Arrowhead Pharmaceuticals Receives FDA Breakthrough Therapy Designation for Plozasiran
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has received FDA Breakthrough Therapy designation for plozasiran, an investigational drug for familial chylomicronemia syndrome (FCS). The PALISADE Phase 3 results showed plozasiran reduced triglycerides by 80% from baseline and decreased the risk of acute pancreatitis by 83% in FCS patients. There are currently no FDA-approved treatments for FCS, a severe genetic disease characterized by extremely high triglyceride levels. Arrowhead plans to submit a New Drug Application to the FDA by year-end 2024 and seek additional global regulatory approvals thereafter. Plozasiran has also received Orphan Drug Designation and Fast Track Designation from the FDA, and Orphan Drug Designation from the European Medicines Agency.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha ricevuto la designazione di terapia innovativa dalla FDA per plozasiran, un farmaco in fase di sperimentazione per la sindrome da chilomicronemia familiare (FCS). I risultati della fase 3 PALISADE hanno mostrato che plozasiran ha ridotto i trigliceridi del 80% rispetto al valore di partenza e ha diminuito il rischio di pancreatite acuta del 83% nei pazienti con FCS. Attualmente non ci sono trattamenti approvati dalla FDA per la FCS, una grave malattia genetica caratterizzata da livelli estremamente elevati di trigliceridi. Arrowhead prevede di presentare una Nuova Domanda di Farmaco alla FDA entro la fine dell'anno 2024 e di cercare ulteriori approvazioni normative globali successivamente. Plozasiran ha anche ricevuto la designazione di Farmaco Orfano e Fast Track dalla FDA, e la designazione di Farmaco Orfano dall'Agenzia Europea per i Medicinali.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha recibido la designación de terapia revolucionaria por parte de la FDA para plozasiran, un medicamento en investigación para el síndrome de quilomicronemia familiar (FCS). Los resultados de la fase 3 PALISADE mostraron que plozasiran redujo los triglicéridos en un 80% desde la línea de base y disminuyó el riesgo de pancreatitis aguda en un 83% en pacientes con FCS. Actualmente no hay tratamientos aprobados por la FDA para la FCS, una enfermedad genética severa caracterizada por niveles extremadamente altos de triglicéridos. Arrowhead planea presentar una Solicitud de Nueva Droga a la FDA para finales de 2024 y buscar aprobaciones regulatorias globales adicionales después. Plozasiran también ha recibido la designación de Medicamento Huérfano y la designación de Vía Rápida de la FDA, así como la designación de Medicamento Huérfano de la Agencia Europea de Medicamentos.
Arrowhead Pharmaceuticals (NASDAQ: ARWR)는 플로자시란에 대해 FDA의 국가적 혁신 치료제 지정을 받았습니다. 이는 유전성 킬로미크론혈증 증후군 (FCS)에 대한 연구용 의약품입니다. PALISADE 3상 시험 결과, 플로자시란은 기준선에서부터 80%의 트리글리세리드 감소를 나타내었고, FCS 환자에서 급성 췌장염의 위험을 83% 감소시켰습니다. 현재 FCS에 대한 FDA 승인 치료제가 없으며, 이는 극도로 높은 트리글리세리드 수치로 특징지어지는 심각한 유전 질환입니다. Arrowhead는 2024년 연말까지 FDA에 새로운 의약품 신청서를 제출하고 이후 추가적인 글로벌 규제 승인을 받을 계획입니다. 플로자시란은 FDA로부터 희귀의약품 지정을 받았으며, 유럽 의약품청에서도 희귀의약품으로 지정되었습니다.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) a reçu la désignation de thérapie innovante de la FDA pour plozasiran, un médicament en cours d'étude pour le syndrome de chylomicronémie familiale (FCS). Les résultats de l'étude PALISADE de phase 3 ont montré que plozasiran a réduit les triglycérides de 80% par rapport à la ligne de base et a diminué le risque de pancréatite aiguë de 83% chez les patients atteints de FCS. Actuellement, il n'existe pas de traitements approuvés par la FDA pour la FCS, une maladie génétique sévère caractérisée par des niveaux de triglycérides extrêmement élevés. Arrowhead prévoit de soumettre une demande de nouveau médicament à la FDA d'ici la fin de l'année 2024 et de chercher ensuite d'autres approbations réglementaires mondiales. Plozasiran a également reçu la désignation de Médicament Orphelin et la désignation de Traitement Accéléré de la FDA, ainsi que la désignation de Médicament Orphelin de l'Agence Européenne des Médicaments.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) hat die FDA-Zulassung für eine bahnbrechende Therapie für plozasiran, ein investigatives Medikament für das familiale Chylomikronämie-Syndrom (FCS), erhalten. Die Ergebnisse der PALISADE-Phase-3-Studie zeigten, dass plozasiran die Triglyceride im Vergleich zum Ausgangswert um 80% reduzierte und das Risiko einer akuten Pankreatitis bei FCS-Patienten um 83% senkte. Derzeit gibt es keine von der FDA zugelassenen Behandlungsmöglichkeiten für FCS, eine schwere genetische Erkrankung, die durch extrem hohe Triglyceridspiegel gekennzeichnet ist. Arrowhead plant, bis Ende 2024 einen Antrag auf Zulassung eines neuen Arzneimittels bei der FDA einzureichen und anschließend weitere globale regulatorische Genehmigungen zu beantragen. Plozasiran hat auch die Orphan Drug Designation und Fast Track Designation von der FDA sowie die Orphan Drug Designation von der Europäischen Arzneimittel-Agentur erhalten.
