The nasal approach

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 whereas under-dosing leads to a lack of efficacy.

Note that children are not the only group that is affected. Other speciality populations such as the elderly and those with chronic or rare disease can also suffer from a lack of medicines specifically created and tested for them and, therefore, often take incorrect doses or inappropriate drugs.

Clinicians treating these patient populations are restricted to fewer products that are authorised and available to them on the market. As a result, this can place patients in an unfortunate position of non-adherence to medicines which is disadvantageous to their well-being, healthcare providers and society at large.

Proof of philosophy in practice

Therakind’s breakthrough version of methotrexate, Jylamvo®, evidences their commitment to extending the availability of established drugs for difficult to treat patient groups. Jylamvo® was formulated as an oral solution primarily to allow young children and older adults who often have difficulty swallowing tablets access to a licensed oral liquid form of methotrexate.

 

Jylamvo® is a versatile medication. It is licensed in the United Kingdom and all European Economic Area countries for use in children over 3 years old and adults 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).

In 2022, Jylamvo® was approved in the United States for use in adults and is expected to obtain indications in children in 2024.

“Therakind’s objective is to reformulate drugs for alternative routes of delivery and medicinal forms to ensure easier administration across different patient populations”

Using the nose to deliver medicines

For most people the first thought on hearing vaccines is an injection in the arm, and medicines are instinctively linked to swallowing tablets. While nasal products such as decongestants, influenza vaccines and corticosteroids have been around for a while, the nose remains an unconventional route of delivery.

 

At Therakind, drug delivery was reimagined to look like a dry powder sprayed up the nose using a specialised intranasal device. This route of delivery offers exciting, new opportunities for the pharmaceutical community to utilise for the advancement of medicines and vaccines. Couple this with easy-to-use, needle-free and simplified dosing regimens, and patients are welcoming the change in administration habits.

 

Small molecules to biologics, daily use to emergency use medicines, metabolic to neurodegenerative diseases, nasal drug delivery is being explored for a wide range of applications. The nasal cavity provides a large surface area for absorption into the bloodstream through which drugs can reach their site of action in the body relatively quicker. Furthermore, they are able to bypass first pass metabolism which the oral route is susceptible to and can reduce the effective drug concentration at the site of action.

Patient preferences for nasal delivery are driving development

Analysis has shown that patient preferences for nasal delivery are driving expansion of the global nasal drug product market. Therakind recognised nasal drug delivery as a perfect fit into its philosophy of improving patient convenience. DriDose® takes a step further to address issues around pharmaceutical distribution with the inclusion of a dry powder which is chemically more stable than a liquid and will not require cold chain storage.

 

The COVID-19 pandemic brought nasal vaccines under the spotlight for all the right reasons from offering increased immune protection to reducing waste caused by needles. DriDose® exemplifies an ideal tool for mass vaccination settings. Unlike having to replace syringes between individuals, it allows multiple doses to be administered using the same medical device.

 

Therakind have so far successfully brought three medicines through the regulatory process and into the market. They have an innovative pipeline of products in development and continuing to build on their experience to mark another major achievement, this time with their proprietary DriDose® intranasal device.