Applied Therapeutics Provides Regulatory Update on Govorestat for the Treatment of Classic Galactosemia
Applied Therapeutics (Nasdaq: APLT) provided a regulatory update on govorestat for the treatment of Classic Galactosemia. The company completed its late-cycle review meeting with the FDA, which no longer intends to hold an Advisory Committee meeting. The FDA's review of the New Drug Application (NDA) continues as planned, with the PDUFA target action date set for November 28, 2024.
The FDA communicated that the Priority Review of the NDA is proceeding as scheduled, with alignment on post-marketing requirements expected in October 2024. Applied Therapeutics expressed satisfaction with the ongoing collaborative dialogue with the FDA and remains committed to bringing the first potential treatment to Classic Galactosemia patients.
Applied Therapeutics (Nasdaq: APLT) ha fornito un aggiornamento normativo su govorestat per il trattamento della Galattosemia Classica. L'azienda ha completato l'incontro di revisione di fine ciclo con la FDA, che non intende più tenere un incontro del Comitato Consultivo. La revisione della Nuova Domanda di Farmaco (NDA) da parte della FDA continua come previsto, con la data di azione target PDUFA fissata per il 28 novembre 2024.
La FDA ha comunicato che la Revisione Prioritaria della NDA sta procedendo come programmato, con un allineamento sui requisiti post-marketing previsto per ottobre 2024. Applied Therapeutics ha espresso soddisfazione per il dialogo collaborativo in corso con la FDA e rimane impegnata a portare il primo potenziale trattamento ai pazienti con Galattosemia Classica.
Applied Therapeutics (Nasdaq: APLT) proporcionó una actualización regulatoria sobre govorestat para el tratamiento de la Galactosemia Clásica. La compañía completó su reunión de revisión de ciclo tardío con la FDA, que ya no tiene la intención de llevar a cabo una reunión del Comité Asesor. La revisión de la Nueva Solicitud de Medicamento (NDA) por parte de la FDA continúa según lo planeado, con la fecha de acción objetivo PDUFA establecida para el 28 de noviembre de 2024.
La FDA comunicó que la Revisión Prioritaria de la NDA está avanzando según lo programado, con un alineamiento en los requisitos post-comercialización esperado para octubre de 2024. Applied Therapeutics expresó satisfacción con el diálogo colaborativo en curso con la FDA y sigue comprometida a llevar el primer tratamiento potencial a los pacientes con Galactosemia Clásica.
Applied Therapeutics (Nasdaq: APLT)는 고전적 갈락토세미아 치료를 위한 govorestat에 대한 규제 업데이트를 제공했습니다. 이 회사는 FDA와의 후기 주기 리뷰 회의를 완료했으며, 더 이상 자문 위원회 회의를 개최할 계획이 없습니다. FDA의 신약 신청(NDA) 검토는 계획대로 진행되고 있으며, PDUFA 목표 조치 날짜는 2024년 11월 28일로 설정되어 있습니다.
FDA는 NDA의 우선 검토가 예정대로 진행 중이며, 2024년 10월에 사후 마케팅 요구 사항에 대한 조정이 예상된다고 전달했습니다. Applied Therapeutics는 FDA와의 지속적인 협력 대화에 만족을 표명하며 고전적 갈락토세미아 환자들에게 첫 잠재적 치료제를 제공하기 위해 최선을 다하고 있습니다.
Applied Therapeutics (Nasdaq: APLT) a fourni une mise à jour réglementaire concernant govorestat pour le traitement de la Galactosémie Classique. La société a terminé sa réunion de révision de fin de cycle avec la FDA, qui n'a plus l'intention de convoquer une réunion du Comité Consultatif. L'examen de la Demande de Nouveau Médicament (NDA) par la FDA se poursuit comme prévu, avec une date d'action cible PDUFA fixée au 28 novembre 2024.
La FDA a communiqué que l'examen prioritaire de la NDA avance comme prévu, avec un alignement sur les exigences post-commercialisation attendu en octobre 2024. Applied Therapeutics a exprimé sa satisfaction quant au dialogue collaboratif en cours avec la FDA et reste déterminée à apporter le premier traitement potentiel aux patients atteints de Galactosémie Classique.
Applied Therapeutics (Nasdaq: APLT) hat ein regulatorisches Update zu govorestat zur Behandlung der klassischen Galaktosämie bereitgestellt. Das Unternehmen hat sein Spätzyklus-Überprüfungstreffen mit der FDA abgeschlossen, die nicht mehr plant, ein Beratungsausschusstreffen abzuhalten. Die Überprüfung des New Drug Application (NDA) durch die FDA erfolgt wie geplant, wobei das PDUFA-Zielaktionsdatum auf den 28. November 2024 festgelegt ist.
Die FDA teilte mit, dass die Prioritätsüberprüfung des NDA planmäßig verläuft, mit einer Erwartung an die Anpassungen der Nachvermarktungsanforderungen im Oktober 2024. Applied Therapeutics äußerte Zufriedenheit mit dem laufenden kollaborativen Dialog mit der FDA und bleibt bestrebt, die erste potenzielle Behandlung für Patienten mit klassischer Galaktosämie zu entwickeln.
- FDA no longer requires an Advisory Committee meeting, potentially streamlining the approval process
- NDA review continues as planned with PDUFA target action date of November 28, 2024
- Priority Review status maintained for govorestat
- Potential to be the first approved treatment for Classic Galactosemia
- None.
