Global ITP News
Grifols to launch TAVLESSE® in Europe and Turkey to continue reinforcing its commercial strategy and commitment to patients
Grifols announced that the European Commission has approved US-based Rigel Pharmaceuticals’s TAVLESSE® (fostamatinib) for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments.
Grifols has exclusive rights to fostamatinib in chronic ITP, as well as any potential future indications like autoimmune hemolytic anemia (AIHA), and IgA nephropathy (IgAN), in Europe and Turkey.
- From Grifols press release
EMA Panel Backs Fostamatinib (Tavalisse) for Chronic ITP
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of fostamatinib disodium hexahydrate (Tavalisse, Rigel Pharmaceuticals, Inc) to treat adults with chronic immune thrombocytopenia (ITP) who fail other treatments.
- By Megan Brooks for Medscape.com
Pakistan's ex-Prime Minister Sharif diagnosed with immune disorder
Sharif, 69, is being treated at a hospital in Lahore, where medical tests suggest he has a low count of blood platelets.
Jailed former Pakistani Prime Minister Nawaz Sharif is suffering from a serious immune disorder, his party leaders say, accusing the government of not providing him adequate medical care.
Sharif, 69, is being treated at a government-run hospital in the eastern city of Lahore, where medical tests suggest he has a dangerously low count of blood platelets, Talal Chaudhry, a senior leader of Sharif's PML-N party, told Al Jazeera on Friday.
"It is acute immune thrombocytopenic purpura, a bleeding disorder, in which the immune system destroys platelets," the doctor told Dawn, speaking on condition of anonymity. "We are hopeful that his condition will improve in a few days."
- by Asad Hashim for aljazeera.com. Photo courtesy of Wikipedia.
New hope for ITP patients dashed by high drug cost
Without access to eltrombopag, Gellein continues to take other medications and suffer from side-effects of anxiety, depression, weakness, nausea and dizziness. She is admitted to hospital several times each year for emergency treatment, which is common for patients with uncontrolled ITP.
She wants people to know the ripple effect it has caused on those around her, and desperately wants her life back.
"My life could have been so much different with these drugs," said Gellein. "Something needs to change, if not for me, for the hundreds of young people being diagnosed with ITP in Canada every year."
- By Dr. Meera Dalal for CBC News. Photo courtesy of Chrystal Gellein.
Buenos Aires First-Ever World ITP Day a Tremendous Success!
On Saturday, October 6, 2019, PTI Argentina held the first celebration of World ITP Day in Buenos Aires, Argentina. Months of hard work with almost no funding proved a big success as people turned out to show their support and listen to the voices of ITP!
After a group walk along Buenos Aires main Avenue, handing out brochures in the street, the group shared a friendly afternoon tea with a well-known hematologist who answered questions about ITP. At the end of the day everyone left with a big smile (and a full stomach), fewer doubts about ITP and a "Mission Accomplished" feeling that will motivate them to continue advocating for and supporting ITP patients.
2019 Global Alliance Meeting
On Thursday, July 25th, PDSA was honored to host 18 delegates from 12 countries prior to ITP Conference 2019 for the 3rd International ITP Alliance Meeting. In addition to updates from Alliance Ambassadors from around the world, the symposium included sessions about the ITP Registry, working with industry partners, the development of globalITP.org and capacity building. International ITP Alliance Ambassadors also had the opportunity to meet one-on-one with ITP industry partners. Representatives from Argentina, Australia, Brazil, China, Denmark, Finland, Israel, Italy, Netherlands, New Zealand, United Kingdom and the United States attended the full-day meeting and also joined in ITP Conference 2019 sessions and festivities!
Chronic immune thrombocytopenia in Denmark, Sweden and Norway: The Nordic Country Patient Registry for Romiplostim
The NCPRR was established in April 2009 as a cohort study of all adult cITP patients in Denmark, Sweden and Norway (combined adult population = 15∙4 million persons) with study inclusion continuing through 2019. The three countries have tax-funded health care systems. ITP patients are referred by their general practitioners to hospital-based hematological specialists. Hospitals are required to report data on all hospital visits to nationwide hospital registries. Virtually complete follow-up for hospitalizations, outpatient clinic visits, and death among ITP patients is available through the countries´ nationwide hospital and population registries, linkable through the personal identification number (PIN) assigned to all residents of each country.
