Vertex Announces FDA Acceptance of New Drug Application for Suzetrigine for the Treatment of Moderate-to-Severe Acute Pain
Vertex Pharmaceuticals (Nasdaq: VRTX) announced that the FDA has accepted its New Drug Application (NDA) for suzetrigine, an investigational non-opioid pain signal inhibitor for moderate-to-severe acute pain. The FDA granted priority review with a PDUFA target action date of January 30, 2025. Suzetrigine, already granted Fast Track and Breakthrough Therapy designations, could be the first new class of acute pain medicine in over 20 years.
The drug aims to fill the gap between well-tolerated but less effective medicines and opioids with known risks. It has the potential to treat millions of patients suffering from acute pain annually in the U.S., addressing a critical unmet need in pain management.
Vertex Pharmaceuticals (Nasdaq: VRTX) ha annunciato che la FDA ha accettato la sua Nuova Richiesta di Farmaco (NDA) per suzetrigine, un inibitore sperimentale non oppioide del segnale del dolore per il dolore acuto da moderato a severo. La FDA ha concesso una revisione prioritaria con una data di azione prevista PDUFA del 30 gennaio 2025. Suzetrigine, già designato come Fast Track e Terapia Innovativa, potrebbe essere il primo nuovo tipo di farmaco per il dolore acuto in oltre 20 anni.
Il farmaco mira a colmare il divario tra farmaci ben tollerati ma meno efficaci e oppioidi con rischi noti. Ha il potenziale di trattare milioni di pazienti che soffrono di dolore acuto ogni anno negli Stati Uniti, affrontando un bisogno critico non soddisfatto nella gestione del dolore.
Vertex Pharmaceuticals (Nasdaq: VRTX) anunció que la FDA ha aceptado su Nueva Solicitud de Medicamento (NDA) para suzetrigina, un inhibidor experimental no opioide de la señal del dolor para el dolor agudo de moderado a severo. La FDA otorgó una revisión prioritaria con una fecha de acción objetivo PDUFA del 30 de enero de 2025. Suzetrigina, que ya ha recibido las designaciones de Vía Rápida y Terapia Innovadora, podría ser la primera nueva clase de medicamento para el dolor agudo en más de 20 años.
El medicamento tiene como objetivo llenar el vacío entre medicamentos bien tolerados pero menos efectivos y opioides con riesgos conocidos. Tiene el potencial de tratar a millones de pacientes que sufren de dolor agudo anualmente en Estados Unidos, abordando una necesidad crítica no satisfecha en el manejo del dolor.
버텍스 제약회사(Nasdaq: VRTX)는 FDA가 수제트리진에 대한 새로운 의약품 신청(NDA)을 승인했다고 발표했습니다. 수제트리진은 중등도에서 중증의 급성 통증에 대한 연구 중인 비오피오이드 통증 신호 억제제입니다. FDA는 우선 심사를 허가했으며, PDUFA 목표 조치일은 2025년 1월 30일입니다. 수제트리진은 이미 신속 승인 및 혁신 치료 지정을 받았으며, 20년 이상 만에 새로운 급성 통증 치료제의 첫 번째 클래스가 될 수 있습니다.
이 약물은 잘 견딜 수 있지만 효능이 낮은 약물과 알려진 위험이 있는 오피오이드 간의 간극을 메우는 것을 목표로 합니다. 이 약물은 미국에서 매년 급성 통증으로 고통 받는 수백만 환자를 치료할 잠재력을 가지고 있으며, 통증 관리에서 매우 중요한 충족되지 않은 필요를 해결하고자 합니다.
Vertex Pharmaceuticals (Nasdaq: VRTX) a annoncé que la FDA a accepté sa Demande de Médicament Nouveau (NDA) pour suzetrigine, un inhibiteur expérimental non opioïde des signaux de douleur pour des douleurs aiguës modérées à sévères. La FDA a accordé une révision prioritaire avec une date d'action cible PDUFA du 30 janvier 2025. Suzetrigine, déjà désigné comme traitement accéléré et thérapie innovante, pourrait être la première nouvelle classe de médicaments contre la douleur aiguë en plus de 20 ans.
Le médicament vise à combler le fossé entre des médicaments bien tolérés mais moins efficaces et des opioïdes aux risques connus. Il a le potentiel de traiter des millions de patients souffrant de douleurs aiguës chaque année aux États-Unis, répondant à un besoin critique non satisfait dans la gestion de la douleur.
Vertex Pharmaceuticals (Nasdaq: VRTX) gab bekannt, dass die FDA ihren Antrag auf Zulassung eines neuen Medikaments (NDA) für Suzetrigine angenommen hat, einem experimentellen nicht-opioiden Schmerzsignalhemmer für akute Schmerzen von mäßiger bis starker Intensität. Die FDA gewährte eine Prioritätsprüfung mit einem angestrebten PDUFA-Aktionsdatum am 30. Januar 2025. Suzetrigine, das bereits die Fast-Track- und Durchbruchtherapie-Status erhalten hat, könnte die erste neue Klasse von akuten Schmerzmitteln seit über 20 Jahren sein.
Das Medikament zielt darauf ab, die Lücke zwischen gut verträglichen, aber weniger effektiven Medikamenten und Opioiden mit bekannten Risiken zu schließen. Es hat das Potenzial, jährlich Millionen von Patienten in den USA zu behandeln, die unter akutem Schmerz leiden, und spricht ein kritisches, nicht erfülltes Bedürfnis im Schmerzmanagement an.
- FDA accepted New Drug Application for suzetrigine
- FDA granted priority review for suzetrigine
- PDUFA target action date set for January 30, 2025
- Suzetrigine could be first new class of acute pain medicine in over 20 years
- Potential to treat millions of patients with moderate-to-severe acute pain annually
- None.
