Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Transthyretin (ATTR) Amyloidosis with Cardiomyopathy
Intellia Therapeutics (NASDAQ:NTLA) has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for nexiguran ziclumeran (nex-z) in treating transthyretin (ATTR) amyloidosis with cardiomyopathy. The therapy, also known as NTLA-2001, has demonstrated significant promise with rapid, durable and consistent reductions in serum TTR after a single dose, positively impacting disease progression markers.
The RMAT designation, established under the 21st Century Cures Act, will help expedite development and review processes. Benefits include early FDA interactions, discussions on surrogate endpoints for potential accelerated approval, and possible priority review of the biologics license application (BLA). Nex-z has also secured RMAT designations for polyneuropathy and Orphan Drug Designation from both U.S. FDA and European Commission.
Intellia Therapeutics (NASDAQ:NTLA) ha ricevuto la designazione di Terapia Avanzata in Medicina Rigenerativa (RMAT) dalla FDA per nexiguran ziclumeran (nex-z) nel trattamento dell'amiloidosi da transtiretina (ATTR) con cardiomiopatia. La terapia, nota anche come NTLA-2001, ha dimostrato un significativo potenziale con riduzioni rapide, durature e costanti del TTR sierico dopo una singola dose, influenzando positivamente i marcatori di progressione della malattia.
La designazione RMAT, stabilita sotto il 21° secolo Cures Act, aiuterà ad accelerare i processi di sviluppo e revisione. I benefici includono interazioni anticipate con la FDA, discussioni sui punti finali surrogati per una potenziale approvazione accelerata e una possibile revisione prioritaria della domanda di licenza biologica (BLA). Nex-z ha anche ottenuto designazioni RMAT per la polineuropatia e la designazione di Farmaco Orfano sia dalla FDA degli Stati Uniti che dalla Commissione Europea.
Intellia Therapeutics (NASDAQ:NTLA) ha recibido la designación de Terapia Avanzada en Medicina Regenerativa (RMAT) de la FDA para nexiguran ziclumeran (nex-z) en el tratamiento de la amiloidosis por transtiretina (ATTR) con cardiomiopatía. La terapia, también conocida como NTLA-2001, ha demostrado una promesa significativa con reducciones rápidas, duraderas y consistentes en el TTR sérico tras una sola dosis, impactando positivamente los marcadores de progresión de la enfermedad.
La designación RMAT, establecida bajo la Ley de Curas del Siglo XXI, ayudará a acelerar los procesos de desarrollo y revisión. Los beneficios incluyen interacciones tempranas con la FDA, discusiones sobre puntos finales sustitutos para una posible aprobación acelerada y una posible revisión prioritaria de la solicitud de licencia biológica (BLA). Nex-z también ha asegurado designaciones RMAT para polineuropatía y designación de Medicamento Huérfano tanto de la FDA de EE. UU. como de la Comisión Europea.
Intellia Therapeutics (NASDAQ:NTLA)는 심장병을 동반한 트랜스티레틴(ATTR) 아밀로이드증 치료를 위해 넥시구란 지클루메란(nex-z)에 대해 FDA의 재생의학 고급 치료(RMAT) 지정을 받았습니다. NTLA-2001로도 알려진 이 치료법은 단일 투여 후 혈청 TTR의 빠르고 지속적이며 일관된 감소를 보여주어 질병 진행 마커에 긍정적인 영향을 미쳤습니다.
RMAT 지명은 21세기 치료법 법안에 따라 설정되었으며, 개발 및 검토 프로세스를 가속화하는 데 도움이 됩니다. 이점으로는 FDA와의 조기 상호작용, 잠재적 가속 승인에 대한 대체 최종점 논의, 생물학적 제제 라이센스 신청(BLA)의 우선 검토 가능성이 포함됩니다. Nex-z는 또한 다발신경병증에 대한 RMAT 지명과 미국 FDA 및 유럽 위원회로부터의 고아약 지명을 확보했습니다.
Intellia Therapeutics (NASDAQ:NTLA) a reçu la désignation de Thérapie Avancée en Médecine Régénérative (RMAT) de la FDA pour nexiguran ziclumeran (nex-z) dans le traitement de l'amyloïdose à transthyrétine (ATTR) avec cardiomyopathie. Cette thérapie, également connue sous le nom de NTLA-2001, a montré un potentiel significatif avec des réductions rapides, durables et cohérentes du TTR sérique après une seule dose, ayant un impact positif sur les marqueurs de progression de la maladie.
La désignation RMAT, établie dans le cadre de la loi sur les Cures du XXIe siècle, aidera à accélérer les processus de développement et d'examen. Les avantages incluent des interactions précoces avec la FDA, des discussions sur des points finaux substituts pour une approbation potentielle accélérée, et une possible révision prioritaire de la demande de licence biologique (BLA). Nex-z a également obtenu des désignations RMAT pour la polyneuropathie et la désignation de Médicament Orphelin tant de la FDA américaine que de la Commission Européenne.
Intellia Therapeutics (NASDAQ:NTLA) hat von der FDA die Bezeichnung für Regenerative Medizin mit fortgeschrittener Therapie (RMAT) für nexiguran ziclumeran (nex-z) zur Behandlung von Transthyretin (ATTR) Amyloidose mit Kardiomyopathie erhalten. Die Therapie, auch bekannt als NTLA-2001, hat vielversprechende Ergebnisse mit schnellen, dauerhaften und konsistenten Reduktionen des Serum-TTR nach einer einzigen Dosis gezeigt, was sich positiv auf die Krankheitsprogressionsmarker auswirkt.
