Biomarin gene therapy for Hereditary Angioedema
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MHRA Grants HAE Patients Early Access to BioCryst’s Berotralstat in United Kingdom
RESEARCH TRIANGLE PARK, N.C. – October 30, 2020 – BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
Under the EAMS, hereditary angioedema (HAE) patients in the UK aged 12 years and older can gain access to berotralstat for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the European Commission (EC).
HAE is a serious, and potentially life-threatening, rare genetic illness characterised by periodic episodes of acute swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals and/or extremities.
Medicines included in the EAMS are those that have a high unmet need, are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options, and are likely to offer significant advantage over methods currently used in the UK. Under the scheme, the MHRA provides a scientific opinion on the benefit-risk balance of the medicine, based on the data available when the EAMS submission was made.
“There are many patients in the UK that don’t have a realistic option for effective HAE prophylaxis. The addition of berotralstat through the EAMS will bring a much needed option for HAE patients suffering with this debilitating disease,” said Dr. Sorena Kiani, Consultant Immunologist at Royal London Hospital, London.
“HAE patients around the world are waiting for an oral, once-daily therapy to prevent attacks and reduce their burden of therapy. With this decision by the MHRA, the wait for many HAE patients in the UK can end sooner,” said Jon Stonehouse, chief executive officer of BioCryst.
The European Medicines Agency (EMA) is reviewing the marketing authorisation application (MAA) for berotralstat under the centralized procedure. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected approximately 12 months from MAA validation, which the company announced on March 30, 2020.
Joint statement from HAE UK, UKPIN, HAE Clinical Network and the NHSE Immunology CRG Chair
24th March 2020
Some patients with HAE have received a communication from the NHS stating that they are at risk of severe illness if they catch COVID-19. This would not be related to their HAE itself but to any additional risk factors such as age, chronic heart disease, chronic lung disease, asthma etc. Patients with HAE are not thought to have any increased risk of harm from COVID-19 compared to the general population. HAE patients should take the same precautions as recommended by the government for the general public https://www.gov.uk/coronavirus
If you need further information, please contact: email@example.com
Coronavirus Covid-19 information.
From the HAE CLINICAL NETWORK
- There is no evidence to suggest that people with HAE (whether on prophylaxis or not) are likely to have a higher risk of severe Covid-19 disease, unless they have any other risk factors or pre-existing disease (hypertension, diabetes, elderly, cardiac/respiratory disease). Swellings may be more frequent during infection, but will return to baseline after the infection is over.
- It would be appropriate to follow current government guidance re washing hands, avoiding unwell people etc – and to keep an eye on this as it is constantly changing. We do not suggest anything extra for HAE patients at present.
- Looking at the data, children actually have mild/milder disease and are a lot less likely to get complications with Covid-19. Re HAE, we would not suggest anything different to what is currently being done with children without HAE. As regards taking children out of school, the UK government hasn’t cancelled schools yet – although a lot of other countries are – which is a matter of debate as to who has done the right thing. So whilst schools are open children should attend.
- Getting more HAE attacks with Covid -19. It is likely that there will be immunity following Covid-19 infection – so, people should be unlikely to get it twice (at least in the immediate future). So, although C1 inhibitor usage may go up during the time they are unwell, this would only be for a relatively short period of time and shouldn’t affect the overall C1 usage (although anxiety/stress around Covid-19 may). For the majority of people, Covid-19 will be a mild self-limiting illness – which is something that is worth reminding people about.
- Do not stockpile your medication! The companies are doing all they can to maintain supply and to ensure there is enough to go around.
I Am Number 17.
Royal London is the first centre in the world to be running a clinical trial of a new oral medication for people with HAE.
Royal London immunology department are enrolling patients for a trial of an oral drug for acute attacks of HAE. The hope is for this drug to control HAE attacks early enough to prevent a full blown swelling. If effective, this would be ideal for patients who are not keen on taking regular medications or using injections for acute treatment.
Male or female patients over 18 years of age, with HAE type 1 or 2 are eligible to be considered for recruitment unless their nearest immunology centre is also planning to run this trial. The medication is to be used as soon as the patient notices the beginning of an HAE attack.
Any patients with 2 or more attacks in 93 days are eligible for this trial. If you are on a prophylactic (preventative) medication (e.g. Danazole, Oxandrolone or Tranexamic acid, regular C1 esterase Inhibitor), you will be asked to stop this before the trial and restart after the trial is completed. The length of time needed for stopping prophylactic medication is varied for different medications.
You would have 4 visits to the clinical research facilities at the Royal London Hospital for the purposes of this trial. There will be a fee of £200 per visit for your time in addition to travel and accommodation if required.
If you are interested to know more, please contact Dr Sorena Kiani on BHNT.Advice-Immunology-Barts@nhs.net.
One of our members, Philippa Adams, completed a University dissertation on HAE, titled ‘The Lived Experience of Women Who Have Hereditary Angioedema’
You can read her dissertation here.
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29th February 2016 was Rare Disease Day. Click here to see the Rare Disease Day supplement from the Independent newspaper
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12th March 2012
Press Release about Firazyr
The next HAE UK Patient Day is to be held in November 2020. More details about this popular event will be available on the events page.
HAE Day is on 16th May each year. Visit the HAE Awareness Day website to find out more and to see how you can help spread awareness of HAE.
The HAE Global Conference is held every two years. The next conference is to be held in Frankfurt in May 2020. Find out more by visiting the HAEi website