Handlungsempfehlungen zur Gentherapie der spinalen Muskelatrophie mit Onasemnogene Abeparvovec – AVXS-101

Ziegler, Andreas; Wilichowski, Ekkehard; Schara, Ulrike; Hahn, Andreas; Müller‐Felber, Wolfgang; Johannsen, Jessika; Hagen, Maja von der; Moers, Arpad von; Stoltenburg, Corinna; Saffari, Afshin; Walter, Maggie C.; Husain, Ralf A.; Pechmann, Astrid; Köhler, Cornelia; Horber, Veronka; Schwartz, Oliver; Kirschner, Janbernd

doi:10.1007/s00115-020-00919-8

Cited by 31 publications

(10 citation statements)

References 16 publications

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“…Post-treatment care including functional and laboratory assessments followed a German consensus paper [ 22 ]. Motor function was assessed by trained physiotherapists with standardized clinical assessments which are also used by the global Treat-NMD registry ( https://treat-nmd.org ), other SMA registries [ 23 – 25 ] and previous clinical trials in SMA [ 11 , 12 , 14 , 16 – 19 ].…”

Section: Methodsmentioning

confidence: 99%

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Stettner

Hasselmann²,

Tscherter

et al. 2023

BMC Neurol

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Background Spinal muscular atrophy (SMA) is a rare neuromuscular disorder leading to early death in the majority of affected individuals without treatment. Recently, targeted treatment approaches including Onasemnogene Abeparvovec (OA) were introduced. This study describes the first real-world experience with OA in Switzerland. Methods Prospective observational case series study using data collected within the Swiss Registry for Neuromuscular Disorders from SMA patients treated with OA. Development of motor, bulbar and respiratory function, appearance of scoliosis, and safety data (platelet count, liver function, and cardiotoxicity) were analyzed. Results Nine individuals were treated with OA and followed for 383 ± 126 days: six SMA type 1 (of which two with nusinersen pretreatment), one SMA type 2, and two pre-symptomatic individuals. In SMA type 1, CHOP Intend score increased by 28.1 from a mean score of 20.5 ± 7.6 at baseline. At end of follow-up, 50% of SMA type 1 patients required nutritional support and 17% night-time ventilation; 67% developed scoliosis. The SMA type 2 patient and two pre-symptomatically treated individuals reached maximum CHOP Intend scores. No patient required adaptation of the concomitant prednisolone treatment, although transient decrease of platelet count and increase of transaminases were observed in all patients. Troponin-T was elevated prior to OA treatment in 100% and showed fluctuations in 57% thereafter. Conclusions OA is a potent treatment for SMA leading to significant motor function improvements. However, the need for respiratory and especially nutritional support as well as the development of scoliosis must be thoroughly evaluated in SMA type 1 patients even in the short term after OA treatment.

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Section: Methodsmentioning

confidence: 99%

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Stettner

Hasselmann²,

Tscherter

et al. 2023

BMC Neurol

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“…Auch die Anforderungen an die Nachbetreuung, einschließlich deren Finanzierung, müssen geregelt sein (definierte Anweisungen für obligate Kontrolluntersuchungen). So erfordern die ambulante Nachsorge und Verlaufskontrolle nach Behandlung mit Zolgensma ® [30] in den ersten 12…”

Section: Qualifikation Der Leistungserbringer In Spezialisierten Zentrenunclassified

