Topics

Drug Development for Rare Paediatric Epilepsies: Current State and Future Directions.

07:00 EST 16th November 2019 | BioPortfolio

Summary of "Drug Development for Rare Paediatric Epilepsies: Current State and Future Directions."

Rare diseases provide a challenge in the evaluation of new therapies. However, orphan drug development is of increasing interest because of the legislation enabling facilitated support by regulatory agencies through scientific advice, and the protection of the molecules with orphan designation. In the landscape of the rare epilepsies, very few syndromes, namely Dravet syndrome, Lennox-Gastaut syndrome and West syndrome, have been subject to orphan drug development. Despite orphan designations for rare epilepsies having dramatically increased in the past 10 years, the number of approved drugs remains limited and restricted to a handful of epilepsy syndromes. In this paper, we describe the current state of orphan drug development for rare epilepsies. We identified a large number of compounds currently under investigation, but mostly in the same rare epilepsy syndromes as in the past. A rationale for further development in rare epilepsies could be based on the match between the drug mechanisms of action and the knowledge of the causative gene mutation or by evidence from animal models. In case of the absence of strong pathophysiological hypotheses, exploratory/basket clinical studies could be helpful to identify a subpopulation that may benefit from the new drug. We provide some suggestions for future improvements in orphan drug development such as promoting paediatric drug investigations, better evaluation of the incidence and the prevalence, together with the natural history data, and the development of new primary outcomes.

Affiliation

Journal Details

This article was published in the following journal.

Name: Drugs
ISSN: 1179-1950
Pages:

Links

DeepDyve research library

PubMed Articles [35886 Associated PubMed Articles listed on BioPortfolio]

Concepts of Artificial Intelligence for Computer-Assisted Drug Discovery.

Artificial intelligence (AI), and, in particular, deep learning as a subcategory of AI, provides opportunities for the discovery and development of innovative drugs. Various machine learning approache...

Contemporary management of bronchiectasis in children.

: Bronchiectasis is increasingly recognised as a major cause of morbidity and mortality worldwide. It affects children of all ethnicities and socioeconomic backgrounds and represents a far greater bur...

Clinical drug development for dementia with Lewy bodies: past and present.

: Dementia with Lewy bodies (DLB) is an under-researched area despite being the second most common type of degenerative dementia after Alzheimer's disease. It is an area of unmet need with no approved...

Allostery in Drug Development.

Allosteric regulation is a ubiquitous strategy employed in nature to control cellular processes by regulating the affinities of biomolecules. Allosteric modulators are able to tune the protein/substra...

Stem cells: a path towards improved epilepsy therapies.

Despite the immense growth of new anti-seizure drugs (ASDs), approximately one-third of epilepsy patients remain resistant to current treatment options. Advancements in whole genome sequencing technol...

Clinical Trials [12543 Associated Clinical Trials listed on BioPortfolio]

Genetic and Electrophysiologic Study in Focal Drug-resistant Epilepsies

Genetic abnormalities in focal epilepsies are well known. A novel gene (DEPDC5) was identified, associated with various genotypes of epilepsies. Recently, DEPDC5 mutations were identified ...

Impact of Thermocoagulation During Invasive EEG Monitoring in Children With Focal Drug-resistant Epilepsies

When focal epilepsies become drug-resistant, it could be eligible for cortical surgical resection. Therefore, an invasive EEG monitoring with depth electrodes is often needed during presur...

South African Paediatric Surgical Outcomes Study (SAPSOS)

There are few data of paediatric peri-operative morbidity and mortality in South Africa. There is little information on the burden or profile of surgical disease in the paediatric populati...

Pharmacokinetics and Tolerability of Sulthiame

This preliminary pilot exploration aims at specifying the pharmacokinetic parameters of sulthiame, formulated as an immediate release tablet, thus helping to design proper clinical trials ...

To Evaluate the Paediatric Analgesia Slide

The Paediatric Analgesia Slide is a device developed for parents. Its development follows research into the Paediatric Analgesia Wheel, a device intended for use by hospital doctors. Previ...

Medical and Biotech [MESH] Definitions

Work consisting of a formal report giving details of the investigation and results of a medical or other scientific problem. When issued by a government agency or comparable official body, its contents may be classified, unclassified, or declassified with regard to security clearance. This publication type may also cover a scientific paper or article that records the current state or current position of scientific research and development. If so labeled by the editor or publisher, this publication type may be properly used for journal articles.

A stochastic process such that the conditional probability distribution for a state at any future instant, given the present state, is unaffected by any additional knowledge of the past history of the system.

Measurable biological parameters that serve for drug development, safety and dosing (DRUG MONITORING).

Agencies established under PL93-641 to coordinate, conduct, and implement state health planning activities. Two primary responsibilities are the preparation of an annual State Health Plan and giving assistance to the Statewide Health Coordinating Council.

State plans prepared by the State Health Planning and Development Agencies which are made up from plans submitted by the Health Systems Agencies and subject to review and revision by the Statewide Health Coordinating Council.

Quick Search


DeepDyve research library

Relevant Topics

Drug Discovery
Clinical Approvals Clinical Trials Drug Approvals Drug Delivery Drug Discovery Generics Drugs Prescription Drugs In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which drugs are dis...

Pharmacy
Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...


Searches Linking to this Article