Amgen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the marketing authorization of Prolia for the treatment of bone loss associated with long-term systemic glucocorticoid therapy in adult patients at increased risk of fracture.

“Today’s positive opinion by the CHMP is an important step for Prolia in helping patients suffering from bone loss associated with systemic glucocorticoid therapy,” said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. “Chronic use of oral glucocorticoids has been associated with an increase in spine and hip fractures,1 and, if approved, an expanded use of Prolia will provide patients and physicians across much of Europe with a new treatment option for this serious condition.”

Glucocorticoid medications, which are used to treat many inflammatory conditions such as chronic obstructive pulmonary disorder (COPD), asthma, multiple sclerosis and rheumatoid arthritis, can cause significant side effects, including bone loss.

The CHMP recommendation is supported by a Phase 3 randomized, double-blind, double-dummy, active-controlled study evaluating the safety and efficacy of Prolia compared with risedronate in patients receiving glucocorticoid treatment.4 The study included two patient groups: those on sustained glucocorticoid therapy and those newly initiating glucocorticoid therapy. The study met the primary endpoint (percent change from baseline in lumbar spine bone mass density [BMD] at 12 months, assessing non-inferiority) and all secondary endpoints (the percent changes from baseline in lumbar spine and total hip BMD at 12 and 24 months, assessing superiority). Study results showed that, in patients on sustained glucocorticoid therapy, Prolia treatment led to greater gains in BMD, compared with risedronate, both at the lumbar spine (4.4 percent versus 2.3 percent, respectively) and total hip (2.1 percent versus 0.6 percent, respectively). Similarly, in patients newly initiating glucocorticoid therapy, Prolia treatment led to greater increases in BMD, compared with risedronate, both at the lumbar spine (3.8 percent versus 0.8 percent, respectively) and total hip (1.7 percent versus 0.2 percent, respectively).

Adverse events and serious adverse events were similar between treatment groups and consistent with the known safety profile of Prolia. No serious adverse events were reported with a subject incidence of two percent or greater in either treatment group.

The CHMP positive opinion will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). Norway, Iceland and Liechtenstein, as members of the European Economic Area (EEA), will take corresponding decisions based on the decision of the EC.

The U.S. Food and Drug Administration (FDA) is currently reviewing a supplemental Biologics License Application for this expanded indication and has set a Prescription Drug User Fee Act (PDUFA) action date of May 28, 2018.

Bron: Amgen

Khondrion, a leading clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announced the publication of a scientific article reporting on the development and unique mode of action of KH176, a clinical-stage drug candidate for the treatment of mitochondrial diseases. The work was carried out by the Khondrion R&D team in collaboration with Inoviem Scientific and the Radboud University Medical Center. The results were published in Scientific Reports.

KH176 is an orally bio-available small molecule in development by Khondrion for the treatment of mitochondrial (-related) diseases. The present study reports on the early development and selection of KH176 as Khondrion’s lead candidate and further explains its mode of action in primary cells obtained from mitochondrial patients.

“Elevated Reactive Oxygen Species (ROS) levels and a high sensitivity to redox-stress are two frequent cellular consequences in mitochondrial disease. Interestingly, we could demonstrate in several patient-derived cells that KH176 is an attractive candidate to correct both pathological phenotypes, illustrating its unique potential for this group of patients” says Dr. Julien Beyrath, Chief Operating Officer at Khondrion and first author of the study.

KH176 displays a dual mode of action, targeting both ROS and Redox disturbances. “Using cutting-edge target deconvolution technology, we could identify peroxiredoxin enzyme as the primary and novel target of KH176” added Dr. Beyrath. Research is ongoing to further unravel the exact molecular mechanism of interaction of KH176 with the peroxiredoxin enzyme, which will help the Khondrion R&D team to further improve their drug candidates. “The results presented highlight the potential of KH176 as a single molecule to be effective in a large group of heterogeneous mitochondrial diseases, but also other diseases with underlying ROS and Redox perturbations” adds Prof. dr. Jan Smeitink, CEO at Khondrion and last author of the study.

Bron: Khondrion

In Australië is onderzoek gedaan naar de effectiviteit van Q-koortstesten voorafgaand aan vaccinatie. Het Australian Rickettsial Reference Laboratory en Sullivan Nicolaides Pathology vergeleken verschillende testen met elkaar, waaronder Q-detect™. Deze test, ontwikkeld door het Nederlandse Innatoss, blijkt veruit de beste test voor screening op Q-koorts. De resultaten werden vandaag gepresenteerd tijdens het Europese ECCMID congres in Madrid.