Insights
The FDA's acceptance of Vertex Pharmaceuticals' New Drug Application (NDA) for suzetrigine, with priority review status, is a significant development in the pain management sector. This non-opioid pain signal inhibitor could potentially disrupt the $4.5 billion acute pain market.
Key points to consider:
- The PDUFA date of January 30, 2025, indicates a potentially swift approval process, which could lead to earlier-than-expected market entry.
- Suzetrigine's designation as a new class of medicine for acute pain treatment in over two decades underscores its innovative nature, potentially commanding premium pricing.
- The Fast Track and Breakthrough Therapy designations highlight the FDA's recognition of suzetrigine's potential to address a significant unmet medical need, which could translate to strong market adoption upon approval.
From an investor's perspective, this development could significantly impact Vertex's revenue streams. If approved, suzetrigine could diversify Vertex's portfolio beyond its core cystic fibrosis franchise, potentially mitigating risk and driving growth. However, it's important to monitor the competitive landscape and potential regulatory hurdles as we approach the PDUFA date.
The development of suzetrigine as a selective NaV1.8 pain signal inhibitor represents a significant advancement in pain management research. This novel mechanism of action targets a specific sodium channel involved in pain signaling, potentially offering a more targeted approach to pain relief compared to current options.
Key scientific implications:
- The selectivity for NaV1.8 channels may result in a better safety profile compared to broader-acting analgesics, potentially reducing off-target effects.
- As a non-opioid alternative, suzetrigine could address the critical need for effective pain management without the risks associated with opioid use, including addiction and respiratory depression.
- The potential efficacy in moderate-to-severe acute pain suggests that suzetrigine might bridge the gap between over-the-counter analgesics and opioids, offering a new option for patients and healthcare providers.
While the clinical data supporting the NDA submission appears promising, it will be important to scrutinize the full efficacy and safety profile once the data is made public. The medical community will be particularly interested in how suzetrigine compares to existing treatments in terms of pain relief, duration of action and side effect profile.
The FDA's acceptance of Vertex's NDA for suzetrigine with priority review status signals a potential shift in pain management policy. This development aligns with ongoing efforts to address the opioid crisis while ensuring adequate pain control for patients.
Policy implications to consider:
- If approved, suzetrigine could influence prescribing guidelines for acute pain management, potentially reducing reliance on opioids as first-line treatments.
- The introduction of a new non-opioid option may impact insurance coverage and reimbursement policies for pain medications, potentially favoring safer alternatives.
- Successful launch of suzetrigine could accelerate research and development in non-opioid pain management, leading to increased investment and innovation in this space.
From a public health perspective, the availability of a new non-opioid pain medication could contribute to efforts in reducing opioid prescriptions and associated risks. However, it will be important to monitor post-market surveillance data to ensure that any unforeseen risks are promptly identified and addressed.
Policymakers and healthcare systems will likely closely watch the adoption and impact of suzetrigine, as its success could inform future strategies in pain management and opioid crisis mitigation efforts.
– FDA grants priority review and assigns a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2025 –
– Suzetrigine, an investigational non-opioid pain signal inhibitor, has the potential to treat millions of patients who suffer from moderate-to-severe acute pain each year –
The FDA has granted suzetrigine priority review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2025. Suzetrigine has already been granted FDA Fast Track and Breakthrough Therapy designations for the treatment of moderate-to-severe acute pain.
“Today’s FDA filing acceptance for suzetrigine marks a critical milestone toward bringing this new, transformative non-opioid analgesic to the millions of patients suffering from moderate-to-severe acute pain each year in the U.S.,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. “The FDA’s granting of a priority review further reinforces the high unmet need in treating acute pain, and the filing brings us one step closer to our objective of filling the gap between medicines with good tolerability but limited efficacy and opioid medicines with therapeutic efficacy but known risks, including addictive potential.”
"In my 24 years practicing medicine, I have seen firsthand the desperate need for new non-opioid therapies for treating pain. Too many people today are either undertreated, dealing with negative side effects of currently available therapies or foregoing pain medications altogether for fear of becoming dependent on opioids,” said Scott Weiner, M.D., M.P.H., Vertex Acute Pain Steering Committee Chair, Associate Professor of Emergency Medicine at Harvard Medical School and Attending Emergency Physician in the Department of Emergency Medicine at Brigham and Women’s Hospital. “Prescribers and patients deserve new options."
About Acute Pain
Acute pain is a disabling condition and is defined as pain lasting less than 3 months. It is estimated that over 80 million people are prescribed a medicine for acute pain every year in the
About Suzetrigine (VX-548)
Suzetrigine (formerly VX-548) is an investigational oral, selective NaV1.8 pain signal inhibitor that is highly selective for NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated sodium channel that is selectively expressed in peripheral pain-sensing neurons (nociceptors), where its role is to transmit pain signals (action potentials). NaV1.8 is a genetically validated target for the treatment of pain, and suzetrigine has demonstrated a favorable benefit/risk profile in three Phase 3 studies and two Phase 2 studies in patients with moderate-to-severe acute pain. Suzetrigine also demonstrated positive results and a well-tolerated profile in a Phase 2 study in patients with pain associated with diabetic peripheral neuropathy, a type of chronic peripheral neuropathic pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of pain signal inhibitors that have the potential to provide effective relief of pain without the limitations of currently available therapies, including the addictive potential of opioids.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Nia Tatsis, Ph.D., and Scott Weiner, M.D., M.P.H., in this press release, and statements regarding our expectations for the benefits of and potential for suzetrigine as a treatment for moderate-to-severe acute pain. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, that future competitive or other market factors may adversely affect the commercial potential for suzetrigine, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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FAQ
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