News 2014 - 2016
SfE patient support grant
July: We are delighted to be among the lucky recipients to be awarded a patient support grant from the Society for Endocrinology. The grant is to be used to help us produce and dicrtribute 5 new patient information leaflets. Thank you to the SfE and all involved in the decision making process.
First European leaders meeting
On 8th July an historic event took place in the hypopara world when the leaders of the European hypopara organisations met for the first time.
Leaders from UK, Ireland, France, Italy, Norway, Sweden and Denmark were brought together by Shire International of Switzerland to form a Patient Advisory Group of European leaders to advise on patient needs.
After a welcome dinner in London we spent the next day listening to each other, learning about patient needs in each country, and discussing ways we could work together to improve the lives of patients across Europe.
We hope that our ideas will be carried forward and that a European Hypopara Alliance will grow out of this meeting. We look forward to working together because together we have a louder voice. Thank you to Shire International and Bioscience Communications for a productive and insightful meeting.
Our CEO wins Britain's Best Volunteer award
May: Our very own founder and chief executive, Liz Glenister, won the Britain’s Best Volunteer award 2016 in recognition of her ten years' work for patients with hypoparathyroidism.
Liz won a holiday for herself and £1250 for Hypopara UK, which was used to support new parathyroid stem cell research in the UK.
Liz commented: ‘I’m absolutely thrilled to have won this award which I see as a fantastic opportunity to raise awareness about this rare condition and our small, but far reaching charity. We are raising funds for vital stem cell research at the moment so this prize will really help towards that goal. Thank you to everyone who voted, to my committee who nominated me and well done to all the finalists who all work so hard and represent such worthy causes.’ Read our press release here
The award is from Markel who run the award with the Small Charities Coalition. Huge thanks to them both. Read their article here and watch the video they made about us and why this award is so important to a small charity like ours.
Celebrating our 10th year 15th July 2005 - 2015
In 2005, our aims were to bring patients together, provide information (none existed online or in the UK at all then), to raise awareness about the need for treatment guidelines and replacement hormone and home testers. We've come a long way since then.
Today we support over 1500 members who communicate online on our website forum and very busy facebook group, by post, email and telephone helplines. Many members have never met another person with hypopara face to face so now we have set up local groups and are getting together around the UK to celebrate our achievements – and each other.
This week, the first ever guideline on the treatment of hypoparathyroidism was published by the European Society for Endocrinology (see below) and presented at the ECE 2015 in Dublin which we were delighted to attend. Hypopara UK was honoured to be invited to comment and to collaborate on the accompanying patient leaflet which will be out soon. We were also delighted to attend the first International Hypoparathyroidism Conference held in Florence in May, and further guidance from this meeting is also expected soon. Here the 3 heads of the USA, UK and Nordic hypopara organisations met in person for the first time - a fitting and moving celebration for each of us.
The parathyroid hormone PTH 1-84 Natpara was approved in the USA in January and has now been granted orphan drug status in 2013 in Europe. We are currently waiting for the EMA marketing approval and hope to know more this Autumn.
We are still working on home testers. Like diabetics we can have life threatening hypos and need to manage unstable levels with regular monitoring but have no replacement hormone or home blood testers to help us which can make life pretty difficult and scary at times. But we’ll get there…
So our tenth year has been pretty fantastic so far and we have much to celebrate. Here's to us, to all our friends and supporters and to all the wonderful doctors working to achieve the same ends - here’s to the next ten years!
ESE Clinical Guideline: Treatment of chronic hypoparathyroidism in adults
13th July 2015: The first ever clinical guideline for the treatment of hypoparathyroidism has just been published by the European Society of Endocrinology. Hypopara UK has been working towards this day for a long time, raising awareness about patient needs and the need to provide information for professionals. Weare very pleased to have been invited to review this paper and we have also collaborated in the development of the accompanying patient leaflet which will be out soon.
We are very pleased to see so many recommendations that agree with our own patient findings and we hope that guidance on the use of PTH therapy will soon be amended.
You can download the guideline from the ESE website here
Grant from NPS Pharma
19th February 2015 - we were delighted to recieve a very generous grant from NPS Pharma in recognition of, and to continue, the work we do to support people with hypoparathyroidism and to raise awareness about the condition. We are especially grateful as this means we will now be able to plan ahead more securely.
FDA approve Natpara
23rd January 2015 - The U.S. Food and Drug Administration today approved Natpara to control hypocalcemia in patients with hypoparathyroidism. Natpara, a bioengineered replica of human PTH, is expected to be available in the USA later this year.
The approval was supported by 12 pharmacology studies and four company-sponsored efficacy and safety studies. The pivotal Phase 3 study, known as REPLACE, was a randomized, double-blind, placebo-controlled study and the largest clinical trial conducted to date in patients with hypoparathyroidism. Our grateful thanks go out to the 10 members of Hypopara UK took part in this trial (and were the second largest cohort after the USA).
This is news that we have been waiting to hear for many years. It marks an important milestone in the treatment of hypoparathyroidism and we now hope for a positive outcome in Europe. Read more
NPS Pharma to be taken over by Shire
January 11th 2015: We have been assured that this take over will be a seamless transition. It is hoped that the commitment to patients, the passion for developing new treatments and the efforts to raise awareness will be enhanced. Read more here
EMA validates marketing authorization review
December - In Europe, the European Medicines Agency (EMA) validated and initiated its review of NPS Pharma's marketing authorization application for Natpar.