- FDA Breakthrough Therapy designation received for plozasiran
- PALISADE Phase 3 results show 80% reduction in triglycerides
- 83% reduction in acute pancreatitis risk for FCS patients
- No current FDA-approved treatments for FCS, giving plozasiran a potential first-mover advantage
- New Drug Application planned for submission by year-end 2024
- None.
Insights
The FDA's Breakthrough Therapy designation for plozasiran is a significant milestone for Arrowhead Pharmaceuticals and patients with familial chylomicronemia syndrome (FCS). This designation, based on the impressive 80% reduction in triglycerides and 83% reduction in acute pancreatitis risk from the PALISADE Phase 3 trial, could accelerate the drug's approval process.
With no current FDA-approved treatments for FCS, plozasiran has the potential to become a first-in-class therapy. The planned New Drug Application submission by year-end 2024 could lead to a faster market entry, potentially providing a much-needed treatment option for this rare genetic disease. This development positions Arrowhead as a pioneer in FCS treatment, potentially opening up a new market segment with competition.
Arrowhead Pharmaceuticals' breakthrough designation for plozasiran could have substantial financial implications. As a potential first-to-market drug for FCS, plozasiran could command premium pricing, typical for orphan drugs. The expedited review process could lead to earlier-than-expected revenue streams, positively impacting Arrowhead's financial outlook.
Investors should note the multiple regulatory designations (Orphan Drug, Fast Track, Breakthrough Therapy) which not only speed up the approval process but also provide market exclusivity and potential tax credits. These factors could significantly enhance the drug's profitability. However, the patient population for FCS means the overall market size may be constrained, though this could be offset by potential label expansions for other triglyceride-related conditions in the future.
The Breakthrough Therapy designation for plozasiran could significantly boost Arrowhead's market position in the rare disease space. With no current FDA-approved treatments for FCS, Arrowhead is poised to capture the entire market share upon approval. This first-mover advantage could establish Arrowhead as the go-to company for FCS treatment, potentially leading to increased investor interest and partnerships in the rare disease sector.
The planned global regulatory submissions indicate Arrowhead's ambition for international market penetration. This could lead to a substantial expansion of the addressable market. However, investors should be aware of the challenges in rare disease drug commercialization, including patient identification and market access. Arrowhead's success with plozasiran could serve as a catalyst for its broader pipeline, potentially increasing the company's overall valuation.
PALISADE Phase 3 results demonstrate plozasiran reduced triglycerides by
“There are currently no FDA-approved therapies to specifically treat FCS, leaving physicians with very few options to help their patients,” said Chris Anzalone, Ph.D., President and CEO of Arrowhead. “Results from clinical studies of investigational plozasiran have been highly encouraging and strongly supportive of further development and commercialization in multiple patient populations. Receiving FDA Breakthrough Therapy designation for plozasiran provides important benefits and the potential to expedite the process of getting plozasiran to the patients who need it.”
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on clinically significant endpoints2. Plozasiran has also previously been granted Orphan Drug Designation and Fast Track Designation by the FDA and Orphan Drug Designation by the European Medicines Agency.
Arrowhead intends to submit a New Drug Application to the FDA by year-end 2024 and plans to seek regulatory approval with additional global regulatory authorities thereafter.
About Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome (FCS) is a severe and rare genetic disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, the therapeutic options that can adequately treat FCS are limited.
About Plozasiran
Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of APOC3, thereby reducing triglycerides and restoring lipids to more normal levels.
In multiple clinical studies, investigational plozasiran demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. Plozasiran has demonstrated a favorable safety profile to date with treatment emergent adverse events reported that generally reflect the comorbidities and underlying conditions of the study populations.
Plozasiran is being investigated in the SUMMIT program of clinical studies, including the PALISADE Phase 3 study in patients with FCS, which recently completed, the SHASTA studies in patients with SHTG, and the MUIR and CAPITAN studies in patients with mixed hyperlipidemia.
Plozasiran has been granted Orphan Drug Designation and Fast Track Designation by the
About PALISADE Phase 3 Study
The PALISADE study (NCT05089084) is a Phase 3 placebo controlled study to evaluate the efficacy and safety of plozasiran in adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint of the study is percent change from baseline in fasting TG versus placebo at Month 10. A total of 75 subjects distributed across 39 different sites in 18 countries were randomized to receive 25 mg plozasiran, 50 mg plozasiran, or matching placebo once every three months. Participants who completed the randomized period were eligible to continue in a 2-part extension period, where all participants receive plozasiran.
About Plozasiran EAP
Arrowhead is committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. The company has established an expanded access program (EAP) for some individuals living with FCS. As with any investigational medicine that has not been approved by regulatory authorities, investigational plozasiran may or may not be effective in treating your diagnosis or condition, and there may be risks associated with its use. If you are a patient or caregiver wishing to know more about this plozasiran EAP for FCS, please discuss this EAP and all treatment options with your treating physician. If you are a treating physician and are seeking information about the plozasiran EAP or would like to request access for a patient, please contact EAP@arrowheadpharma.com.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
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- European Society of Cardiology Congress 2024
- https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy (accessed 9/9/24)
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
ir@arrowheadpharma.com
Investors:
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Brian Ritchie
212-915-2578
britchie@lifesciadvisors.com
Media:
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Kendy Guarinoni, Ph.D.
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Source: Arrowhead Pharmaceuticals, Inc
FAQ
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