The Director of the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA) issued a notice summarizing the grounds for refusing a new drug application (NDA) submitted by Vanda Pharmaceuticals, Inc. (NASDAQ:VNDA) for Tradipitant for symptoms in gastroparesis.

The notice offers Vanda an opportunity to request a hearing on the matter.

Also Read: Over 200% Stock Upside – Vanda Pharmaceuticals Undervalued With Robust Portfolio And Pipeline: Analyst

In September, the FDA issued a Complete Response Letter to the company. The agency disregarded the evidence provided and instead suggested that Vanda conduct additional studies with a design and duration inconsistent with the advice of key experts in the field and not appropriate based on the scientific understanding and natural course of the disorder. 

Last week, Vanda Pharmaceuticals wrote a letter to the FDA Commissioner highlighting faulty gastroparesis NDA review.

The FDA identified:

• In the 3301 Phase 3 Study (Group 1), the drug did not show a statistically significant improvement over placebo for nausea severity or other gastroparesis symptoms. Secondary measures results also lacked meaningful differences and sometimes even favored the placebo.
• 2301 Phase 2 Study: While it showed significant results for nausea at Week 4, the study had methodological flaws and inadequate data handling, reducing the reliability of its findings. Additionally, Study 3301 failed to confirm the results of Study 2301.
• Confirmatory Evidence: Post hoc analyses and pooled data from the studies were insufficient to support efficacy claims. Other sources, such as open-label studies and unrelated motion sickness trials, could not confirm the drug’s effectiveness.
• Safety: Data from trials were limited to 12 weeks, which is inadequate for a chronic condition like gastroparesis that requires long-term treatment. Animal and lab studies did not provide enough information to assess long-term safety risks for humans.

The FDA recommended conducting two new, well-designed clinical trials in adults with idiopathic or diabetic gastroparesis to address the deficiencies and perform a long-term toxicity study in a non-rodent species.

Vanda could either resubmit the application addressing all deficiencies, withdraw the application, or request a hearing.

In November 2024, Vanda received a formal notice of opportunity for a hearing.

Price Action: VNDA stock is down 2.45% at $4.34 at the last check on Thursday.

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