Tradipitant

FDA Budget Cuts Linked to Drug Approval Delays as Small Biotech Faces PDUFA Postponement

24 days ago

• Recent government efficiency cuts at the FDA appear to be causing delays in drug approval timelines, with 66% of healthcare professionals surveyed expressing concern about potential PDUFA date postponements. • Stealth BioTherapeutics' treatment for rare Barth syndrome, elamipretide, has experienced an unexplained decision delay, with no typical safety or manufacturing concerns cited as reasons for the postponement. • Industry experts warn that while widespread disruption isn't yet evident, continued FDA resource constraints could significantly impact clinical trial oversight and create approval backlogs lasting years.

Vanda Pharmaceuticals Battles FDA Over Gastroparesis Drug Hearing Delays

a month ago

• 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.

Advancements in Acute Lymphocytic Leukemia (ALL) Treatment Landscape

5 months ago

• The FDA approved obecabtagene autoleucel (Aucatzyl) for adults with relapsed or refractory B-cell precursor ALL, marking a significant advancement in CAR T-cell therapy. • A pivotal clinical trial revealed transformative findings poised to change the standard of care for children with acute lymphoblastic leukemia (ALL), marking a milestone in pediatric oncology. • Pfizer's BESPONSA received FDA approval for pediatric patients aged 1 year and older with relapsed or refractory CD22-positive B-cell ALL, expanding treatment options for this population.

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

5 months ago

• 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 Reports Revenue Increase and Advances Pipeline in Q3 2024

7 months ago

• 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.

Guselkumab Approved for Ulcerative Colitis, Tradipitant Receives CRL: GI News from September 2024

8 months ago

• Guselkumab (Tremfya) received FDA approval for treating moderately to severely active ulcerative colitis, marking it as a novel dual-acting monoclonal antibody. • Vanda Pharmaceuticals' tradipitant received a Complete Response Letter from the FDA for treating gastroparesis symptoms, prompting the need for additional studies. • Research indicates that the effectiveness of fecal microbiota transplantation (FMT) for IBS may depend on the delivery method used. • A study found an association between upper gastrointestinal mucosal damage and an increased risk of developing Parkinson's disease.

Novo Nordisk's $1bn Oral Weight Loss Drug Shows Promise but Raises Questions in Phase IIa Trial

8 months ago

• Novo Nordisk's monlunabant, an oral cannabinoid receptor 1 inverse agonist acquired for $1bn, demonstrated statistically significant weight loss in a Phase IIa trial but raised concerns about optimal dosing. • The 10mg dose achieved a 7.1kg weight reduction compared to 0.7kg with placebo, but higher doses showed limited additional benefit while increasing dose-dependent gastrointestinal and neuropsychiatric side effects. • Investor confidence wavered with Novo Nordisk's stock dropping over 6% following the announcement, as the company plans a Phase IIb trial next year to better determine the optimal efficacy-to-safety ratio.

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

8 months ago

• 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.

FDA Nears Decisions on Vanda, Zevra, Heron, Merck, BMS, Sanofi/Regeneron

8 months ago

• Vanda Pharmaceuticals awaits FDA decision on tradipitant for gastroparesis, a condition affecting millions in the U.S., with a verdict expected by September 18. • Zevra Therapeutics anticipates potential approval of arimoclomol for Niemann-Pick disease type C (NPC) by September 21, following positive advisory committee support. • The FDA is set to decide on Heron Therapeutics' extended-release needle for Zynrelef by September 23, designed to simplify drug preparation and administration. • Merck seeks approval for Keytruda in pleural mesothelioma, with a decision due September 25, based on Phase II/III KEYNOTE-483 trial data showing improved survival. • Bristol Myers Squibb awaits FDA decision on KarXT for schizophrenia by September 26, potentially offering a novel mechanism of action targeting muscarinic receptors. • Sanofi and Regeneron anticipate a decision on Dupixent for COPD by September 27, supported by Phase III BOREAS and NOTUS trials demonstrating reduced exacerbations.

FDA Nears Decisions on Key Therapies for Gastroparesis, Niemann-Pick Disease, Schizophrenia, COPD, Mesothelioma, and Post-Surgical Pain

8 months ago

• The FDA is expected to decide on Vanda Pharmaceuticals' tradipitant for gastroparesis by September 18, potentially offering a novel NK-1R antagonist treatment option. • Zevra Therapeutics awaits a verdict by September 21 on arimoclomol for Niemann-Pick disease type C, an ultrarare neurodegenerative disorder with no approved therapies. • A decision is anticipated by September 26 on Bristol Myers Squibb's KarXT for schizophrenia, representing a new pharmacological approach targeting muscarinic receptors.

September 2024: FDA Actions Span Diabetes, Neurology, and Rare Diseases

8 months ago

• The FDA approved Embecta's tubeless patch pump for insulin delivery in adults with type 1 and type 2 diabetes, offering a 300-unit reservoir based on patient feedback. • Sparsentan (Filspari) received full FDA approval to slow kidney function decline in adults with primary IgA nephropathy (IgAN), based on positive Phase 3 PROTECT study data. • Approvals for arimoclomol (Miplyffa) and levacetylleucine (Aqneursa) mark the first specific treatments for neurological symptoms of Niemann-Pick disease type C (NPC). • Dupilumab (Dupixent) gained approval for COPD, chronic rhinosinusitis with nasal polyps, and was submitted for label expansion for adult growth hormone deficiency.

Vanda Pharmaceuticals Faces Challenges Despite New Drug Launches and Takeover Bids

9 months ago

• 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.

Drug Development Updates