New gene-therapy may be approved in EU
New treatment Glybera has been recommended for marketing in the European Union as a treatment for severe and multiple pancreatitis attacks due to lipoprotein lipase deficiency (LPL).
Glybera (alipogene tiparvovec) is the first gene-therapy medicine to be recommended for authorisation in the European Union. Designed to replace defective genes with a working copy, the gene-therapy treatment Glybera will add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells.
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LPL deficiency is an extremely rare inherited disorder estimated to affect one or two people per million. Due to a defective gene, patients with this disorder cannot produce enough LPL, an enzyme responsible for breaking down fats. So far, management of patients with the disorder consists of strict reduction of dietary fat to less than 20% of the daily caloric intake. It is very difficult to comply with such a dietary regimen and as a consequence many patients experience life-threatening pancreatitis attacks requiring admission to hospital.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of market authorisation in ‘exceptional circumstances’, based on the requirement that the company marketing Glybera will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera.
“The evaluation of this application has been a very complex process, but the use of Glybera in a more restricted indication than initially applied for, which targets the patient population with greatest need for treatment, and additional analyses by the Committee for Advanced Therapies (CAT) have added to the robustness of the data provided and allowed the CHMP to conclude that the benefits of Glybera are greater than its known risks”, said Dr Tomas Salmonson, acting Chair of the CHMP.
Introducing Dosis - the AI powered dosing platform
Cloud-based platform Dosis uses AI to help patients and clinicians tailor their medication plans. Shivrat Chhabra, CEO and co-founder, tells us how it works.
When and why was Dosis founded?
Divya, my co-founder and I founded Dosis in 2017 with the purpose of creating a personalised dosing platform. We see personalisation in so many aspects of our lives, but not in the amount of medication we receive. We came across some research at the University of Louisville that personalised the dosing of a class of drugs called ESAs that are used to treat chronic anaemia. We thought, if commercialised, this could greatly benefit the healthcare industry by introducing precision medicine to drug dosing.
The research also showed that by taking this personalised approach, less drugs were needed to achieve the same or better outcomes. That meant that patients were exposed to less medication, so there was a lower likelihood of side effects. It also meant that the cost of care was reduced.
What is the Strategic Anemia Advisor?
Dosis’s flagship product, Strategic Anemia Advisor (SAA), personalises the dosing of Erythropoiesis Stimulating Agents (ESAs). ESAs are a class of drugs used to treat chronic anaemia, a common complication of chronic kidney disease.
SAA takes into account a patient’s previous ESA doses and lab levels, determines the patient’s unique response to the drug and outputs an ESA dose recommendation to keep the patient within a specified therapeutic target range. Healthcare providers use SAA as a clinical decision support tool.
What else is Dosis working on?
In the near term, we are working on releasing a personalised dosing module for IV iron, another drug that’s used in tandem with ESAs to treat chronic anaemia. We’re also working on personalising the dosing for the three drugs used to treat Mineral Bone Disorder. We’re very excited to expand our platform to these new drugs.
What are Dosis' strategic goals for the next 2-3 years?
We strongly believe that personalised dosing will be the standard of care within the next decade, and we’re honored to be a part of making that future a reality. In the next few years, we see Dosis entering partnerships with other companies that operate within value-based care environments, where tools like ours that help reduce cost while maintaining or improving outcomes are extremely useful.
What do you think AI's greatest benefits to healthcare are?
If designed well, AI in healthcare allows for a practical and usable way to deploy solutions that would not be feasible otherwise. For example, it’s possible for someone to manually solve the mathematical equations necessary to personalise drug dosing, but it is just not practical. AI in healthcare offers an exciting path forward for implementing solutions that for so long have appeared impractical or impossible.