Vir Biotechnology Receives Positive Opinion on Orphan Drug Designation for Tobevibart and Elebsiran in Chronic Hepatitis Delta from European Medicines Agency
Vir Biotechnology (VIR) has received a positive opinion from the European Medicines Agency's Committee for Orphan Medicinal Products for orphan drug designation of tobevibart and elebsiran in treating chronic hepatitis delta (CHD). The opinion follows encouraging preliminary data from the Phase 2 SOLSTICE trial, with 24-week data to be presented at AASLD The Liver Meeting. This development comes after receiving FDA fast track designation in June 2024. CHD is considered the most aggressive form of chronic viral hepatitis, with patients often progressing to cirrhosis within 5 years. The orphan designation could provide 10 years of market exclusivity in the EU upon approval.
Vir Biotechnology (VIR) ha ricevuto un parere positivo dal Comitato per i Medicinali Orfani dell'Agenzia Europea dei Medicinali per la designazione di farmaco orfano di tobevibart ed elebsiran nel trattamento dell'epatite delta cronica (CHD). Il parere segue dati preliminari incoraggianti provenienti dalla fase 2 dello studio SOLSTICE, con dati a 24 settimane che saranno presentati all'AASLD The Liver Meeting. Questo sviluppo arriva dopo aver ricevuto la designazione di percorso accelerato dalla FDA a giugno 2024. La CHD è considerata la forma più aggressiva di epatite virale cronica, con pazienti che spesso progrediscono verso la cirrosi entro 5 anni. La designazione orfana potrebbe garantire 10 anni di esclusività di mercato nell'UE una volta approvata.
Vir Biotechnology (VIR) ha recibido un dictamen positivo del Comité de Medicamentos Huérfanos de la Agencia Europea de Medicamentos para la designación de medicamento huérfano para tobevibart y elebsiran en el tratamiento de la hepatitis delta crónica (CHD). El dictamen sigue a datos preliminares alentadores del ensayo SOLSTICE de fase 2, con datos de 24 semanas que se presentarán en AASLD The Liver Meeting. Este desarrollo se produce tras recibir la designación de vía rápida por parte de la FDA en junio de 2024. La CHD se considera la forma más agresiva de hepatitis viral crónica, siendo los pacientes propensos a progresar a cirrosis en un plazo de 5 años. La designación huérfana podría proporcionar 10 años de exclusividad de mercado en la UE tras su aprobación.
Vir Biotechnology (VIR)는 유럽 의약품청의 고형약품위원회로부터 만성 델타 간염(CHD) 치료를 위한 토베비바르트와 엘렙시란의 고형의약품 지정에 대한 긍정적인 의견을 받았습니다. 이 의견은 SOLSTICE 2기 시험의 고무적인 초기 데이터에 따른 것으로, 24주 데이터를 AASLD The Liver Meeting에서 발표할 예정입니다. 이번 개발은 2024년 6월 FDA의 패스트트랙 지정을 받은 이후에 이루어졌습니다. CHD는 만성 바이러스성 간염의 가장 공격적인 형태로 간주되며, 환자들은 종종 5년 이내에 간경변으로 진행됩니다. 고형약품 지정은 승인 시 EU에서 10년의 시장 독점권을 제공할 수 있습니다.
Vir Biotechnology (VIR) a reçu un avis positif du Comité des médicaments orphelins de l'Agence européenne des médicaments pour la désignation de médicaments orphelins de tobevibart et élébsiran dans le traitement de l'hépatite delta chronique (CHD). Cet avis fait suite à des données préliminaires encourageantes de l'essai de phase 2 SOLSTICE, avec des données de 24 semaines qui seront présentées lors de l'AASLD The Liver Meeting. Ce développement intervient après avoir reçu la désignation de voie rapide de la FDA en juin 2024. La CHD est considérée comme la forme la plus agressive d'hépatite virale chronique, les patients progressant souvent vers la cirrhose dans les 5 ans. La désignation orpheline pourrait offrir 10 ans d'exclusivité sur le marché dans l'UE après approbation.