- From thelancet.com
Chi-Med Initiates a Phase I Trial of HMPL-523 in Patients with ITP in China
Hutchison China MediTech Limited (“Chi-Med”) has initiated a Phase I study of HMPL-523, its novel spleen tyrosine kinase (“Syk”) inhibitor, in patients with immune thrombocytopenia (“ITP”), an autoimmune disorder that can lead to increased risk of bleeding. The first ITP patient was dosed on August 12, 2019 in China.
The study is a randomized, double-blinded, placebo-controlled Phase Ib clinical trial investigating the safety, tolerability, pharmacokinetics and preliminary efficacy of HMPL-523 in adult patients with ITP. The primary endpoint is the number of patients with any adverse event. Approximately 50 to 60 patients will be enrolled.
- From Hutchison China MediTech Limited News Release
First Irish patient day in more than a decade for rare blood condition
On 13 April 2019, immune thrombocytopenia (ITP) patients from around Ireland, were welcomed to the ITP Support Association Patient Day in Dublin supported by Novartis. Chaired by Mr. Mervyn Morgan (Chief Executive of ITP Support Association), the meeting provided patients with an opportunity to discuss their experience of ITP with other patients and to learn about their disease from experts in the field.
Last held in Dublin in 2004, this year’s meeting featured talks from Ms. Nicola Harten (Clinical Nurse Specialist, Connolly Hospital, Dublin), Dr. Gerard Crotty (Consultant Haematologist, Midland Regional Hospitals, Tullamore, Portlaoise and Mullingar), Prof. Adrian Newland (Professor of Haematology, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, UK) and Mr. Derek Elston (ITP Patient Mentor).
Additional highlights from the meeting included a Q+A session, an overview of the ITP pocket log and patient breakout groups. The meeting was attended by 55 ITP patients from across the country.
Intas Becomes the First to Launch Romiplostim in India, Significantly Reducing the Treatment Cost of Chronic ITP
One of India's leading pharmaceutical companies, Intas Pharmaceuticals, takes a revolutionary step towards making therapy for chronic immune thrombocytopenia (ITP) more accessible to patients by being the first to launch Romiplostim in India under the name Romy®
Romiplostim is one of the most efficacious agents with proven long-term benefits in chronic ITP. It represents the standard of care but has been available only internationally since 2008. It is estimated that around 42,000 patients are diagnosed with ITP in India every year. Less than 10% of patients which include adults as well as children, have access to the treatment which is required to maintain adequate platelet counts for a longer duration. In ITP, long term use of drugs is required to maintain adequate platelet count.
- From Intas Pharmaceuticals Press Release
ITP Support Association is hosting a patient day
Patients living with Immune Thrombocytopenia (ITP), and their families, are invited to attend an upcoming ITP Patient Day organised by patient group ITP Support Association on Saturday 13th of April at the Ashling Hotel, Dublin. The event, which is supported by Novartis, aims to bring together people living with ITP and their families to learn from one another and leading experts in haematology about the management of ITP in a relaxed and informal setting.
- From KilkennyPeople.ie
ITP Weekend Planned: London, ON Canada – April 27-28, 2019
PDSA Canada has planned a weekend all about ITP in London, ON Canada.
On Saturday, April 27, 2019 the community will Pump It Up For Platelets!
On Sunday, April 28, 2019 the Platelet Disorder Support Association will host its 2nd Canadian ITP Regional Meeting.
Fourth China Platelet Day on 20th, March 2019
Many gather with the power of love to improve quality of life in patients.
Genetic breakthroughs showcased on China Platelet Day
Domestic doctors can conduct precise diagnosis on platelet diseases by identifying all the 85 diseases-related genes as well as the introduction of more targeted medicine, doctors said during China Platelet Day on Wednesday.
A charity program was also launched to support patients with immune thrombocytopenia, or ITP, a type of disease characterized by a low platelet count and increased tendency to bleed.
“ITP is the most common cause of bleeding, almost covering one-third of patients with bleeding. People with ITP can suffer bleeding under the skin, inside the brain and digestive channel and people with serious conditions can die,” said Dr Liu Peng, director of the hematology department of Shanghai’s Zhongshan Hospital.
- By Cai Wenjun, Metro Managing Editor, SHINE News
China Investigating Reports Of HIV-Tainted Medication (IVIG)
Chinese authorities say they are investigating a batch of medication that is suspected to be tainted with HIV.
