uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease
uniQure N.V. (NASDAQ: QURE) announced that the U.S. FDA has granted Orphan Drug Designation to AMT-191, their investigational gene therapy for Fabry disease. This follows the dosing of the first patient in a U.S. multi-center, open-label Phase I/IIa trial in August 2024. AMT-191 is a one-time intravenously administered AAV5-based gene therapy targeting the liver to produce GLA protein.
The Phase I/IIa trial will include two cohorts of up to six adult male patients each, with low and high doses. Patients will be followed for 24 months to assess safety, tolerability, and early efficacy signs. The Orphan Drug Designation provides incentives including tax credits, grants, fee waivers, and seven years of market exclusivity upon approval.
uniQure N.V. (NASDAQ: QURE) ha annunciato che la FDA statunitense ha concesso la Designazione di Farmaco Orfano a AMT-191, la loro terapia genica in fase di sperimentazione per la malattia di Fabry. Questo segue la somministrazione del primo paziente in uno studio clinico multicentrico, aperto, di Fase I/IIa negli Stati Uniti nell'agosto 2024. AMT-191 è una terapia genica basata su AAV5 somministrata una sola volta per via intravenosa, mirata al fegato per produrre la proteina GLA.
Lo studio di Fase I/IIa includerà due coorti di fino a sei pazienti maschi adulti ciascuna, con dosi bassa e alta. I pazienti saranno seguiti per 24 mesi per valutare la sicurezza, la tollerabilità e i primi segni di efficacia. La Designazione di Farmaco Orfano fornisce incentivi tra cui crediti d'imposta, sovvenzioni, esenzioni dalle tasse e sette anni di esclusività di mercato all'approvazione.
uniQure N.V. (NASDAQ: QURE) anunció que la FDA de EE. UU. ha otorgado la Designación de Medicamento Huérfano a AMT-191, su terapia génica en investigación para la enfermedad de Fabry. Esto sigue a la dosificación del primer paciente en un ensayo clínico multicéntrico, abierto, de Fase I/IIa en EE. UU. en agosto de 2024. AMT-191 es una terapia génica basada en AAV5 administrada por vía intravenosa una sola vez, dirigida al hígado para producir la proteína GLA.
El ensayo de Fase I/IIa incluirá dos cohortes de hasta seis pacientes masculinos adultos cada una, con dosis baja y alta. Los pacientes serán seguidos durante 24 meses para evaluar la seguridad, la tolerabilidad y los primeros signos de eficacia. La Designación de Medicamento Huérfano proporciona incentivos como créditos fiscales, subvenciones, exenciones de tarifas y siete años de exclusividad en el mercado tras la aprobación.
uniQure N.V. (NASDAQ: QURE)는 미국 FDA가 파브리병에 대한 조사 중인 유전자 치료제 AMT-191에 희귀의약품 지정을 부여했다고 발표했습니다. 이는 2024년 8월 미국의 다기관 오픈 라벨 1상/2a 시험에서 첫 환자에게 용량을 투여한 이후의 일입니다. AMT-191은 AAV5 기반의 정맥주사 단회 투여 유전자 치료제로, 간을 표적으로 하여 GLA 단백질을 생성하도록 설계되었습니다.
1상/2a 시험에는 저용량과 고용량 각각 최대 6명의 성인 남성 환자로 구성된 두 개의 코호트가 포함될 것입니다. 환자들은 안전성, 내약성 및 초기 효능 징후를 평가하기 위해 24개월 동안 추적 관찰됩니다. 희귀의약품 지정을 통해 세금 크레딧, 보조금, 수수료 면제 및 승인 후 7년의 시장 독점권 등 인센티브가 제공됩니다.
uniQure N.V. (NASDAQ: QURE) a annoncé que la FDA américaine a accordé la désignation de médicament orphelin à AMT-191, leur thérapie génique expérimentale pour la maladie de Fabry. Cela fait suite à la dose administrée au premier patient dans un essai clinique multicentrique, ouvert, de phase I/IIa aux États-Unis en août 2024. AMT-191 est une thérapie génique basée sur AAV5 administrée une fois par voie intraveineuse, ciblant le foie pour produire la protéine GLA.
L'essai de phase I/IIa comprendra deux cohortes de jusqu'à six patients masculins adultes chacune, avec des doses faibles et élevées. Les patients seront suivis pendant 24 mois pour évaluer la sécurité, la tolérance et les premiers signes d'efficacité. La désignation de médicament orphelin offre des incitations, y compris des crédits d'impôt, des subventions, des exonérations de frais et sept années d'exclusivité sur le marché après approbation.
uniQure N.V. (NASDAQ: QURE) hat angekündigt, dass die U.S. FDA AMT-191, ihre experimentelle Gentherapie für die Fabry-Krankheit, die Orphan Drug Designation erhalten hat. Dies folgt der Verabreichung des ersten Patienten in einer multizentrischen, offenen Phase I/IIa-Studie in den USA im August 2024. AMT-191 ist eine einmalig intravenös verabreichte AAV5-basierte Gentherapie, die auf die Leber abzielt, um das GLA-Protein zu produzieren.