Vir Biotechnology (VIR) hat eine positive Empfehlung des Ausschusses für Orphan-Arzneimittel der Europäischen Arzneimittel-Agentur für die Einordnung von Tobevibart und Elebsiran als Orphan-Arzneimittel zur Behandlung der chronischen Deltahepatitis (CHD) erhalten. Diese Stellungnahme folgt auf ermutigende vorläufige Daten aus der Phase-2-Studie SOLSTICE, deren 24-Wochen-Daten auf dem AASLD The Liver Meeting präsentiert werden sollen. Diese Entwicklung erfolgt nach der Fast-Track-Zulassung durch die FDA im Juni 2024. CHD gilt als die aggressivste Form der chronischen Virushepatitis, wobei Patienten häufig innerhalb von 5 Jahren zu einer Zirrhose fortschreiten. Die Orphan-Einstufung könnte im Falle einer Genehmigung 10 Jahre Marktexklusivität in der EU bieten.
- Received positive opinion for EU orphan drug designation
- Previously secured FDA fast track designation in June 2024
- Potential for 10 years of market exclusivity in EU upon approval
- Addresses unmet medical need with no approved treatment in US
- None.
Insights
The EMA's positive opinion for orphan drug designation of tobevibart and elebsiran represents a significant regulatory milestone for Vir Biotechnology's CHD program. This development, coupled with the FDA's fast track designation, establishes a favorable regulatory pathway in both major markets.
The orphan designation offers substantial benefits including 10 years of market exclusivity in the EU, fee reductions and specialized scientific advice. For a rare disease like CHD, which affects a patient population but has severe consequences including cirrhosis within 5 years of infection, these incentives are important for commercial viability.
While the Phase 2 SOLSTICE trial data presentation is pending, the positive regulatory momentum suggests promising efficacy signals. The lack of approved treatments in the US and options globally positions Vir's combination therapy to potentially capture significant market share if approved.
– Orphan designation in E.U. supports development of treatments for life-threatening or chronically debilitating conditions with significant unmet medical need –
– Phase 2 SOLSTICE 24-week primary endpoint data for tobevibart and elebsiran in chronic hepatitis delta to be presented today at AASLD The Liver Meeting –
– Positive opinion on E.U. orphan drug designation follows
CHD is a severe, progressive liver disease caused by the hepatitis delta virus (HDV)1. It is considered the most aggressive form of chronic viral hepatitis2, with patients often progressing to cirrhosis and liver failure within 5 years of infection3. Currently, there is no approved treatment in
“Chronic hepatitis delta dramatically raises the risk of severe liver disease, including cancer, and eventually death, so new therapeutic options are urgently needed,” said Mark Eisner, M.D., M.P.H., Executive Vice President and Chief Medical Officer, Vir Biotechnology. “The COMP’s positive opinion on tobevibart and elebsiran reflects the potential of this combination to address a critical gap in hepatitis delta care. Our clinical data to date has been encouraging, suggesting that this approach could meaningfully improve outcomes for patients living with this devastating disease.”
The European Commission will evaluate the COMP’s positive opinion and consider tobevibart and elebsiran for orphan drug designation. This designation is for medicines intended to treat rare, life-threatening or chronically debilitating conditions where no other satisfactory treatment option is available, or where the medicine can be of significant benefit to those affected by a specific condition. The designation provides special incentives in the E.U., including access to specific scientific advice, fee reductions, and 10 years of market exclusivity once the medicine is approved.
In June 2024, the
Investor Conference Call
Vir Biotechnology will host an investor conference call on November 19, 2024 at 5.15 a.m. PT / 8.15 a.m. ET. A live webcast will be available on https://investors.vir.bio/ and will be archived on www.vir.bio for 30 days.
About the Phase 2 SOLSTICE Trial
SOLSTICE is a Phase 2 study to evaluate the safety, tolerability, and efficacy of tobevibart, alone or in combination with elebsiran, in patients with chronic hepatitis delta. This Phase 2 study is a multi-center, open-label, randomized study. Primary endpoints include proportion of participants with undetectable hepatitis delta virus (HDV) RNA (defined as HDV RNA equal or greater than 2 log10 decrease from baseline or below limit of detection) up to week 24, alanine aminotransferase (ALT) normalization (defined as ALT below upper limit of normal) up to week 24, and treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) up to 118 weeks. Secondary endpoints include proportion of participants with undetectable HDV RNA and different timepoints and up to 192 weeks. More information about this trial can be found at clinicaltrials.gov (NCT05461170).