According to local media, the batch contains more than 12,000 doses of human immunoglobulin — intravenous treatments used to boost weakened immune systems.
China's National Health Commission said in a statement that on Tuesday it asked medical institutions to stop using the suspect medicine, seal it and monitor any patients who might be impacted. It launched a recall of the product, which it says was produced by Shanghai Xinxing Pharmaceutical Company.
- By Merrit Kennedy, NPR.org
Statement from PPTA - Plasma Protein Therapeutics Association
Safety & Immunoglobulin Manufacturing
Recent news reports called into question the safety of immunoglobulin therapies outside
of Europe and the U.S. While the facts in this case are not clear, PPTA can provide
assurance that plasma protein therapies manufactured by PPTA member companies are
safe. All undergo viral removal and inactivation steps during production. These steps are
effective in helping to eliminate risks from HIV, hepatitis, and other blood-borne infectious
agents, and the safety record of current therapies is excellent; there have been no viral
transmissions for nearly three decades.
PPTA member companies adhere to or exceed all requirements set out by regulatory
authorities around the world, including the U.S. Food and Drug Administration (FDA), the
European Medicines Agency (EMA), and others. Additionally, PPTA member companies
comply with a robust set of voluntary industry standards, including the International
Quality Plasma Program (IQPP) and the Quality Standards of Excellence, Assurance and
Leadership (QSEAL). Learn more about PPTA's Pathogen Safety program here.
Underwood terminal cancer patient feels 'helpless' after he was refused pain drug
He was due to receive his ninth cycle of this particular drug, but developed immune thrombocytopenic purpura (ITP), which causes low platelets in his blood. ITP is not directly related to his myeloma and it is uncommon for the two conditions to occur together.
In order to treat ITP, Ian was taken off his lenalidomide until his platelet level returned to an acceptable level, which in this case took six months. NHS policy requires a treatment break application to be submitted for patients who have had a pause in their treatment.
- By Hannah Mitchell, Eastwood & Kimberly Advertiser. Photo courtesy of Nottingham Post
We Live Day by Day
Cooper Higgins, 7, battling rare blood disorder known as idiopathic thrombocytopenic purpura
Cooper stopped playing hockey and had to be home-schooled because of his condition. A simple nose bleed could be fatal and even a minor bump on the head could lead to internal bleeding in the brain.
Over the next year, the family cycled through medication and treatments, trying anything they could to make a difference. But nothing worked longer than a few weeks and often the side effects were even worse.
Kelly estimates they have spent thousands of dollars on medicines. However, they finally found something that is working … at least for the time being.
- By Eric Bowling, The Westlock News. Photo courtesy of the family.
Paras Biopharmaceuticals Finland Oy Announces Successful Development of Biologically Active Romiplostim (N-Plate® Biosimilar) Production Tsful Development of Biologically Active Romiplostim (N-Plate® Biosimilar) Production Technologyechnology
Jan. 30, 2019 / PRZen / OULU, Finland -- PARAS BIOPHARMACEUTICALS FINLAND OY today announced the achievement of a major milestone – the successful development of biologically active Romiplostim (N-Plate® biosimilar) production technology.
Romiplostim has been produced with a proprietary and innovative continuous manufacturing process at the company's Finnish-based facility in Oulu-Finland. Romiplostim is used for the treatment of thrombocytopenia (low platelet count) in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP).
ITP is a potentially serious blood disorder characterized by increased destruction and impaired production of platelets. It can lead to extensive bruising and bleeding. Thrombocytopenia is common in cancer patients, resulting from chemotherapy or radiation treatment, in addition to the underlying disease itself. ITP leads to several complications. Low platelet count increases spontaneous bleeding, complicates surgical operations and can affect chemotherapy and radiation therapy.
Momenta Pharmaceuticals Announces First Subject Dosed in Phase 1/2 Clinical Trial of M254, Hypersialylated Immunoglobulin G
CAMBRIDGE, Mass., Jan. 29, 2019 (GLOBE NEWSWIRE) -- Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA), a biotechnology company focused on discovering and developing novel biologic therapeutics to treat rare immune-mediated diseases, today announced the dosing of the first subject in the Phase 1/2 clinical trial of M254, hypersialylated Immunoglobulin G (hsIgG). The Phase 1/2 study will enroll normal healthy volunteers and patients with Immune Thrombocytopenic Purpura (ITP). The multi-part study includes single and multiple dose parts, and a placebo-controlled, randomized double-blinded cross-over study comparing M254 to IVIg.