Die Phase I/IIa-Studie wird zwei Kohorten von bis zu sechs erwachsenen männlichen Patienten umfassen, jeweils mit niedrigen und hohen Dosen. Die Patienten werden über einen Zeitraum von 24 Monaten beobachtet, um Sicherheit, Verträglichkeit und erste Wirksamkeitszeichen zu bewerten. Die Orphan Drug Designation bietet Anreize wie Steuervergünstigungen, Zuschüsse, Gebührenbefreiungen und sieben Jahre Marktexklusivität nach Genehmigung.
- FDA granted Orphan Drug Designation to AMT-191 for Fabry disease treatment
- Phase I/IIa clinical trial of AMT-191 has begun with first patient dosed
- Potential for one-time treatment addressing limitations of current chronic therapies
- Orphan Drug Designation provides incentives and market exclusivity upon approval
- Early-stage clinical trial with efficacy yet to be proven
- patient population (up to 12 adult males) in the initial study
LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024, uniQure announced the dosing of the first patient in its U.S., multi-center, open-label Phase I/IIa trial of AMT-191.
“This important designation highlights the need for new gene therapies like AMT-191 for patients with Fabry disease with the potential of delivering meaningful benefit given the suboptimal effectiveness of current chronic treatments,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “This designation supports our Phase I/IIa clinical trial and we look forward to rapidly generating clinical proof-of-concept data and providing initial data in 2025.”
In patients with Fabry disease, a pathogenic variant in the galactosidase alpha (GLA) gene leads to α-galactosidase A (aGAL-A) enzyme deficiency, which in turn results in a progressive accumulation of lipids in multiple cell types, including kidney and heart cells, eventually resulting in a multi-system disorder. AMT-191 is a one-time intravenously administered investigational AAV5-based gene therapy that uses a proprietary, highly potent promoter to deliver a GLA transgene designed to target the liver to produce GLA protein.
The Phase I/IIa clinical trial of AMT-191 will be conducted in the United States. The multicenter, open-label trial consists of two cohorts with up to six adult male patients each: a low-dose cohort of 6x1013 gc/kg and a high-dose cohort of 3x1014 gc/kg delivered through a one-time intravenous infusion. Patients will continue to receive their regular enzyme replacement therapy until the criteria for withdrawal is met and will be followed for a period of 24 months. The trial will explore the safety, tolerability, and early signs of efficacy by measuring the expression of lysosomal enzyme aGLA-A. Additional details are available on www.clinicaltrials.gov (NCT06270316).
The FDA’s Orphan Drug Designation provides a special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the United States. Orphan drug status provides certain incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials as well as seven years of market exclusivity in the United States following drug approval.
About Fabry Disease
Fabry disease is an Xlinked- genetic disorder resulting from a deficiency of GLA. Based on a 2020 study published in the Journal of Therapeutics and Clinical Risk Management, the prevalence is estimated to be between one in 40,000 and one in 117,000 individuals. The current standard of care for Fabry disease is bi-weekly infusions of enzyme replacement therapy, a treatment with limited effectiveness in many patients due to poor cross-correction, with inefficient clearance of substrates in the target organs, in particular the kidney and the heart.
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com
uniQure Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements regarding the availability of initial clinical and proof-of-concept data in the Company’s open-label U.S. Phase I/IIa trial for Fabry disease; the Company’s plans to announce initial clinical data in 2025; and the AMT-191 trial design and the differentiated profile relative to other Fabry programs currently in clinical development. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, without limitation, risks associated with the timing and advancement of the Company’s clinical programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; risks associated with the implementation of the Company’s restructuring plans; the Company’s ability to continue to build and maintain Company infrastructure and personnel needed to achieve its goals following planned workforce reductions; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed February 28, 2024, its Quarterly Reports on Form 10-Q filed May 7, 2024 and August 1, 2024, and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.
uniQure Contacts:
| FOR INVESTORS: | FOR MEDIA: |
| Chiara Russo | Tom Malone |
| Direct: 617-306-9137 | Direct: 339-970-7558 |
| Mobile: 617-306-9137 | Mobile:339-223-8541 |
| c.russo@uniQure.com | t.malone@uniQure.com |
FAQ
What is AMT-191 and what disease does it target?
What designation did the FDA grant to AMT-191?
When did uniQure begin its clinical trial for AMT-191?
How is AMT-191 administered to patients?
What are the benefits of receiving Orphan Drug Designation for AMT-191?
