providing a chance of seizure freedom, which is essentially another chance at life really; for the patient and the family or carers who are impacted so significantly by this awful disease Lisa Suchet, whose 10-year-old son has LGS Helen Knight, director of medicines evaluation at Nice, added: 'The often distressing and life-limiting nature of this very difficult-to-control epilepsy means that any new treatment options are particularly welcome. 'In recommending fenfluramine, the independent committee took into account the rarity and severity of Lennox Gastaut syndrome, the significant impact it has on the quality of life of people with the condition and their families and carers, and the high need for effective treatments, particularly for people who aren't able to take cannabidiol with clobazam.' Lisa Suchet, whose 10-year-old son has LGS, welcomed the recommendation. 'My son has suffered with seizures since he was five weeks of age, including drop seizures, which are the most devastating,' she said. 'They occur without warning, cause immediate collapse and the risk of head injury is high due to the sudden fall. 'There are so few effective drugs for seizure control. Many don't work for all patients; many have debilitating side effects or interactions with other meds a patient might have to take. 'That there is another option now available, which has shown effective results and minimal side effects is a huge comfort. 'It is essentially providing a chance of seizure freedom, which is essentially another chance at life really; for the patient and the family or carers who are impacted so significantly by this awful disease.'
Sky News
20-02-2025
- Health
- Sky News
New drug offers hope to children with rare form of epilepsy
Why you can trust Sky News Hundreds of children with a rare and severe form of epilepsy are hoping a new drug being rolled out on the NHS may provide a better quality of life. Fenfluramine has been recommended by the National Institute for Health and Care Excellence (NICE) for patients aged two and over with Lennox-Gastaut syndrome (LGS). LGS is a rare and severe form of epilepsy that starts in early childhood and is resistant to many treatments. It is thought that between one and two percent of the 60,000 children in England with epilepsy have LGS. Fenfluramine is the first non-cannabis-based treatment approved for LGS, according to NHS officials. The drug is taken as an oral liquid medicine daily and works by increasing the levels of serotonin in the brain to reduce seizures. Clinical trials suggest fenfluramine can cut the frequency of seizures that cause patients to lose consciousness and muscle control, known as drop seizures, by 26.5% on average. Previously, the only NICE-recommended treatment for LGS was the cannabidiol Epidyolex with the medication clobazam, which was approved in 2019. Professor Stephen Powis, national medical director at NHS England said the latest recommendation offers patients and their families "new hope". "For children and families living with Lennox-Gastaut syndrome, every day can be challenging, facing unpredictable and life-limiting seizures, and this new treatment option on the NHS will now offer new hope, giving many the chance for greater stability and a better quality of life. "It's fantastic news for hundreds of families that there's a proven, evidence-based new medicine that can be taken at home to help control and reduce their child's seizures and, for example, lower the risk of them experiencing injuries and needing to go to hospital," he said. What is epilepsy? Epilepsy is a condition that affects the brain and causes frequent seizures. It can start at any age, but usually starts in childhood or in people over 60. Seizures are bursts of electrical activity in the brain that temporarily affect how it works. They can cause a wide range of symptoms. Symptoms include: • uncontrollable jerking and shaking, known as a "fit"; • losing awareness; • becoming stiff; • strange sensations, such as a "rising" feeling in the belly, unusual smells or tastes, and a tingling feeling in your limbs; • collapsing. Seizures can be triggered in numerous ways including flashing lights, sleep deprivation, misuse of drugs or missing medications. The condition affects more than 600,000 people in the UK. NHS England is using its Innovative Medicines Fund to fast-track the treatment to eligible patients. Helen Knight, director of medicines evaluation at NICE, added: "The often distressing and life-limiting nature of this very difficult-to-control epilepsy means that any new treatment options are particularly welcome. "In recommending fenfluramine, the independent committee took into account the rarity and severity of Lennox Gastaut syndrome, the significant impact it has on the quality of life of people with the condition and their families and carers, and the high need for effective treatments, particularly for people who aren't able to take cannabidiol with clobazam."