Arzneimittel für neuartige Therapien – Perspektiven, Chancen, Herausforderungen

Rascher

Klingebiel

Herting

et al. 2020

Monatsschr Kinderheilkd

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ZusammenfassungArzneimittel für neuartige Therapien (ATMP) wie somatische Gentherapie und Zelltherapie besitzen ein hohes therapeutisches Potenzial für Krankheiten, die sehr früh im Leben beginnen, und die bisher nicht behandelbar waren. Sie werden oft in einem sehr frühen Entwicklungsstadium zugelassen, wenn an wenigen Betroffenen die Wirksamkeit gezeigt wurde und sich ein bisher nie dagewesener Therapieerfolg auftut, vor allem, wenn die Therapie vor Eintritt von Organschäden greift. Dadurch ergeben sich für Pädiater neue arzneimittelrechtliche und ethische Fragen.Um die neuen Behandlungsmöglichkeiten adäquat einzusetzen, muss die Diagnose früher als bisher gestellt werden, oder neue Screeningmethoden müssen zur Verfügung stehen. Denkbar ist, dass das Neugeborenenscreening in zeitkritische Krankheiten in den ersten 72 h nach Geburt und ein genetisches Screening (z. B. in der 4. bis 5. Lebenswoche) aufgeteilt wird.ATMP sind bei ihrer Zulassung noch nicht in ausreichender Anzahl angewendet worden, sodass die notwendigen Erkenntnisse für Wirksamkeit und Sicherheit noch nicht vorliegen (Nutzen-Risiko-Verhältnis). Deswegen werden sie unter strengen Auflagen in spezialisierten Behandlungszentren nach Qualitätskriterien eingesetzt, die der Gemeinsame Bundesausschuss (G-BA) nach Beratung mit den Fachgesellschaften festlegt. Der Aufwand der Therapie und der Dokumentation des Verlaufes in Registern ist erheblich und muss entsprechend vergütet werden.Der Wert eines ATMP wird erst mit seiner breiteren Anwendung nach der Zulassung klar, ähnlich wie die Sicherheit eines Arzneimittels nicht mit der Zulassung vollumfänglich bekannt ist. Für die Pädiatrie ergeben sich neue Herausforderungen und Chancen.

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“…In the same year, a rst patient was treated with Onasemnogene Abeparvovec, the net drug cost amounted to € 1.945 million. (19) The conditional marketing authorization of Onasemnogene Abeparvovec by the EMA was granted in May 2020 which has led to a rapid increase in demand for the drug. During 2020, a total of 10 patients were treated with Onasemnogene Abeparvovec, resulting in total drug spending worth € 19.45 million plus import VAT.…”

Section: Resultsmentioning

confidence: 99%

Spinal Muscular Atrophy: The High Costs of Innovative Therapies for Rare Diseases

Brennenstuhl

Green

Störzinger

et al. 2021

Preprint

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Background: Despite numbers of prescriptions filled remaining constant, expenditures for drugs are rising. Among others, this is caused by high prices for orphan drugs and advanced therapy medicinal products (ATMPs). Despite attempts by policymakers to intervene, the increasing use of these therapies poses numerous challenges on the health care system.Results: Using data from the University Hospital Heidelberg, we found that the division of pediatric neurology is experiencing a strong increase in drug costs, caused by two pharmaceuticals for the treatment of spinal muscular atrophy (SMA), Nusinersen and Onasemnogene Abeparvovec. To put this finding in a broader context, we conducted a survey of 41 German SMA treatment centers revealing a general lack of human and infrastructure resources for therapy and follow-up care, which demonstrates insufficient reimbursement of treatment. To improve structural conditions, we propose that disease-independent registries for medication surveillance that comply with the rules of the European Union are needed as well as the development of a fair funding model.Conclusion: Innovative forms of therapy require a critical discussion and concise regulation of treatment application and follow-up reimbursement as well as international industry-independent registry work. Based on the previously described model of “evidence-based dynamic pricing” by the Techniker Krankenkasse, we propose a revised model which offers an internationally scalable solution to meet these challenges.

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Handlungsempfehlungen zur Gentherapie der spinalen Muskelatrophie mit Onasemnogene Abeparvovec – AVXS-101

Cited by 31 publications

References 16 publications

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Arzneimittel für neuartige Therapien – Perspektiven, Chancen, Herausforderungen

Spinal Muscular Atrophy: The High Costs of Innovative Therapies for Rare Diseases

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