“We believe M254 has the potential to be a significantly better option for patients than conventional IVIg. Our aim for this study is to show clinically what we have observed in extensive preclinical models, which is that hypersialylated IgG is substantially more potent than intravenous immunoglobulin G (IVIg) in ITP and other inflammatory disorders,” said Santiago Arroyo, M.D., Ph.D., Senior Vice President of Development and Chief Medical Officer of Momenta Pharmaceuticals. “We look forward to obtaining initial clinical data in the first half of 2020.”
The four part study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenous (IV) M254 in approximately 65 subjects, including healthy volunteers and patients with ITP. Parts A and B are double-blind, placebo-controlled, single ascending dose cohort studies in healthy volunteers and ITP patients, respectively. In Part C, ITP patients will receive M254 or IVIg in a cross-over dosing design, while in Part D, ITP patients will receive multiple doses of M254. The primary efficacy endpoint is an assessment of platelet response.
Caroline Kruse Named President and CEO of the Platelet Disorder Support Association
The Platelet Disorder Support Association (PDSA) Board of Directors is pleased to announce that Executive Director Caroline Kruse has been named President and Chief Executive Officer. Kruse, who has been Executive Director of PDSA since January 2009, will serve in the dual role focusing on the core functions of the organization including strategy, finance, board management, fundraising, and program delivery.
Under Caroline’s dedicated leadership, PDSA has developed new relationships with key disease stakeholders and government agencies and strengthened existing relationships with partners and supporters. Her focus on mission has resulted in a greater emphasis on advocacy, expanding programs for children, teens and families affected by ITP, increasing education initiatives and advancing research to improve the quality of life for those living with immune thrombocytopenia (ITP) and other platelet disorders.
International Alliance Meeting in Chester
It was a beautiful sunny Saturday morning when all the international participants of the ITP Convention and the Alliance meeting got together at the racecourse in a beautiful city of Chester. Most of the attendees already knew each other well and the reunion was filled with joy and happiness but there were also new faces to get familiar with which was very exciting, too. Patient group representatives outside England came from the USA, Brazil, India, Italy, the Netherlands, Norway, Denmark, Sweden and Finland.
How patients are helping drive research and drug development
On October 15, 2018, PDSA President & CEO, Caroline Kruse joined this panel discussion moderated by Christopher P. Austin, M.D., Director, NCATS, NIH at the NORD Rare Diseases & Orphan Products Breakthrough Summit. The panel discussed innovative collaborations and how the role of patients, caregivers and patient organizations has become instrumental in helping to advance research and development of therapies.
Rigel Receives EMA Validation of the Marketing Authorization Application for Fostamatinib Disodium Hexahydrate in Chronic ITP in Adult Patients
First SYK inhibitor for the treatment of adult chronic ITP
SOUTH SAN FRANCISCO, Calif., Oct. 11, 2018 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for fostamatinib in adult chronic immune thrombocytopenia. The validation was received on October 4, 2018 and initiated the MAA review process. The company anticipates a decision from the Committee on Human Medicinal Products by the fourth quarter of 2019. Currently, fostamatinib is commercially available in the U.S. under the brand name TAVALISSE™ (fostamatinib disodium hexahydrate), which is the first and only SYK inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment.
- From Rigel Press Release
Five-year-old Wrexham boy with unusual and rare disease takes part in photo project to raise awareness
"Charlie Clarke and his Mum Carrie, from Wrexham, are raising awareness of ITP (Immune Thrombocytopenia) by taking part in the Rare Aware photography project by Mold-based organisation Same but Different.
"It’s important to us that there is more awareness, which is why we wanted to take part in the Rare Aware campaign.
"Knowledge is power. The more people that know about ITP and other rare conditions, the better."
- By Steve Craddock, Chief Reporter, The Leader. Photo Courtesy Ceridwen Hughes.