About Tobevibart
Tobevibart is an investigational, broadly neutralizing monoclonal antibody targeting the hepatitis B surface antigen. It is designed to inhibit the entry of hepatitis B and hepatitis delta viruses into hepatocytes, and to reduce the level of circulating viral and subviral particles in the blood. Tobevibart, which incorporates Xencor’s Xtend™ and other Fc technologies, has been engineered to have an extended half-life and was identified using Vir Biotechnology’s proprietary monoclonal antibody discovery platform. Tobevibart is administered subcutaneously, and it is currently in clinical development for treatment of patients with chronic hepatitis B and patients with chronic hepatitis delta.
About Elebsiran
Elebsiran is an investigational, hepatitis B virus-targeting small interfering ribonucleic acid (siRNA) designed to degrade hepatitis B virus RNA transcripts and limit the production of hepatitis B surface antigen. Current data indicates that it has the potential to have direct antiviral activity against hepatitis B virus and hepatitis delta virus. Elebsiran is administered subcutaneously, and it is currently in clinical development for treatment of patients with chronic hepatitis B and patients with chronic hepatitis delta. It is the first asset in Vir Biotechnology’s collaboration with Alnylam Pharmaceuticals, Inc. to enter clinical studies.
About Vir Biotechnology, Inc.
Vir Biotechnology, Inc. is a clinical-stage biopharmaceutical company focused on powering the immune system to transform lives by discovering and developing medicines for serious infectious diseases and cancer. It’s clinical-stage portfolio includes infectious disease programs for chronic hepatitis delta and chronic hepatitis B infections and programs across several clinically validated targets in solid tumor indications. Vir Biotechnology also has a preclinical portfolio of programs across a range of other infectious diseases and oncologic malignancies. Vir Biotechnology routinely posts information that may be important to investors on its website.
References:
1 NIH National Institute of Diabetes and Digestive and Kidney Diseases Hepatitis D - NIDDK (nih.gov), accessed September 2024.
2 WHO Hepatitis Delta Factsheet - Hepatitis D (who.int), accessed September 2024
3 CDC What is Hepatitis D - FAQ | CDC
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “plan,” “potential,” “aim,” “expect,” “anticipate,” “promising” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Vir Biotechnology’s expectations and assumptions as of the date of this press release. Forward-looking statements contained in this press release include, but are not limited to, statements regarding Vir Biotechnology’s strategy and plans, the potential clinical effects of tobevibart and elebsiran, the potential benefits, safety and efficacy of tobevibart and elebsiran, the timing, nature and significance of data from Vir Biotechnology’s multiple ongoing trials evaluating tobevibart and elebsiran, Vir Biotechnology’s plans and expectations for its CHD and CHB programs, and risks and uncertainties associated with drug development and commercialization. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data or results observed during clinical trials or in data readouts; the occurrence of adverse safety events; risks of unexpected costs, delays or other unexpected hurdles; difficulties in collaborating with other companies; successful development and/or commercialization of alternative product candidates by Vir Biotechnology’s competitors; changes in expected or existing competition; delays in or disruptions to Vir Biotechnology’s business or clinical trials due to geopolitical changes or other external factors; and unexpected litigation or other disputes. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented. Other factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Vir Biotechnology’s filings with the
View source version on businesswire.com: https://www.businesswire.com/news/home/20241118366724/en/
Media
Arran Attridge
Senior Vice President, Corporate Communications
aattridge@vir.bio
Investors
Richard Lepke
Senior Director, Investor Relations
rlepke@vir.bio
Source: Vir Biotechnology, Inc.
FAQ
What did the EMA COMP recommend for Vir Biotechnology's (VIR) hepatitis delta treatment?
What regulatory designations has VIR received for tobevibart and elebsiran in 2024?
When will VIR present the SOLSTICE trial data for tobevibart and elebsiran?