Het vaccineren van werknemers die een hoger risico op Q-koorts lopen is in Australië verplicht. Het vaccin dat hiervoor gebruikt wordt kan echter bijwerkingen geven bij mensen die al eerder zijn blootgesteld aan de Q-koortsbacterie. Deze mensen mogen niet gevaccineerd worden. Om hen uit te sluiten, wordt een screening uitgevoerd. Hiervoor moet een antistoftest gedaan worden, en als die negatief is een huidtest.

In het Australische vaccinatie-onderzoek onder leiding van Q-koortsexpert Dr. Stephen Graves werden verschillende Q-koortstesten vergeleken. De Nederlandse Q-detect test, die tijdens de Nederlandse Q-koortsepidemie werd uitgevonden door het RadboudUMC, werd voor het eerst in een dergelijk onderzoek meegenomen.

Nederlandse Q-detect test blijkt veruit de beste

De resultaten spreken voor zich. Van alle testen in het onderzoek is Q-detect de enige die het predicaat “goede test” krijgt. Het is de enige test in het onderzoek die alle 8 personen die eerder aan de Q-koortsbacterie werden blootgesteld, wist te identificeren.

Buitengewoon verrassend zijn de resultaten van de testen die op dit moment worden gebruikt voor screening.. Zo vindt men met de huidtest slechts 3 van de 8 personen. Zelfs in combinatie met de hoogst aangeschreven antistoftest, de IFA test, werden niet alle personen gevonden.

“Q-detect is niet alleen de meest gevoelige test in ons onderzoek, maar de test is ook de veel minder ingrijpend en makkelijker inzetbaar in Australië dan de huid- en antistoftest die op dit moment gebruikt worden,” aldus Dr. Graves.

Nederlandse overheid aan zet

Op dit moment is alleen in Australië het menselijke Q-koortsvaccin beschikbaar. In Nederland dus nog niet. Barbara Schimmer, onderzoeker bij het RIVM, adviseerde vorige week bij de verdediging van haar proefschrift om vaccinatie serieus te overwegen bij beroepsgroepen met een hoog risico op Q-koorts.

Anja Garritsen, directeur van Innatoss, ziet mogelijkheden voor de Nederland om voorop te lopen: “Er wordt in Europa nog niet gevaccineerd tegen Q-koorts. Het Australische vaccin wordt echter al heel lang succesvol ingezet. Het RIVM heeft ook al laten zien dat de bijwerkingen bij een goede screening vooraf beperkt zijn. Hopelijk gaat de Nederlandse overheid op korte termijn een vaccinatie-proef starten waarbij gescreend wordt met de nieuwe Q-detect test. Zo kan de Q-koortsvaccinatie op een zo eenvoudig en effectief mogelijke manier ingevoerd worden ten behoeve van deze beroepsgroep”.

Q-detect is niet de enige Q-koorts gerelateerde innovatie van Innatoss. Het bedrijf uit Oss werkt ook mee aan de ontwikkeling van een nieuw Q-koortsvaccin, zonder de nare bijwerkingen die het Australische vaccin kan hebben. Dit project wordt gefinancierd door het Amerikaanse ministerie van Defensie.

De originele presentatie als wetenschappelijke poster op ECCMID 2018 is hier te vinden

Bron: q-koorts.nl

MDxHealth SA, a world leader in molecular diagnostics for urological cancers, announced the inclusion of SelectMDx for Prostate Cancer, its non-invasive ‘liquid biopsy’ test that helps to identify patients at increased risk of having aggressive prostate cancer, in the Dutch Diagnosis Related Groups (DRG) reimbursement system.

The DRG reimbursement system was introduced in the Netherlands in 2005. It specifies the reimbursable amounts for 30,000 different treatments and is regulated by the Dutch Healthcare Authority (NZA). Diagnosing and treatment of prostate cancer are also reimbursed under the DRG-system. The SelectMDx test may be used under the DRG-system. Reimbursement amounts are directly negotiated between the healthcare insurers and the healthcare providers. Several Dutch hospitals have already incorporated the SelectMDx test in their prostate cancer diagnostic workflow.

Dr. Jan Groen, Chief Executive Officer of MDxHealth, commented: “This is a positive development for MDxHealth and serves as an important step in our strategy to increase the adoption and acceptance of our SelectMDx CE marked test in Europe among urologists and payors. We believe that in light of the ageing male population in the US and Europe, the use of SelectMDx in active monitoring and primary care settings could quadruple the market opportunity for SelectMDx in the mid-term to more than 4 million patients annually in the US and Europe. With the EAU guidelines inclusion for SelectMDx announced last month, we look forward to providing further updates on our progress in European markets.”

Bron: MDxHealth