MedPath

Tasimelteon

Generic Name
Tasimelteon
Brand Names
Hetlioz
Drug Type
Small Molecule
Chemical Formula
C15H19NO2
CAS Number
609799-22-6
Unique Ingredient Identifier
SHS4PU80D9
Background

Tasimelteon is a selective dual melatonin receptor agonist indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (N24HSWD). Occurring commonly in blind individuals without light perception, this condition is often characterized by periods of night-time insomnia and day-time sleepiness. In blind individuals, a lack of light stimulation causes an extension of the 24-hour circadian cycle and can lead to progressively delayed sleep onset. By activating melatonin receptors MT1 and MT2 in the suprachiasmatic nucleus of the brain, tasimelteon has been shown to improve sleep by resynchronizing the circadian rhythm through its "non-photic" mechanism. Tasimelteon is currently the only drug available for the treatment of N24HSWD and was granted orphan drug status by the FDA in 2010.

Indication

Tasimelteon oral capsules are indicated for the treatment of non-24 hour sleep-wake disorder in adult patients and for the treatment of nighttime sleep disturbances in Smith-Magenis Syndrome in patients ≥16 years old. Tasimelteon oral suspension is indicated for the treatment of nighttime sleep disturbances in Smith-Magenis syndrome in patients 3 to 15 years of age.

Associated Conditions
Non 24 Hour Sleep Wake Disorder, Sleep Disturbance

Vanda Pharmaceuticals Battles FDA Over Gastroparesis Drug Hearing Delays

• Vanda Pharmaceuticals has accused FDA officials of unlawfully delaying a hearing on its gastroparesis drug tradipitant, claiming the agency is using recent staff reductions as a false excuse for a six-month postponement. • The company alleges FDA bureaucrats have systematically avoided scrutiny by denying all new drug approvability hearing requests for at least a decade, calling for Commissioner Makary to intervene. • The dispute centers on tradipitant for gastroparesis, which the FDA rejected in September 2024, claiming the drug failed to demonstrate statistically significant treatment effects.

FDA Rejects Vanda's Hetlioz Application for Insomnia Treatment Due to Insufficient Trial Data

• FDA Acting Commissioner Sara Brenner has denied Vanda Pharmaceuticals' request for a hearing on Hetlioz (tasimelteon) for sleep-onset insomnia, citing lack of adequate trial data. • The rejection highlights the FDA's strict requirement for well-controlled clinical trials in demonstrating drug efficacy for new indications. • The decision marks a significant setback for expanding Hetlioz's therapeutic applications beyond its current approved uses.

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Vanda Pharmaceuticals Challenges FDA Rejection of Tradipitant for Gastroparesis, Seeks Hearing

• The FDA issued a Complete Response Letter for Vanda Pharmaceuticals' tradipitant, a neurokinin-1 receptor antagonist, for treating gastroparesis, citing insufficient evidence of efficacy. • Vanda disputes the FDA's assessment, asserting that tradipitant has demonstrated substantial evidence of efficacy and a favorable benefit-risk profile in clinical studies. • Vanda has formally accepted the FDA's offer for a hearing to discuss the tradipitant NDA, aiming to address the concerns raised by the agency and advocate for approval. • The D.C. Circuit Court of Appeals upheld the FDA's denial of fast-track approval for tradipitant, supporting the agency's right to consider the drug's development plan.

Vanda Pharmaceuticals Navigates Hetlioz Sales Decline with Expansion Efforts

• Vanda Pharmaceuticals reported a 28% drop in Hetlioz sales due to generic competition, despite a slight increase in Q3 sales. • The company is pursuing FDA approvals for Hetlioz to treat jet lag and insomnia, and has launched a program for pediatric insomnia. • Vanda is actively defending its Hetlioz patent in court with a trial set for 2026, and awaits EMA decision for Smith-Magenis Syndrome in 2025.

Vanda Pharmaceuticals Reports Revenue Increase and Advances Pipeline in Q3 2024

• Vanda Pharmaceuticals reported a 23% increase in Q3 2024 revenues, reaching $47.7 million, driven by sales of Fanapt, HETLIOZ, and PONVORY. • The company is advancing its psychiatry portfolio, with plans to submit an NDA for milsaperidone for schizophrenia and bipolar I disorder in early 2025. • Vanda initiated the commercial launch of PONVORY for multiple sclerosis and is exploring its use in psoriasis and ulcerative colitis with IND submissions expected in Q4 2024. • A New Drug Application for tradipitant in motion sickness is expected to be submitted in Q4 2024, building on positive clinical trial results.

FDA Rejects Vanda's Tradipitant for Gastroparesis, Citing Insufficient Evidence

• The FDA issued a Complete Response Letter for Vanda Pharmaceuticals' tradipitant, a neurokinin-1 receptor antagonist, for treating gastroparesis symptoms. • The FDA's decision was based on the need for additional studies, which Vanda argues are inconsistent with expert advice and current understanding of gastroparesis. • Vanda plans to continue pursuing marketing authorization for tradipitant and will proceed with a separate NDA submission for motion sickness prevention. • Gastroparesis, affecting both diabetic and non-diabetic individuals, lacks effective treatments, making this rejection a setback for patients.

Vanda Pharmaceuticals Faces Challenges Despite New Drug Launches and Takeover Bids

• Vanda Pharmaceuticals' Fanapt, approved for bipolar 1 disorder, faces challenges due to competition from generic antipsychotics with established physician experience. • Tradipitant's Phase 3 trial for gastroparesis failed to meet primary endpoints, and motion sickness treatment faces competition from affordable generics like scopolamine. • Despite takeover bids, Vanda rejected offers, citing undervaluation, raising concerns about cash management and strategic direction given pipeline challenges. • Vanda's internally developed Hetlioz is experiencing revenue decline due to generic competition, impacting overall financial performance.
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