Die RMAT-Bezeichnung, die im Rahmen des 21st Century Cures Act eingerichtet wurde, wird helfen, die Entwicklungs- und Überprüfungsprozesse zu beschleunigen. Zu den Vorteilen gehören frühzeitige Interaktionen mit der FDA, Diskussionen über surrogate Endpunkte für eine potenzielle beschleunigte Genehmigung und eine mögliche priorisierte Überprüfung des Antrags auf biologische Lizenz (BLA). Nex-z hat auch RMAT-Bezeichnungen für Polyneuropathie sowie die Orphan Drug Designation sowohl von der US-FDA als auch von der Europäischen Kommission erhalten.
- Received FDA RMAT designation, potentially accelerating approval process
- Demonstrated strong efficacy with single-dose treatment showing consistent TTR reductions
- Secured multiple regulatory designations (RMAT for two indications, Orphan Drug status in US and EU)
- None.
Insights
The FDA's RMAT designation for Intellia's nexiguran ziclumeran represents a significant regulatory milestone that could substantially accelerate the development timeline for this CRISPR-based therapy. This designation is particularly valuable as it's granted only to therapies showing preliminary clinical evidence indicating potential to address unmet medical needs for serious conditions.
ATTR amyloidosis with cardiomyopathy is a progressive, often fatal condition caused by misfolded TTR protein depositing in heart tissue. What makes nex-z scientifically noteworthy is its one-time treatment approach - using in vivo CRISPR gene editing to reduce TTR protein production at its source, contrasting with current therapies requiring ongoing administration.
The regulatory benefits are substantial: early FDA interactions, guidance on surrogate endpoints for potential accelerated approval, and possible priority review. This could trim years from the traditional approval pathway. Combined with the previously granted RMAT for polyneuropathy and Orphan Drug Designations in both US and EU, Intellia has secured an exceptionally favorable regulatory position across the ATTR disease spectrum.
The reference to "unprecedented rapid, durable and consistent reductions in serum TTR" suggests the therapy is demonstrating the sustained protein knockdown essential for meaningful clinical benefit in amyloidosis, where disease progression correlates with TTR levels.
This RMAT designation significantly strengthens Intellia's position in the competitive ATTR therapeutic landscape. The expedited regulatory pathway offers concrete financial advantages by potentially reducing development time and associated costs - critical for a clinical-stage biotech with cash resources relative to its larger competitors.
The cardiomyopathy indication represents the larger commercial opportunity within ATTR, with estimates suggesting approximately 200,000-500,000 patients worldwide. Current standard-of-care treatments for ATTR-CM (like tafamidis) generate billions in annual revenue but require lifelong administration at substantial cost.
Intellia's gene editing approach could disrupt this market with its one-time treatment paradigm. If successful, this could translate to premium pricing power while simultaneously reducing long-term healthcare system costs - an increasingly important consideration in reimbursement decisions.
The dual RMAT designations across both major ATTR indications (cardiomyopathy and polyneuropathy) creates a compelling regulatory package that enhances the asset's commercial potential and strategic value. With Regeneron as development partner, Intellia also has access to substantial development resources while maintaining significant economics in the program.
While still years from potential commercialization, each regulatory milestone de-risks the development pathway and enhances the therapy's probability of technical and commercial success.
CAMBRIDGE, Mass., March 26, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).
“This is a meaningful step forward in our efforts to accelerate the development of nex-z to bring this potentially transformative treatment to patients as quickly as possible,” said Intellia President and Chief Executive Officer John Leonard, M.D. “In both cardiomyopathy and polyneuropathy, nex-z has been shown, after a single dose, to lead to unprecedented rapid, durable and consistent reductions in serum TTR, which positively impacts markers of disease progression.”
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).
Nex-z has been granted Regenerative Medicine Advanced Therapy designations by the U.S. FDA for both cardiomyopathy and polyneuropathy. Nex-z has also been granted Orphan Drug Designation by the U.S. FDA and European Commission.
About nexiguran ziclumeran (nex-z, also known as NTLA-2001)
Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.
About Intellia Therapeutics
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.
Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding: the safety, efficacy, success and advancement of its clinical program for nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) pursuant to its clinical trial applications and investigational new drug application, including the potential of NTLA-2001 to be a transformative treatment for patients with ATTR-CM; and the expected timing of regulatory filings, completion of clinical trials, submission of marketing authorizations, and commercialization.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; and uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including nex-z; risks related to the results of preclinical or clinical studies, including that they may not be positive or predictive of future results; the risk that one or more of Intellia’s product candidates, including nex-z, will not be successfully developed and commercialized; and risks related to Intellia’s reliance on collaborations, including that its collaboration with Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
Intellia Contacts:
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Senior Manager, Investor Relations
brittany.chaves@intelliatx.com
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FAQ
What does the RMAT designation mean for Intellia's NTLA-2001 development timeline?
How effective is Intellia's nex-z (NTLA-2001) in treating ATTR amyloidosis?
What regulatory designations has NTLA-2001 received as of March 2025?
What conditions is Intellia's NTLA-2001 being developed to treat?
