A Phase 2 trial will explore effect of AM1476 on skin thickness, lung function by Andrea Lobo, PhD | February 6, 2024 AM1476, AnaMar’s investigational anti-fibrotic medication, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for treating scleroderma.
To get this designation, a medication must be intended to treat a life-threatening rare disease that affects fewer than five in 100,000 people in Europe or under 200,000 people in the U.S. The designation provides certain incentives, including regulatory fee reductions and the potential for extensive market exclusivity — seven years in the U.S. and 10 in the European Union — if approved. The company plans a Phase 2 clinical trial to evaluate the treatment in people with scleroderma and interstitial lung disease (ILD), a group of diseases marked by lung scarring. The study will evaluate AM1476’s effects on skin thickness and lung function in 60 patients with diffuse cutaneous scleroderma and ILD over a year. “This is a significant milestone and underscores the significant unmet need for novel medicines to prevent, heal, and slow organ scarring from fibrotic diseases, which are often progressive and can have a poor prognosis,” Ulf Ljungberg, PhD, AnaMar’s CEO, said in a company press release. Scleroderma, also known as systemic sclerosis, is marked by inflammation and fibrosis, or uncontrolled tissue hardening and scarring, in the skin and even the heart, kidneys, and lungs. Up to 80% of people with SSc may develop ILD, which leads to reduced lung function and breathing problems. Cabaletta Bio developing CAR T-cell therapy for other autoimmune conditions by Marisa Wexler, MS | January 9, 2024 The U.S. Food and Drug Administration (FDA) has granted fast track designation to the cell therapy CABA-201 for organ dysfunction in people with scleroderma.
The agency also granted CABA-201 fast track status to reduce disease activity in people with dermatomyositis, another rare disorder that’s marked by muscle weakness and skin rash. The FDA had previously given CABA-201 fast track designation as a potential treatment for lupus. “The additional Fast Track Designations for CABA-201 … provide the opportunity for expedited development and review of CABA-201 for the treatment of these autoimmune indications where there is a significant unmet need, despite currently available therapies,” David Chang, MD, chief medical officer at Cabaletta Bio, which is developing CABA-201, said in a company press release. Kedron-Wavell Services Club has generously provided a community donation of $300 for the cost of room hire for our event with Associate Professor Tony Kenna in September.
Generous donations such as this from #kw allows Scleroderma Queensland to raise such important funds for much-needed research and awareness. THANK YOU AGAIN to Kedron-Wavell Services Club. We gratefully acknowledge their support. #KW #KedronWavell #eventsatkw https://kedron-wavell.com.au/ About 2% of people with scleroderma are positive for more than one disease-associated antibody that targets tissues, causing damaging inflammation, according to a new study.
Results suggest that certain antibody combinations may be associated with distinct clinical features. Variants of immune-regulating human leukocyte antigen (HLA) genes were associated with the risk of systemic scleroderma, subtypes of the condition, and the presence of self-reactive antibodies, a large-scale genetic analysis showed.
These findings underscored the genetic contribution to the disease and support future investigations into immunological susceptibility and external environmental stimuli that trigger autoimmunity in people with systemic scleroderma, the researchers noted. Join us on Saturday 25 June 2022 for our Annual Scleroderma Seminar.
Our annual seminar is a wonderful opportunity to come together to hear from guest speakers, learn from each other and enjoy support and friendship. Date Saturday 25 June 2022 Time 8.30am - 3.30pm Venue Kallangur Community Hall 1480 Anzac Avenue Kallangur QLD 4503 Cost $25 Financial Members, $30 Non-Financial Members Will you Snuggle Up for Scleroderma Australia and help raise funds for a specialist scleroderma nurse?
Scleroderma symptoms can vary greatly from person to person, however, up to half of all scleroderma patients also develop lung problems.
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialised facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted the online survey from November to December 2021.
Read the article here. |
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