Project Description

Children are not small adults

When it comes to the use of medicines, children cannot be treated as small adults. Simply using existing solutions and trying to estimate the correct dose by reducing it according to body size is fraught with potential dangers. Estimating dosages, for example, based on label recommendations may cause adverse reactions and unintended harm – especially to the functions and maturity of organs such as the brain, liver and kidneys – whereas under-dosing leads to a lack of efficacy.

Clinicians face a Catch 22 situation

So doctors (and parents) have a problem. Put simply, the effects of medicines on the body (pharmacodynamics) and/or the effects of the body on medicines (pharmacokenetics) may be different in children.

Clinicians with a need for children’s medicines are restricted to very few paediatric products that have been tested in children and are authorised and available to them on the market. As a result, this has left clinicians with no alternative but to use ‘adult’ drugs on children, despite the significant risks this course of action poses.

It’s estimated that between a third and a half of all prescribed and dispensed medicines, from capsules and tablets to ointments and inhalers, are not used as recommended. This non-adherence is disadvantageous to patients, particularly children, healthcare providers and society at large.

Note that children aren’t the only group in the unfortunate position of having what’s known as an ‘adherence’ problem. The elderly and those with chronic or rare diseases, for example, can also suffer from a lack of medicines specifically created and tested for them and, therefore, often take incorrect doses or inappropriate drugs.

“Therakind’s objective is to increase the availability of authorised paediatric medicines and ensure that children’s medicine used today is both safe and effective.”

That situation has recently changed for one group of medicines

UCL spinout business, Therakind, is a speciality pharmaceutical company focused on developing licensed medicines which address adherence problems encountered by speciality populations such as children. They recently announced that the European Commission has adopted the recommendation of the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) to grant a marketing authorisation for Jylamovo, Therakind’s 2 mg/ml oral solution of methotrexate, for children.

As Dr Susan Conroy, the company’s CEO, commented ‘Therakind’s objective is to increase the availability of authorised paediatric medicines and ensure that children’s medicine used today is both safe and effective. For chronic conditions in particular, the benefit of taking medicine more easily should not be discounted.’

Proof of that philosophy in practice

The company’s new and breakthrough version of methotrexate evidences their commitment to extending the availability of child-centric medication. Methotrexate is a disease-modifying anti-rheumatic drug (DMARD) which can reduce the activity of the body’s immune system and interfere with the growth of certain cells, especially those that reproduce quickly, such as bone marrow and skin cells. In adults, it is used to treat certain types of cancer including those of the breast, skin, lung or head and neck.

In addition, it’s used to treat severe psoriasis and rheumatoid arthritis. In the latter disease, methotrexate modifies the underlying disease process to limit or prevent joint damage and disability, rather than simply treating the symptoms.

Benefiting children across Europe

Jylamvo is a versatile medication. It is now licensed in all European Economic Area countries for use in adults and children over 3 years old for the treatment of rheumatological disorders (adult and juvenile) and adult psoriasis, as well as for the maintenance treatment of acute lymphoblastic leukaemia (ALL). It is also the first oral liquid formulation of methotrexate to be authorised across the continent for the treatment of juvenile idiopathic arthritis (JIA).

This is another major achievement for the company. Therakind has now successfully brought three medicines through the regulatory process and into the market. What’s more they have an exciting and innovative pipeline of products in development covering a range of therapeutic areas, from diabetes to sedation and antibiotics to endocrinology.

There’s no doubt that clinicians treating speciality populations – such as children – who have traditionally faced ‘adherence’ issues, will have a greater choice of specifically designed and developed medications to choose from in the future.

Dr Conroy concluded, ‘Jylamvo was formulated as an oral solution primarily so that young children and older adults who often have difficulty swallowing tablets would have access to a licensed oral liquid form of methotrexate. Additionally, taking a medicine as a liquid can make any dosing adjustment easier.

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