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Novartis’ LUTATHERA for GEP-NET Treatment: Ray of Hope for Pediatric Patients
2024 could be an eventful year for LUTATHERA and radioligand therapies (RLTs) around their future commercial prospects. On 23rd April 2024, the FDA approved LUTATHERA (also known as lutetium Lu 177 dotatate) for the treatment of pediatric patients of 12 years and older with somatostatin receptor (SSTR)-positive gastro-entero-pancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors. Somatostatin receptor type 2 (SSTR2) is expressed in 80-90% of GEP-NET tumors. LUTATHERA has already received approval for GEP-NET treatment for adults in 2018. The recent approval was based on pharmacokinetic (PK), dosimetry, and…
Analyzing the Potential of AAV2-GDNF Gene Therapy in Parkinson’s Disease Treatment: Insights from Phase Ib Trial Data
AB-1005 represents a pioneering gene therapy utilizing an adeno-associated viral vector serotype 2 (AAV2) to deliver the human glial cell line-derived neurotrophic factor (GDNF) transgene directly into the brain. This innovative technique allows for precise and sustained expression of GDNF, offering promising therapeutic potential for conditions like Parkinson’s disease characterized by the degeneration of midbrain dopaminergic neurons. GDNF, a member of the transforming growth factor-β superfamily, has demonstrated remarkable neuroprotective effects in preclinical studies. By promoting the survival and differentiation of dopaminergic neurons in the midbrain, GDNF holds the promise…
LIBERVANT Buccal Film: Pioneering Pediatric Epilepsy Treatment Advancements
LIBERVANT, a diazepam buccal formulation based on PharmFilm technology serves as a beacon of hope, offering convenient alternatives to existing device-based products, for managing acute repetitive seizure emergencies in young patients. LIBERVANT utilizes "fast-melt strips" for "buccal delivery of diazepam, offering promises to rewrite the narrative of acute repetitive seizure emergencies, ushering in an era of renewed optimism and therapeutic advancement. At the 76th Annual Meeting of the American Academy of Neurology (AAN) 2024 in Denver from April 13 to 18, Aquestive Therapeutics presented an abstract for the crossover study data evaluating the…