Rare disease medicines on the up
The European Medicines Agency approved a record number of medicines for rare diseases in 2014. Read more
British Thyroid Association Guidelines for the Management of Thyroid Cancer
The new 2014 edition has now been published. Hypopara UK is very pleased to be acknowledged as a reviewer and to have been able to help update the information on post surgical hypoparathyroidism and the patient information leaflets. Read the Guidelines here
Thyroid Cancer Alliance
Hypopara UK would like to thank Kate Farnell for all her hard work as the first President of the TCA and to welcome Soledad Rodrigruez Perea, CEO of Actira in Argentina as the incoming President. Congratulations, Sole! Judith Taylor (secretary) of Hypopara UK and Carmen Villar of AECAT, Spain, are on the board.
World Orphan Drug Congress
Hypopara UK were delighted to be invited to attend the World Orphan Drug Congress in November. We were ably represented by Judith Taylor, our PR Officer. More soon...
SfE Clinical Update 2014
Liz and Bill Glenister and Ray Finlay were pleased to be able to set up the Hypopara stand at the Society for Endocrinology Clinical Update exhibition (3-5th November) thanks to a travel grant from the SfE. We were also able to attend the final year workshops in which two of our advisory team were involved as part of the packed training programme. Neil Gittoes convened the workshop on the parathyroid glands with Dr Rachel Crowley and Dr Peter Selby who also lectured on Vitamin D. Fascinating - more in the newsletter! We are also delighted to announce that Dr Rachel Crowley from Dublin will be joining our Advisory Team and getting involved in our Irish Hypopara Group too.
Hypopara UK wins £2000 award from Lloyds
Hypopara UK committee members, Claire Butchers, applied for a Lloyds of London charity grant set up for employees who are involved with a charity. Obviously they liked what she wrote and liked what we do, as we won the award. Thank you Claire, very well done!
FDA push back Natpara approval
The goal date for approval of Natpara (PTH 1-84) was due on October 24th 2014 but has been moved back 3 months. An announcement is now expected on January 24th 2015. Read the NPS Pharma press release here
Parathyroidectomies on children
Tom Kurzawinski of the British Association of Endocrine & Thyroid Surgeons (BAETS) is inviting his colleagues to contribute to a national survey of parathyroidectomies done in children and teenagers under the age of 18 years operated in the last decade (2004-2014). His personal experience of 38 such operations performed at the Great Ormond Street Hospital is already very impressive but through a national contribution BAETS hope to reach even higher numbers. Read more
Autosomal Dominant Hypocalcaemia study
NPS Pharma has initiated a Phase 2a study in the USA to evaluate the safety and tolerability of NPSP795 in adult patients with Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disorder of calcium homeostasis. Read more here
New BTA Thyroid Cancer Guidelines
Hypopara UK was delighted to have been invited to review the new edition of the British Thyroid Association Thyroid Cancer Guidelines patient information and help update the information on post surgical hypoparathyroidism. Read the Guidelines here
Hypopara UK attends US Hypoparathyroidism conference
Hypoapra UK were able to send representatives Judith Tayler and Claire Butchers to the US Hypoparathyroidism conference held in Sacramento in June 2014 thanks to a grant from NPS Pharma. Read about it in the next newsletter!
Hypopara UK are delighted to be working in partnership with NPS Pharmaceuticals as the company continues to study novel approaches to parathyroid hormone replacement in patients with hypoparathyroidism.
Oral PTH UK trial - STILL RECRUITING!
Professor Bill Fraser, lead advisor for Hypopara UK, will soon be trialling a new oral PTH 1-84. The trial will take place in Norwich was due to begin in March 2014 but there have been delays due to the prodeucer reloacting. Thank you to everyine who has applied so far, we will keep you posted. Meanwhile, if you are a UK hypopara patient and would still like to particiapte click here for details.
New Emergency Guidelines
The Society for Endocrinology's clinical committee have produced 2 new sets of emergency endocrine guidelines for non-specialist healthcare professionals:
UK Strategy for Rare Diseases
The UK Strategy for Rare Diseases was published in Nov 2013. It contains 51 commitments which all four countries of the UK have agreed to achieve by 2020. Last week NHS England published a statement on how they plan to deliver them. Read more
NHS Change Day
Hypopara UK supports NHS CHange Day and has made a two way pledge - to provide our members with information about accessing healthcare, and to campaign to raise awareness about rare parathyroid conditions to healthcare professionals. Will you support us? Click here
Rare Disease Day 2014
We are launching our new website address and redesign to mark Rare Disease Day this year. We hope you like it! You can help to raise awareness too by joining the Rare Disease UK campaign and writing to your MP.
FDA Accepts License Application for Natpara
7th January 2014: In the USA, NPS announced that the FDA has accepted and filed for review their License Application for Natpara for the treatment of hypoparathyroidism. Goal date for a decision by the FDA is October 24, 2014. Read more
Natpara designated EU orphan drug status
3rd January 2014: NPS announced that the European Commission has granted orphan drug designation to Natpara® (recombinant human parathyroid hormone (rhPTH[1-84]) for the treatment of hypoparathyroidism. Read more