International ITP Alliance Members Attend Events
Global ITP advocacy partners from Denmark, Finland, Netherlands, and New Zealand attended the 18th ITP Conference 2018 for the first time as the International ITP Alliance. The three-day event and scholarship opportunity presented by the Platelet Disorder Support Association (PDSA), offered attendees information on the latest advancements in the diagnosis and treatment of ITP, small group sessions where patients shared their stories, an educational dinner program, a fun-filled hike and seek event and PDSA’s 20th Anniversary celebration at the legendary Rock and Roll Hall of Fame.
ITP Alliance at the 23rd Congress of European Hematology Association
International ITP Alliance members from Denmark, Italy, Sweden and the United States participated in the 23rd Congress of the European Hematology Association (EHA) in Stockholm, Sweden on June 14-17, 2018. It was the largest EHA Congress held to date with more than 11,000 participants from around the world. The program covered every subspecialty in hematology and attendees networked with worldwide experts. This year’s educational and scientific program highlighted state-of-the-art clinical practice, the latest findings in hematology research and other interesting sessions, provided satellite programs and an exhibition organized by pharmaceutical companies.
PATIENT FILES: ITP
"Derek Elston, trustee of the ITP Support Association, tells PharmaTimes about his experiences of being diagnosed and living with the rare bleeding disorder.
I was first diagnosed in 1993 with ITP after working in the Far East for 14 months. At the end I was fairly shattered both with the intensity of the work and commuting regularly. After about four weeks, I decided to visit the doctor. He took a sample of blood. This was followed by a frantic telephone call at midday. My platelet count was much lower than normal and he arranged for me to see the haematologist consultant at our local hospital. Normally the platelet count should be between 150 and 450, mine was ten!
After asking many questions and another blood sample, he concluded I had been infected with a virus and it had affected my autoimmune system. This he informed was called idiopathic (now immune) thrombocytopenic purpora or ITP for short, a non-malignant bleeding disorder."
- By Selina McKee for PharmaTimes.com
NOVARTIS' REVOLADE PROVED EFFECTIVE FOR SENIORS
"Novartis Korea said Tuesday additional data analysis from its EXTEND clinical trial for Revolade (ingredient: eltrombopag) proved it safe and effective for those 65 years and older with idiopathic thrombocytopenic purpura (ITP).
The sub-set study findings were presented at the first International Congress of BMT symposium held in Seoul on Aug. 26.
Revolade (known as Promacta in the U.S.) was developed by GlaxoSmithKline (GSK) and transferred to Novartis노바티스 when it acquired the former’s oncology portfolio."
- By Marian Chu, Korea Biomedical Review
BISHOP SUTTON STUDENT'S WARRIOR SPIRIT
"...Whilst studying for a degree in Stage Management at the Bristol Old Vic Theatre School Megan is aiming to raise £100,000 to support her consultant Dr Charlotte Bradbury and her research team at Bristol Haematology and Oncology Centre in their study of ITP. The team are developing a tracer and blood scanning techniques that will enable them to identify suitable and effective treatments for the individual patients."
- By Selina Cuff, for http://www.chewvalleygazette.co.uk
GIRL WITH RARE BLOOD DISORDER FORCED TO BEG FOR COSTLY MEDICINE
"...Instead of lying in a hospital bed and receiving medical care, 10-year old Bakhtawar Shafiq, who is suffering from a rare blood disorder, was forced to hold a protest demonstration in front of the Karachi Press Club (KPC) on Tuesday for a medicine that is vital for her survival but very costly and beyond the means of her poor parents.
“Doctors have diagnosed Bakhtawar with chronic immune thrombocytopenia purpura (ITP), which is a bleeding disorder in which the blood doesn’t clot as it should. They have prescribed her tablet Revolade 50mg, whose single tablet costs around Rs3,660,” said the girl’s mother after the demonstration."
By M. Waqar Bhatti. From thenews.com.pk.
DOCS RESCUE DENGUE PATIENT WITH RARE BLOOD DISORDER
"...Dengue can lead to unforeseen complications if not diagnosed and treated in time.
A 34-year-old motivational speaker and trainer Anne Anish suffered multiple complications after being gripped by dengue. However, she returned to recovery following constant monitoring by a team of intensivists, doctors and support staff at a city hospital recently.
An underlying medical condition — immune thrombocytopenia (ITP), which she had been living with since childhood, led to rapid deterioration in her health. The blood disorder can lead to excessive bleeding resulting from unusually low levels of platelets — the cells that help blood clot."
- By Umesh Isalkar, The Times of India