A new treatment called Tryngolza (olezarsen) has been recommended by NICE (National Institute for Health and Care Excellence) for adults in England and Wales who have a rare genetic disorder called familial chylomicronaemia syndrome (FCS). This medication will only be available if diet and usual triglyceride-lowering treatments have not worked well enough. FCS is a very rare inherited condition that causes dangerously high levels of fat in the blood, increasing the risk of severe pancreatitis, a painful and potentially life-threatening inflammation of the pancreas. Symptoms include intense stomach pain, frequent hospital stays, long-term organ damage, and in some cases, can be fatal. The recommendation is based on a major clinical trial called the Balance study, which showed that Tryngolza 80 mg significantly lowered fasting triglyceride levels at 6 months, and this effect continued for up to 12 months. The treatment also reduced the number of acute pancreatitis attacks over the same period. Sobi, the company behind Tryngolza, described the decision as an important step forward for people living with FCS. Professor Handrean Soran, a Consultant Physician and Endocrinologist at Manchester University NHS Foundation Trust, explained that people with FCS have extremely high triglyceride levels from childhood, causing severe pain and unpredictable, life-threatening pancreatitis attacks. Historically, treatment options have been very limited. He said that access to Tryngolza through the NHS in England and Wales will be a major improvement, offering adults with FCS a convenient once-a-month treatment that helps maintain lower triglyceride levels, reduces the risk of pancreatitis, and lowers the chance of hospitalisation due to pancreatitis. Jill Prawer, Chair of Action FCS, a support group for people with FCS, welcomed the decision, saying that the strict fat-free diet required for managing FCS affects every part of daily life, including every meal. She also highlighted the constant worry of sudden, painful pancreatitis attacks that can lead to hospital stays, taking time away from family, education, and work. Sharon Hall, General Manager at Sobi UK, called the NICE recommendation a significant milestone for people living with FCS in England and Wales. She stated that this decision marks meaningful progress in reducing the heavy burden of this ultra-rare condition and is an important achievement for the FCS community. She added that Sobi’s next steps will focus on working with the NHS, healthcare experts, and the FCS community to ensure the smooth introduction of this new treatment and to make sure eligible patients can access it.