Insights
The FDA's decision to cancel the Advisory Committee meeting for govorestat is a positive development for Applied Therapeutics. This suggests that the FDA may not have significant concerns about the drug's safety or efficacy that would require external expert input. The maintained PDUFA date of November 28, 2024, indicates that the review process is progressing smoothly.
Classic Galactosemia, a rare genetic disorder affecting galactose metabolism, currently has no FDA-approved treatments. If approved, govorestat would be a first-in-class therapy, potentially addressing a significant unmet medical need. The Priority Review status further underscores the drug's importance.
However, investors should note that while these developments are encouraging, FDA approval is never guaranteed. The company's focus on post-marketing requirements suggests they are preparing for potential approval, but also highlights the need for ongoing safety monitoring post-launch.
Applied Therapeutics' regulatory update on govorestat is potentially market-moving. The cancellation of the Advisory Committee meeting and the maintained PDUFA date reduce regulatory uncertainty, which could positively impact the stock price.
As a small-cap biotech (
However, investors should be cautious. The company reported only
The potential approval of govorestat represents a significant market opportunity for Applied Therapeutics. Classic Galactosemia affects approximately 1 in 50,000 newborns in the United States, creating a niche but potentially lucrative market for an effective treatment.
The company's focus on commercial preparation, including patient access programs and payor engagement, is crucial. Rare disease treatments often command premium pricing, but securing reimbursement can be challenging. Applied Therapeutics' proactive approach to these issues could facilitate a smoother market entry if govorestat is approved.
However, the success of govorestat will depend on factors beyond just FDA approval. Physician education and patient identification will be critical, given the rarity of the condition. The company's ability to execute on these fronts will be key to realizing the full market potential of govorestat.
- Company completed Late-Cycle review meeting with FDA
- FDA no longer intends to hold Advisory Committee meeting
- FDA Review of NDA continues to proceed as planned with PDUFA target action date of November 28, 2024
NEW YORK, Sept. 18, 2024 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced an update on the ongoing New Drug Application (NDA) review of govorestat for the treatment of Classic Galactosemia. The Company recently completed its late-cycle review meeting with the United States Food and Drug Administration (FDA). The FDA communicated that an Advisory Committee meeting would no longer be required, which was previously tentatively scheduled for October 9, 2024. The FDA informed the Company that the Priority Review of the NDA is continuing as planned with alignment on post-marketing requirements expected in October 2024. The previously announced Prescription Drug User Fee Act (PDUFA) target action date remains on track for November 28, 2024.
“We are incredibly pleased by the ongoing collaborative dialogue with the FDA during the NDA review process, and we look forward to continuing to work together with the agency to bring the first potential treatment to Classic Galactosemia patients,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “Galactosemia is a progressive disease in urgent need of treatment, and the potential approval of govorestat will be transformative for the many patients and families living with this serious disease. Our commitment to the Classic Galactosemia community is further supported by our thoughtful commercial preparation, focused on establishing an effective patient access program, high physician awareness and strong payor engagement.”
About Galactosemia
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). There are approximately 3,000 patients with Galactosemia in the US and 80 new births per year, and approximately 4,000 patients with Galactosemia in the EU and 120 new births per year. Newborn screening for Galactosemia is mandatory in the US and most EU countries, leading to rapid identification of affected patients.
About Govorestat (AT-007)
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG.
In a study in children with Galactosemia aged 2-17, treatment with govorestat demonstrated clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremor. Govorestat also significantly reduced plasma galactitol levels in both adults and children with Galactosemia. Galactitol is a toxic metabolite responsible for tissue damage and long-term complications in Galactosemia.
In the Phase 3 INSPIRE trial in patients with SORD Deficiency, an interim analysis at 12 months demonstrated a statistically significant reduction in blood sorbitol levels with govorestat treatment as compared to placebo, as well as a statistically significant correlation of sorbitol with change in clinical outcomes, such as 10-meter walk run test, dorsiflexion and 6-minute walk test. Govorestat also demonstrated highly statistically significant effects on the CMT Health Index (CMT-HI) patient reported outcome measure, with benefit of govorestat shown on lower limb function, mobility, fatigue, pain, sensory function, and upper limb function.
Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and SORD Deficiency. Govorestat has also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia, PMM2-CDG, and SORD Deficiency; Pediatric Rare Disease designation for Galactosemia and PMM2-CDG; and Fast Track designation for Galactosemia.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company’s lead drug candidate, govorestat, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy.
To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @Applied_Tx.
Forward-Looking Statements
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Such risks and uncertainties include, without limitation, (i) our plans to develop, market and commercialize our product candidates, (ii) the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs, (iii) our ability to take advantage of expedited regulatory pathways for any of our product candidates, (iv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing, (v) our ability to successfully acquire or license additional product candidates on reasonable terms and advance product candidates into, and successfully complete, clinical studies, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain and timing of regulatory approval of our current and future product candidates, (viii) the anticipated indications for our product candidates, if approved, (ix) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (x) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (xi) the implementation of our business model and strategic plans for our business and product candidates, (xii) our intellectual property position and the duration of our patent rights, (xiii) developments or disputes concerning our intellectual property or other proprietary rights, (xiv) our expectations regarding government and third-party payor coverage and reimbursement, (xv) our ability to compete in the markets we serve, (xvi) the impact of government laws and regulations and liabilities thereunder, (xvii) developments relating to our competitors and our industry, (xviii) our ability to achieve the anticipated benefits from the agreements entered into in connection with our partnership with Advanz Pharma and (xiv) other factors that may impact our financial results. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur at all. 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 our filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
Contacts
Investors:
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646-970-0543
appliedtherapeutics@argotpartners.com
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