Applied Therapeutics Announces MAA Validation and NDA Submission of Govorestat (AT-007) for Treatment of Classic Galactosemia
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Insights
The submission of a New Drug Application (NDA) and a Marketing Authorization Application (MAA) for govorestat represents a significant milestone for Applied Therapeutics, Inc. as it transitions from the clinical development phase to the potential commercialization of its drug candidate. Govorestat's focus on Classic Galactosemia, a condition with no current treatments, addresses a clear unmet medical need. The positive outcomes from the ACTION-Galactosemia Kids study suggest a potential paradigm shift in the management of this rare disease.
The market for orphan drugs, such as govorestat, is often characterized by lower competition and higher pricing flexibility, which can result in substantial profit margins. However, the small patient population—over 3,000 in the U.S.—may limit revenue potential. Moreover, the favorable safety profile, if confirmed by regulatory agencies, could facilitate market penetration and adoption, providing a competitive edge.
Long-term implications for stakeholders include the potential for govorestat to become a reference treatment for Galactosemia, encouraging further research and investment in the field. The company's valuation could significantly benefit from the approval and successful commercialization of the drug, with a likely positive impact on stock performance contingent upon regulatory decisions and market acceptance.
Applied Therapeutics' announcement is a critical inflection point for the company's financial trajectory. The market's reaction to the NDA and MAA submissions will be closely tied to investor confidence in govorestat's approval and commercial prospects. The biopharmaceutical sector is known for its high-risk, high-reward nature and the success of a single product can have a disproportionate effect on a company's financial health, especially for a clinical-stage company like Applied Therapeutics.
Investors will be monitoring the FDA's 60-day filing review period and the EMA's review process, as these will provide early indicators of govorestat's regulatory prospects. Approval in both the U.S. and European markets would not only validate the company's research and development capabilities but also open up significant revenue streams. The company's stock price could see volatility during this period, with potential spikes upon positive news or declines if regulatory hurdles are encountered.
It is important to note that the biotech industry is subject to rapid changes in investor sentiment, often driven by clinical trial outcomes and regulatory milestones. Applied Therapeutics' future funding needs and partnership opportunities may also be influenced by the perceived potential of govorestat, impacting the company's strategic financial planning.
The therapeutic landscape for rare diseases like Classic Galactosemia is often underserved, presenting opportunities for companies like Applied Therapeutics to capitalize on first-mover advantages. The market potential for govorestat hinges on several factors, including market exclusivity periods, insurance coverage and the ability of the company to establish effective distribution channels.
Given the rarity of Galactosemia, the company may adopt a targeted marketing strategy, focusing on specialized treatment centers and advocacy groups such as the Galactosemia Foundation. The endorsement by patients and caregivers, as evidenced by Brittany Cudzilo's testimonial, can be pivotal in driving early adoption and creating a positive brand image.
Additionally, the company's strategy in setting the price for govorestat will be critical. Orphan drugs often command high prices due to limited competition and the high cost of development, but they must balance this with the need for accessibility and ethical considerations. Market access negotiations with payers will be crucial in determining the commercial success of govorestat.
NDA submitted to FDA and MAA validated by EMA in December 2023
Submissions are based on positive results from Phase 3 registrational ACTION-Galactosemia Kids study demonstrating consistent long-term clinical outcomes benefit across a range of functional measures and favorable safety profile
NEW YORK, Jan. 03, 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 it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for govorestat (AT-007) for the treatment of Classic Galactosemia. The NDA was submitted in December 2023. In addition, the Company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the fourth quarter of 2023, which was subsequently validated and accepted for review in December 2023.
“The submissions of both the NDA and MAA for govorestat are supported by rapid and sustained reduction in galactitol, which resulted in a meaningful benefit on clinical outcomes across pediatric patients, alongside a favorable safety profile,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “We look forward to working closely with both regulatory agencies throughout the review process and hope to bring the first treatment to patients with Galactosemia soon.”
“There are currently no treatments available for Galactosemia, a serious progressive disease affecting over 3,000 patients in the United States,” said Brittany Cudzilo, Vice President of the Galactosemia Foundation. “Govorestat offers hope to many patients and families affected by Galactosemia. I saw the positive impact of govorestat first-hand in my daughter, Ansell, who participated in the ACTION-Galactosemia Kids pediatric study. As a community, we will do everything possible to ensure that the regulatory agencies understand the importance of approving this treatment for our loved ones with Galactosemia.”
The NDA and MAA submission packages include clinical outcomes data from the Phase 3 registrational ACTION-Galactosemia Kids study in children age 2-17 with Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review. The MAA has been validated and will move to review by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
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 AT-007 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.
Govorestat is also being studied in the ongoing Phase 3 INSPIRE trial, which is evaluating the effect of AT-007 vs. placebo in patients with SORD Deficiency on sorbitol reduction as well as clinical outcomes in approximately 50 patients aged 16-55 in the U.S. and Europe. In an interim analysis, AT-007 reduced sorbitol by a mean of
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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Contacts
Investors:
Maeve Conneighton
(212) 600-1902 or
appliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
FAQ
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